>> GOOD MORNING. WELCOME, EVERYONE, TO THE SECOND DAY OF THE AUGUST 2016 ADVISORY COMMITTEE ON HERITABLE DISORDERS IN NEWBORN CHILDREN MEETING. WE'LL START BY DOING A ROLL CALL. DON BAILEY. >> I AM HERE. >> KAY BAKER. >> HERE. >> JEFF RUSCO. >> HERE. >> CARLA CUTHBERT. >> HERE. >> FRED LORI. >> HERE. >> STEVE MCDONNELL. >> HERE. >> MELISSA PERISI. >> HERE. >> ANNA MARIE SARININ. I KNOW SHE'S HERE. >> HERE. >> BETH TARINI. >> HERE. >> CATHY IS NOT ABLE TO BE HERE TODAY. DEBBIE SARCAR. >> HERE. >> ORGANIZATIONAL REPRESENTATIVES. ROBERT OSTRANDER. >> HERE. >> MICHAEL WATSON. >> HERE. >> THIS JOSEPH VICCIO BY PHONE. SUSAN TANKSLEY. >> HERE. >> ADAM CANNIS BY PHONE. >> HERE. >> NATASHA MONHOM. >> HERE. >> DINA DOYLE. >> HERE. >> KATE BUCKLEY. >> HERE. >> CAROL GREEN. >> HERE. >> THANK YOU ALL. >> TODAY WE'RE GOING TO START WITH A COUPLE OF PRESENTATIONS RELATED TO NEWBORN SCREENING TIMELINESS. AND FIRST WE HAVE YVONNE KELLER GUENTHER WHO WILL DISCUSS NEWBORN SCREENING TIMELINESS, COLLABORATIVE IMPROVEMENT INNOVATION NETWORK THE COIIN NETWORK. DR. KELLAR-GUENTHER IS AN ASSOCIATE PROFESSOR AT THE COLORADO SCHOOL OF PUBLIC HEALTH. HE'S PROGRAM EVALUATOR FOR NEW STEPS AND ASSOCIATE DIRECTOR FOR NEW STEPS 360, BOTH HRSA FUNDED PROJECTS. SHE WAS ALSO LEAD FOR NEW STEPS TIMELINESS COIIN INITIATIVE. IN ADDITION TO WORK WITH NEW STEPS DR. KELLAR-GUENTHER IS THE PROGRAM EVALUATOR ON SEVERAL PUBLIC HEALTH PROJECTS AND TEACHES PROGRAM EVALUATION AT CSPH. WELCOME. LOOK FORWARD TO YOUR PRESENTATION. >> THANK YOU. SO THANKS FOR INVITING ME TO SPEAK THIS MORNING. I'M VERY EXCITED AND HONORED TO SHARE WITH YOU OUR -- THE WORK WE DID AS PART OF COIIN AND ALSO TELL YOU ABOUT SOME OF THE OTHER TIMELINESS WORK WE'RE DOING AT NEW STEPS. FIRST I'M GOING TO START WITH WHAT IS A COIIN? IT'S A LOVELY ACRONYM, I FORGET IT EACH TIME. SO IT'S COLLABORATIVE IMPROVEMENT AND INNOVATION NETWORK. THE IDEA COIIN IS A LEARNING COLLABORATIVE SO WE BROUGHT TOGETHER SEVEN STATES THAT HAVE TO SHARE, SHARE RESOURCES, THEY SHARE SUCCESS BUTLY ALSO SHARE FAILURE SO THE OTHER PART OF COIIN, EMPHASIS IS ON QUALITY IMPROVEMENT NOT QUALITY ASSURANCE SO TOGETHER WE LEARN, WE LEARNED FROM WHAT'S GOING ON, WHAT'S NOT GOING ON. WHAT'S GOING WELL AND NOT AND THE OTHER PART OF COIIN, WE USE TECHNOLOGY SO WE MET VIA TELECONFERENCE BUT WE STARTED MIGHT FACE TO FACE. IF YOU'RE GOING TO TELL PEOPLE WHAT'S NOT GOING WELL OR NOT WORKING YOU NEED TO SEE EACH OTHER TO GET TRUST IN SOME RELATIONSHIPS GOING. SO THIS WAS A 15 MONTH PROGRAM. AND IT WAS UNFUNDED FOR THE STATES. SO THEY PUT IN AN APPLICATION, SAID YES, PLEASE, I WOULD LIKE TO DO WORK WITH YOU BUT UNFUNDED FOR 15 MONTHS. SO WE HAD SEVEN STATES THAT JOINED US. AND WE HAD -- WE REQUIRED THE STATES TO HAVE TEAMS AND THREE TO FIVE PEOPLE BUT WE REQUIRED AN INTEREST DISCIPLINARY APPROACH SO WE HAD TO HAVE A NEWBORN SCREENING LABORATORY, WE HAD TO HAVE NEWBORN SCREENING FOLLOW-UP AND HAD TO HAVE A HOSPITAL INVOLVED SO WE HAVE BEEN HEARING THROUGHOUT THIS MEETING NEWBORN SCREENING IS A SYSTEM, WE'RE VERY INTERESTED IN HAVING THE PARTS OF THAT SYSTEM THERE AS PART OF THE TEAM. SO THESE SHOULD LOOK VERY FAMILIAR. THESE ARE THE TIMELINESS RECOMMENDATIONS PUT FORTH BY THIS COMMITTEE. THESE CAME OUT A MONTH AFTER WE STARTED. BUT WE STILL MOST BENCHMARKS I WILL TELL YOU WHERE WE DEVIATED FROM WHAT YOU SUGGESTED. BUT WE WERE LOOKING AT ACTIVITIES THAT WOULD IMPROVE THE PERCENTAGE OF CHILDREN WHOSE REPORT -- WHOSE FINDINGS WERE REPORTED OUT FIVE DAYS OF LIFE FOR CRITICAL CONDITIONS, NO LATER THAN SEVEN DAYS OF LIFE FOR REPORTS FOR NEWBORN SCREENING. THESE ARE THE OTHER PARTS OF THE RECOMMENDATIONS, SO WE'RE VERY INTERESTED IN HOW PEOPLE COULD COLLECT -- GET THE COLLECTION FROM 48 HOURS OF BIRTH, GET THAT FIRST BLOOD SPOT COLLECTED, U AND HOW THEY CAN BE CONCEIVED AT THAT TIME LAB. SO WE DIDN'T GO 24 HOURS COLLECTION, 48 HOURS COLLECTION, THAT'S ONE PLACE WE DEVIATED. WE USE THE NEWBORN SCREENING NEW STEPS QUALITY IMPROVEMENT INDICATORS TO LOOK AT THE DIFFERENT PIECES OF THE SYSTEM. SO WE WERE INTERESTED IN TIME IT TOOK FROM BIRTH TO COLLECTION WITH EMPHASIS ON 48 HOURS. WE WERE INTERESTED IN THE TIME IT TOOK SPECIMEN COLLECTION TO RECEIPT BY LAB WE USE 48 HOURS. WE ARE INTERESTED IN TIME TO SPECIMEN RECEIPT TO REPORTING OUT COMPLETE RESULTS AND OF COURSE THE BIG ONE FROM BIRTH TO COMPLETE OUT RESULTS. WHAT DID WE LEARN? FOR THE FIRST INDICATOR, SPECIMEN COLLECTION BEFORE 48 HOURS OF LIFE. SO THIS GRAPH REPRESENTS HOW THE STATES DID AS A GROUP. SO IT'S ALL SEVEN STATES, MEDIAN EACH MONTH PERCENTAGE OF DRIED BLOOD SPOTS THAT WERE COLLECTED WITHIN 48 HOURS OF BIRTH AND WE HAD SOME -- WE STARTED AT 91.6%, AS A GROUP WE WERE ABLE TO MEETS 95% BENCH MARK. AS A CONGLOMERATE WE REACHED THAT GOAL. THIS ACTUALLY SHOWS THE INDIVIDUAL STATES. SO HIDING IN THERE, 95% IS A BAR, A PURPLE BAR, THAT'S GOAL WHERE WE WERE GOING. SO AS YOU CAN SEE THERE WERE FOUR STATES THAT WERE ASKED WHERE WE WANTED THAT 95% AND OTHER STATES HAVE SHOWN PROGRESS. WHAT'S IMPORTANT, WE STARTED THIS AND WE DIDN'T TELL THEM THEY HAD TO ACTUALLY -- THIS WASN'T ONE WE MANDATED SO FIVE STATES WERE WORKING ON THIS. SO THESE ARE THE FIVE STATES TRYING TO IMPROVE COLLECTION WITHIN 48 HOURS OF BIRTH. THEY ALL SHOW PROGRESS SO IN A SHORT PERIOD OF TIME PEOPLE MADE STRIDES AND PEOPLE STARTED LOW. SO SOME PEOPLE WHERE UNDER 80% AND WERE ABLE TO GET CLOSE OR JUMP TO 95%. HOW DID THEY MAKE THESE CHANGES? LEARNING COLLABORATIVE, WE TALK ABOUT BARRIERS WE TALK ABOUT HOW TO OVERCOME THOSE BARRIERS. SO ONE OF THE FIRST BARRIERS IS HOSPITALS DON'T KNOW THE RECOMMENDATIONS, RIGHT? AND AGAIN GIVEN TIMING THAT MAKES SENSE. WHEN THIS OCCURRED BUT WE HAVE TO ACTUALLY LET HOSPITALS KNOW HERE IS THE BAR WE'RE LOOKING FOR. AND WHEN THEY DID KNOW THE BAR THEY DIDN'T KNOW HOW WELL THEY WERE DOING. SO ONE OF THE THINGS THAT CAME OUT WAS TO PROVIDE HOSPITAL REPORTS SO THIS IS A SAMPLE REPORT, WE HOOD SEVERAL STATES BUT THIS IS A SAMPLE REPORT FROM ONE OF THE STATES, THIS -- THERE'S A LOT OF THINGS THAT I LIKE ABOUT THIS REPORT. WHY IS THIS VERY CLEAR TO SEE WHERE THE STATE AVERAGE IS, IT'S THE BLUE BAR AND EASILY FOR THIS HOSPITAL TO KNOW WHERE THEY ARE. SO THE YELLOW BAR OPT BOTTOM. I WANT TO MOVE TO THE SCREEN, THAT'S WHY I KEEP LOOK LIKE I'M MOVING THAT DIRECTION. YOU CAN WATCH THIS AND ACTUALLY THE HOSPITALS CAN SEE THEIR BAR MOVE. ONE THING TO KNOW IS THIS STATE SHOWS TO DEIDENTIFY OR KEEP DEIDENTIFIED. YOU CAN SEE NUMBERS BUT THEY DON'T KNOW WHO'S IN THE TOP, THEY DON'T KNOW WHO IS IN THE BOTTOM WHICH IS IMPORTANT. SOME STATES CHOSE TO MAKE IT IDENTIFIABLE, OTHERS DID NOT. THE OTHER THING YOU'LL NOTICE ABOUT THE REPORT, YOU WILL SEE RED, YELLOW AND GREEN. ONE OF OUR FIRST EARLY LEARNING SESSIONS WE BROUGHT IN A DATA VISUALIZATION EXPERT WHO TALKED TO US ON THE PHONE ABOUT LAY OUT, COLOR, HORIZONTAL, IS IT VERTICAL, SO THIS REPORT REFLECTS THINGS WE LEARNED DATA VISUALIZATION EXPERTS. THE OTHER THING I LIKE ABOUT THIS REPORT, THIS WORKED CLOSELY WITH THE HOSPITALS AND THEY KEEP BRINGING VERSIONS TO MAKE SURE IT WAS CLEAR. SO IT'S NOT JUST GET IT OUT THERE, BUT IN A WAY THAT MAKES SENSE AND AT A GLANCE PEOPLE CAN SEE WHERE THEY ARE, WHERE THEY NEED TO BE GOING. REPORT CARDS WERE AWESOME. THEY DON'T MAKE TO IT THE PEOPLE WHO NEED TO SEE THE REPORT. SO THAT WAS ANOTHER LESSON LEARNED. WHOEVER YOU' PEOPLE WERE SENDING IT TO DIFFERENT ROLES WITHIN THE HOSPITAL, SOMETIMES THEY GOT SHARED, SOMETIMES THEY DIDN'T. SO THERE WAS A LOT OF EDUCATION TO HOSPITALS ABOUT THE VALUE OF SHARING THE REPORT. AND A LOT OF DISCUSSION WHO YOU AIMED TO TO GET THE REPORT OUT TO. SO YOU GET INTO THE NURSING SUPERVISOR, SHE MAY OR MAY NOT SHARE IT WITH STAFF AND THAT WOULD BE GREAT BUT TURNS OUT YOU BRING RISK MANAGEMENT IN A HOSPITAL THEY MAY ADD MORE BUY IN. RIGHT? SO IT WAS HOSPITAL TO HOSPITAL BUT THE STATES SPENT TIME FIGURING OUT WHO SHOULD GET THE REPORT AND OFTEN ADDED TO THE LIST VERSUS SUBSTITUTEK PEOPLE ON THE LIST. SO MORE PEOPLE WERE GETTING THE REPORT. THREE STATES DID SURVEYS TO HOSPITALS TO FIND OUT WHO THEY KNEW, WHERE THEY WERE AT AND ONE THING THAT CAME OUT FOR ONE STATE, SLIGHTLY MORE THAN A THIRD RECALLED WATCHING THE VIDEO, ALL HOSPITALS GOT IT. IT'S IN THE HOSPITAL SOMEWHERE BUT PEOPLE ARE NOT CALLING IT OUT TO EDUCATE SO WHAT THIS STATE DECIDED TO DO WAS POINT OF CARE MESSAGES SO THEY CREATED POSTERS HUNG UP IN THE NURSERY, IN THE NICU SO PEOPLE THERE AND THEN COULD SEE AND BE REMINDED OF THE MESSAGE. SO THIS IS CREATED. THEY WORKED WITH LOCAL UNIVERSITY TO GET THIS DONE ON A VERY GOOD BUDGET. IT HIGHLIGHTS EVERYTHING ON THIS POSTER, I KNOW WHEN THE COLLECT, HOW LONG TO DRIVE, WHEN IT'S SUPPOSED TO BE SHIPPED SO IT'S ALL RIGHT THERE AND I CAN PLACE IT AND IT DOESN'T MATTER IF IT'S NIGHT SHIFT OR MORNING SHIFT, I HAVE IT THERE IN FRONT OF ME. THE SAME PROGRAM ALSO CREATED ANOTHER POSTER WHERE THEY WERE AT THE -- EMPHASIZING DEMOGRAPHIC INFORMATION. IF YOU CAN'T FIND OUT HOW TO CONTACT THEM ABOUT THE RESULTS YOU'RE STILL MISSING THAT END PIECE. SO THIS WAS ANOTHER POSTER THAT THEY HAD. AND FOR ONE OF THEIR OWN BENCHMARKS THEY WERE LOOKING AT ACCURACY AND INFORMATION. ANOTHER BARRIER IS STATE LEGISLATION. WE'RE SAYING HEY WE WANT IT NO LATER THAN 48 HOURS OF LIFE BUT THIS DAY WHEN WE STARTED THEIR STATE LEGISLATION SAID THAT THEY BLOOD SPECIMENS SHOULD BE COLLECTED BETWEEN THEND AND #th -- 6TH DAY OF AGE. SOME HOSPITALS SAY STATE SAYING 48, SOME ARE SAYING NO WAY, THE LEGISLATION SAYS DON'T START UNTIL 48, I WON'T GO FOR IT. THIS WON'T SHOCK YOU IN THE 15 MONTHS THEY DIDN'T GET IT CLANGED BUT IN 19 MONTHS THEY DID. SO ADS OF JULY, THEY HAVE NEW LEGISLATION AND RIGHT NOW READS SPECIMEN COLLECTION OCCURS AFTER 12 HOURS BUT NO LATER THAN 96 BUT THE GOOD NEWS IS, IT'S OPEN COMMENT PERIOD AND THEY'RE WORKING TO GET IT DOWN TO 48. SO IT'S -- THAT'S A HARD CHANGE TO MAKE. BUT ABLE TO DO IT IN 19 MONTHS. THAT'S OUR COLLECTION TIME. SO ANOTHER THING WE LOOKED AT IS SPECIMEN RECEIPT. AND IT'S REALLY INTERESTED, I WAS TALKING TO STAN YESTERDAY ABOUT TIMELINESS AND YOU FOCUS THINGS THAT AFFECT THE CONTINUUM BUT THERE ARE CHANGES YOU CAN MAKE THAT MAKE CHANGES IN DAYS INSTEAD OF JUST HOURS. AND THIS QUALITY INDICATOR IS A PLACE TO MAKE CHANGES IN DAYS. WE'RE LOOK AT SPECIMENS RECEIVED IN NEWBORN SCREENING LAB AND 48 HOURS OF COLLECTION. SO AS A GROUP, THIS IS ALL I'M SEVEN STATES AS A GROUP, THE MEDIAN WAS 68% WHEN WE STARTED. BOOSTED UP TO 80 WHICH IS A BIG JURY. WHAT YOU SEE, THERE WAS A LOT OF MOVEMENT HERE. SO THERE ARE ACTIVITIES THAT WE FOUND THAT REALLY HELP INCREASE THIS. SO ONE OF THE BIGGEST BARRIERS STILL IS EDUCATION, HOSPITALS DON'T KNOW WHAT THEY'RE AIMING FOR. THIS IS ANOTHER STATE TO PROVIDE REPORTS AND IF YOU LOOK AT THOSE PURPLE DOTTED LINES, THAT FOLLOWS THE REPORTS WERE RELEASED. SO THEY RELEASED THIS REPORT IN AN IDENTIFIABLE WAY. EVERYONE ELSEWHERE KNEW WHERE YOU WERE, THAT LEADS TO ACTION FOR A FEW MONTHS SO THEY GET A LOT OF CALLS PEOPLE WERE INTERESTED IN EDUCATION, THEN YOU SEE A PLATEAU, THEY RELEASED ANOTHER REPORT AND AGAIN THEY GOT SOME ACTION. SO THE TIMING OF REPORTS IS SOMETHING THAT WE SEE OUR 360 SITES LOOKING AT, HOW OFTEN DO YOU REPORT BECAUSE YOU DO GET MOVEMENT. ONE WAY TOO MAKE A BIG CHANGE IS CHANGE LABORATORY HOURS SO THE STATES THAT CAME TON THE COIIN IN THAT FIRST DAY WE WERE MEETING WE WERE TALKING ABOUT ROOT CAUSES OF TIMELINESS, THEY IDENTIFIED THEIR LAB ONLY BEING OPEN FIVE DAYS A WEEK AS A MAJOR PROBLEM, MAJOR BARRIER TO THEM SO TWO STATES WERE IMPACTED BY THE LAB IN THE COIIN AND IN MARCH, THEY -- THIS LABORATORY BEGAN TO BE OPEN SIX DAYS A WEEK INSTEAD OF FIVE DAYS. THIS IS IMPORTANT, THEY WERE OPEN ON THE 6TH DAY TO RECEIVE AND PROCESS WHICH IS DIFFERENT. SOME LABS JUST OPEN TO RECEIVE BUT NOT TO PROCESS. SO THIS ONE WAS OPEN TO RECEIVE AND TO PROCESS. ONE STATE WENT FROM 12% RECEIVED 48 HOURS TO 53% WITHIN 48 HOURS. AND I ACTUALLY NEED TO BACK UP. TO HERE. THE GOLD LINE IS ORANGE BECAUSE HERE IS A PLACE WE DEVIATED, YOU ARE RECOMMENDING 24, YES LOOK AT 48 SO OUR BAR IS A LITTLE DIFFERENT THAN WHAT THIS COMMITTEE RECOMMENDED. SO I DIDN'T GIVE A PURPLE BAR THERE. SO CHANGING THE LABORATORY HOUSE WAS GREAT WITH A BIG BUMP BUT ONE LESSON LEARNED IS OPENING AN EXTRA DAY IS NOT THE SILVER BULLET FROM 0 TO 95%. I THINK THAT'S IMPORTANT. THERE'S OTHER WAYS THAT YOU CAN GET THE EXTRA MOVEMENT BUT FROM A QUALITY IMPROVEMENT STANDPOINT ONE THING YOU SEE IS THIS CHANGE IS IMPORTANT WITH A BIG IMPACT BUT IT PLATEAUS. SO THEY WERE ABLE TO GET VERY HIGH, 70%, 53%, IOWA ALLOWED MANY E TO DEIDENTIFY THEM FOR THIS IOWA IS OPEN SEVEN DAY AS WEEK, 24 HOURS A DAY, ONLY STATE FOR AT THE 48 HOUR TO BE OVER THAT 95% BENCHMARK WE SET SO BY DOING THAT THEY WERE ABLE TO REACH THAT 95% BUT HERE IS AN IMPORTANT MESSAGE TO CONSIDER. IOWA SUSPECT AT 95% FOR 24 HOURS, SPECIMENS WITHIN 24 HOURS, THEY'RE AT 50%. WHAT I SAY IS MAYBE -- THE 48 HOUR IS POTENTIALLY BENCH MARK SHOULD BE CONSIDERED BECAUSE IOWA IS 100% FOR RESULTS REPORTED WITHIN 7 DAY IT IS OF LIFE, AT 100% FOR CRITICAL RESULTS REPORTED WITHIN FIVE DAYS. SO THEY'RE MEETING THE TRUE END GOAL BUT NOT MEETING THIS ONE BENCHMARK. SO SOMETHING TO THINK ABOUT AS WE THINK ABOUT THE DIFFERENT BENCHMARKS MOVING FORWARD FOR TIMELINESS. SO BEING THERE TO RECEIVE IT IS GREAT BUT IT HAS TO GET THERE. SO THE OTHER BIG CHANGE THAT WE SAW HERE WAS CAREER SERVICE SO SPECIMENS SPEND TOO MUCH TIME IN TRANSPORT. A HORRIBLE WAY TO GET SPECIMENS BUT ONE OUR SITES BEGAN A CAREER SYSTEM AND OTHERS EXPANDED THEIR CAREER SYSTEM. SO THIS IS FROM A SITE THAT BEGAN A CARRIER SYSTEM AND ROLLED OUT WITHIN THREE REGIONS OF THE STATE. SO THEY CUT THE STATE INTO THREE PIECES. THE TOTAL HOURS FROM BEFORE THE CHANGE AND HOW LONG IT TOOK SPECIMENS TO GET TO THE LAB VERSUS AFTER THE CHANGE. SO IN THE EASTERN PART OF THE STATE IT TOOK 84 HOURS TO GET TO THE LAB, DROPPED DOWN TO 44. NOT 24, 44. YOU GOT THE SAME WITH 64, 39, FROM 89 HOURS TO 49 HOURS. SO ADDING A COURIER STATEWIDE GOT THEM THAT BENCHMARK FOR COIIN. THIS HAS A STATE SYSTEM BUT NOT STATEWIDE. BUT ONE THING THEY DID IS ADDED MORE BIRTHING CENTERS. SO THEY ADDED 25% MORE FACILITIES TO CAREER PROGRAM AND YOU SEE IN THAT ADDITION THEY'RE ABLE TO GET UP AND GET A BUMP. SO THE COURIERS DEFINITELY HELP MEET TIMELINESS AS WE SAID IT IN TERMS OF COLLECTION TO RECEIVE BY LAB. ANOTHER FAVORITE LESSON LEARN FROM COIIN, WE HAD A STATE WHO HAD COURIER SERVICE IN THE CONTRACT THEY HAD SATURDAY COURIER PICK UP BUT TIME PASSED AND THEY REALIZED THAT THE COURIER WASN'T RUNNING ON SATURDAY. SO A VERY EASY THING TO CHANGE BUT WHEN THEY CAME TOGETHER FOR THAT FACE TO FACE MEETING, THEY'RE LIKE THAT'S INTERESTING WE HAVE SATURDAY IN OUR CONTRACT BUT IT'S NOT HAPPENING. WE THINK ABOUT IT IN JANUARY BUT IT DIDN'T GET CHANGED UNTIL JUNE BECAUSE WHILE SOUNDS EASY TO SAY IN THE CONTRACT DO IT, IT'S NOT. BECAUSE PART OF THE PROBLEM WITH THE HOSPITALS WERE SAYING, DON'T COME. THAT WAS BECAUSE THE HOSPITALS THOUGHT THEY WERE PAYING FOR THE COURIER THOUGH THEY WEREN'T. SO THERE WAS EDUCATION IN HOSPITAL, YOU'RE NOT THE ONE PAYING FOR THIS AND THEY NEED TO DOCUMENT, EVEN IF YOU DON'T HAVE ANYTHING. SO IT TOOK A WHILE TO GET THAT SYSTEM CHANGED. BUT ONCE IT GOT REINSTATED YOU SEE MORE SAMPLES GETTING TO THE LAB IN A TIMELY MANNER. ANOTHER THING WE RAN INTO, TALKING TO THE STATES IS THE WAY THE COURIER WORKS, THEY HAVE A ROUTE. ONE STATE WAS LOOKING AT WHICH HOSPITALS WEREN'T WORKING AND THEY WERE LOOKING AT THAT TIME 24 HOUR GOAL. AT WHO WASN'T MEETING THAT 24 HOUR GOAL. AND THEY FOUND IT WAS THE ONES THAT WAS EARLIER, CLOSER TO THE STATE HEALTH DEPARTMENT BUT EARLIER ON THE COURIER ROUTE HAVING A HARD TIME GETTING THE SPECIMENS READY FOR PICK UP. SO A LOT OF OUR STATES HAVE WORKED VERY CLOSELY WITH HOSPITALS, WE SAID THERE WAS A HOSPITAL REP AND THEY TALKED HOW TO TROUBLESHOOT THIS SPECIFIC STATE HASN'T FIGURED OUT THE 24 HOUR PIECE BUT IN TERMS OF 48 HOUR PIECE, PEOPLE TALKED ABOUT HAVING SOME HOSPITALS HAVE LABORATORY STAFF, GATHER THE SPECIMENS INSTEAD OF NURSING TO DO AT A SPECIFIC TIME, ONE HOSPITAL CHANGED WHERE THE PICK UP OCCURRED. SO FOR THIS HOSPITAL THIS IS A HOSPITAL, FOR THIS HOSPITAL THERE'S IS BUSY AND COULDN'T GET THE DRIED SPECIMENS DOWN TO THE LAB FOR THE COURIER. THEY HAD MEETING, LET'S HAVE THE COURIER GO TO THE BIRTHING UNIT. THEY WENT OVER 30% BEING LATE TO LESS THAN 10% BEING LATE. SO THAT LITTLE CHANGE HAD A BIG IMPACT FOR THAT HOSPITAL. SEW THAT COMMUNICATION, WORKING WITH THAT SYSTEM IS IMPORTANT AS WE TROUBLESHOOT AND THINK THROUGH TIMELINESS. JUST LIKE LABORATORY HOURS, COURIERS HIT A PLATEAU SO IT'S HELPFUL THAT YOU HIT A SPOT WHERE YOU NEED A LITTLE BIT MORE TO GET PAST. ONE THING TO THINK IS THE NUMBER OF DAYS THE COURIER PICKS UP. SO COURIER TO COURIER, IT WAS A HUGE JUMP, UNDER 40% CLOSE TO 80. THEY THOUGHT THE COURIER WOULD GET TO 95 BUT THE COURIER IS COMING SIX DAYS A WEEK SO ONE POTENTIAL THING TO THINK ABOUT IS COULD IT BE SEVEN. AND THIS IS OKLAHOMA IS PART OF THE 360 PROJECT THAT PRESENTED TO US ON THEIR COURIER SYSTEM. WE NOTICED SOMETHING WHEN PRESENTING THEY DO TWO GRAPHS ONE FOR THEIR HOSPITALS ON SEVEN DAY AND ONE FOR THEIR HOSPITAL ON FIVE. YOU CAN SEE THEY ACTUALLY -- ALLOW TO SHARE THIS, THEY IDENTIFY HOSPITALS. SO FOR THE SEVEN DAY THEY HAVE A 95% BENCHMARK IN 48 HOURS, NOT ALL ARE MAKING IT BUT THERE ARE SOME THAT ARE MAKING IT. FIVE -- IS MAKING IT SO ANYING ABOUT HOW TO GET THE SPECIMENS THERE, HOW OFTEN TO GET THEM THERE AND ARE PEOPLE THERE TO RECEIVE THEM AND RUN THEM. SO OUR NEXT QUALITY INDICATOR, NEXT PIECE OF QUALITY INDICATOR WE LOOKED AT IS RESULTS REPORTED OUT WITHIN THREE DAYS OF LAB RECEIPT. NOW SEEING DROP OFFS SO THE LAST TWO PIECES WERE HARD FOR OUR STATES TO GIVE US DATA BECAUSE OF WHAT WAS COLLECTED WHEN WE STARTED THIS PROJECT. WE AIMED FOR THREE DAYS. THAT WAS MADE UP BY US BUT WE DIDN'T -- WE TOOK THE TIME LINE WHEN WE WANTED THINGS REPORTED OUT, WE TOOK THE OTHER RECOMMENDATIONS RIGHT SO 48 HOURS TO COLLECT THE SPECIMEN, THEY FOR US HAD 48 HOURS TO GET IT IN, SO THEY REPORT OUT AND SEVEN DAYS THEY HAD NO MORE THAN THREE TO -- FROM WHEN THEY RECEIVED UNTIL WHEN THEY REPORTED OUT. SO AGAIN, OUR BENCHMARK IS ORANGE BECAUSE IT'S ONE THAT WE SET. BUT YOU CAN SEE, THERE WAS A LOT OF PROGRESS BUT WE HIT A PEAK THEN CAME DOWN. SO INTERESTING THINGS WERE HAPPENING SO AS A GROUP WE WENT FROM 25% TO ENDING UP AT 57. WHAT'S GOING ON? THESE ARE THE INDIVIDUAL STATES. SO YOU CAN SEE THAT THE GREEN STATE HAS SOME ONGOING THINGS HAPPENING SO ONE MONTH THEY HAVE IT, ONE MONTH THEY DON'T. THEN DEIDENTIFY THE PURPLE STATE BECAUSE THEY ALLOWED ME TO, THAT'S CALIFORNIA, ONE OF THEIR CHANGES IS THEY BROUGHT ON SCID, THEY HAD SCID. SORRY. THEY HAD SCID WHILE DOING COIN. BUT IN CALIFORNIA THEY HAD REGIONAL LABS AND THEN THEY HAVE A STATE LAB. WHEN A NEW CONDITION COMES ON, IF THERE'S NO FDA APPROVED TEST THEN IT HAS TO GO TO STATE LAB. SO THEY CAN RELEASE MOST OF THEIR RESULTS, IN A TIMELY FASHION BUT THE ONE RESULT TAKES LONGER WHEN THEY DON'T HAVE TO GO THROUGH THE STATE LAB. SO THE MINUTE THEY GOT FDA APPROVED THEY SHOT UP BUT THAT WAS THE PROBLEM FOR THEM. THEY'RE GOING TO ROLL OUT NEW TESTING SOON. SO THEY EXPECT TO SEE A REPEAT OF THIS. THE OTHER PHASE THAT HAS A LINE THAT LOOKS LIKE A STRUGGLE, THEY HAD PERSONNEL SO THEY HAD SHIFT IN PERSONNEL. BUT THEN WHEN THE SCID TESTING BEGAN IN JANUARY THEY TOOK A DIVE BECAUSE THEY WERE SHORT STAFFED, IT'S THE HOLIDAYS AND THEY HAVE A NEW TEST, THEY HAVE THINGS TRYING TO WORK OUT. SO WE SHOULD EXPECT THAT AS NEW TESTS ARE BEING ROLLED OUT, THERE'S GOING TO BE SOME HITS TO TIMELINESS IN TERMS OF REPORTING OUT. WE DON'T KNOW ENOUGH ABOUT THIS YET. BUT NEW STEPS I JUST RECEIVED AN WARED FOR NEW DISORDERS COOPERATIVE AGREEMENT FROM HRSA SO WE'LL BE EXPLORING THAT AS WE MOVE FORWARD WITH THAT WORK. THE OTHER THING ONE OF THE STATES ON HERE, HAD -- THEY WERE -- THEY HAVE A PEAK SO THEY GO UP. DURING THE HOLIDAY SEASON. THE THING WE HEARD IS HOLIDAYS ARE KILLER BUT WITH NEED TO FIGURE HOW TO DEAL WITH THE HOLIDAY KILLER THESE STATES HAVE QUALITY IMPROVEMENT, LET'S TRY SOMETHING DIFFERENT. WE KNOW THIS IS COMING, LET'S TRY LOOKING AT WHAT'S GOING ON AND STAFFING DIFFERENTLY. THEY GET SPECIMEN THROUGHOUT THE DAY BUT TWO PRIMARY TIMES THAT SPECIMENS COME IN. WHEN A SPECIMEN COMES IN, IT'S NOT NECESSARILY READY TO RUN, TOUGH DO WORK ON IT, YOU HAVE TO GET IT READY SO WHAT WAS HAPPEN ING IS LATER AFTERNOON WOULD COME IN AND BE READY WHEN PEOPLE FROM THE LABORATORY WERE GOING ONLY. SO SPECIMENS ARE SITTING THERE, NOT BEING TESTED. SO THEY SHIFTED THE LABORATORY HOURS BACK SO THEY HAVE TIME TO RUN THAT SECOND SET BEFORE THEY WENT HOME AND THEY SAW SUCCESS. AND THEY ARE AGAIN QUALITY IMPROVEMENT STATE THEY GET IT, THIS IS THEIR STUDY AND SO IN JULY THEY STARTED THEIR APP WHICH IS TO ACTUALLY NOW THEY DO THIS AS NEW WAY OF DOING BUSINESS AND WE'RE LOOKING FORWARD TO THE DATA TO SEE THE IMPACT LONG TERM. SO THE LAST PIECE OF QUALITY INDICATOR SLIDES THAT E LOOKED AT WAS RESULTS REPORTED OUT AT SEVEN DAYS OF BIRTH, WE DIDN'T DO FIVE DAYS BECAUSE WE ONLY HAD ONE STATE TO PROVIDE THAT DATA. SO WE HAVE TWO STATES THAT CAN PROVIDE THIS DATA. SO YOU HAVE ONE STATE THAT DID VERY WELL, THEY WERE ABOVE THE GOAL LINE. BUT THERE'S ROOM FOR IMPROVEMENT. THEY WENT FROM 9 # TO # HUNDRED% -- 98 TO 100% SO STILL DOING ACTIVITIES TO IMPROVE. THIS OTHER STATE HAD AMAZING GROWTH FROM 92% TO 100%. SO THIS IS STATE ALREADY DOING SOME ACTIVITY, BUT WHAT THEY DECIDED TO FOCUS ON FOR COIIN WAS POOR PERFORMING HOSPITALS. SO BEFORE THIS THEY HAD BEEN LOOKING AT POOR PERFORMER BUS LOOKING AT LARGE HOSPITALS THAT WERE POOR PERFORMERS FOR COIIN THEY LOOKED AT ALL HOSPITALS SO REGARDLESS OF SIZE. AND THEY DID SOME TARGETED EDUCATION EFFORTS. AND THEN THEY LOOKED AT COURIER SERVICE AND TRIED TO SEE IF THERE WAS A WAY THAT THEY CAN GET TO THESE POOR PERFORMERS TO FIGURE OUT WAY POOR PERFORMERS GET TO A COURIER. SO BETWEEN THOSE EFFORTS THEY WERE ABLE TO GET 20% PLUS BOOST. WE MADE A LOT OF PROGRESS IN 15 MONTHS. ALL STATES IMPROVED. IN TERMS OF THEIR OWN GOALS, THREE STATES MET AT LEAST ONE OF THEIR GOALS, BUT ALSO IMPROVEMENT SOME OF YOU HIGH ACHIEVERS AND WE WRITE GOALS. BUT IT WAS GREAT. THE STATES HAD -- CAN SHOW IMPROVEMENT SO NOW WHAT? NEW STEPS GOT SOME FUNDING, NEW STEPS 360, A HRSA FUNDED PROJECT THAT BEGAN A YEAR AGO TO THE DAY. AND WE'RE -- TO ME IT'S LIKE COIIN ON STEROIDS. SO WE TOOK THE COIIN MODEL AND BLUE IT UP. -- BLEW IT UP. SO INSTEAD OF WORKING WITH SEVEN STATES, WE'RE WORKING WITH 20. ON IS THE AIRPLANE RIDE HOME I'M REVIEW APPLICATIONS FOR ROUNDS 2. WE MEET NEXT WEEK. SO IN SEPTEMBER WE'LL HAVE MORE STATES THAT ARE JOINING AND DOING EFFORTS TO IMPROVE TIMELINESS IN THEIR STATES. THE GOALS ARE THE SAME. SO WE'RE AIMING FOR THAT 95% OF THE TIMELINESS GOALS. MOSTLY IT'S THE SAME THERE'S A FEW DIFFERENCES. NOW WITH GIVE THE STATES MONEY. WHICH IS A HUGE DIFFERENCE, TO HELP START SOME OTHER EFFORTS. THEIR EFFORTS HAVE TO BE SUSTAINABLE AND FUNDING ENDS SO WE'RE NOT PAYING FOR A SERVICE THAT WHEN FUNDING GOES AWAY THEY CAN'T CONTINUE TO PAY FOR. AND THEN FOR COIIN I CALL STATES PERIODICALLY AND TALK BUT NOW WE'RE TARGETED IN OUR SUPPORT IN OUR COACHING. SO ALL STATES HAVE A COACH, A CQI COACH WHO CALLS EVERY MONTH OR EVERY OTHER MONTH TO HELP ALONG. IN THAT CALL WE GET DATA. SO NOW WE HAVE RIGOROUS DATA AND WE'RE REALLY GOING TO UNDERSTAND PEAKS AN TROUGHS BECAUSE OF THE WAY WE'RE COLLECTING DATA THIS TIME. BUT IT'S SO SHORT OF RESOURCES, IT'S STILL LOOKING AT THE QUALITY INDICATOR DATA. SO WE HAD SOME -- WE HAD SOME SUCCESS BUT I HAVE A FEW STORIES WHICH PEOPLE ALLOWED ME TO SHARE WITH U YOU. I HAVE -- WITH YOU. I HAVE SHOWN YOU OKLAHOMA. THEY RECENTLY PRESENTED ONE OF OUR WEBINARS. AND BEFORE THEY WERE PART OF COIIN THEY DID -- THEY STARTED THIS EFFORT TESTIFY DAY THAT COUNTS AND THEY'RE EXPANDING AS PART OF THE COIIN. ONE CHANGE THEY MADE IS HOW OFTEN THE HOSPITAL REPORTS COME OUT. SO YOU REALLY NEED TO LOOK ALONG THE BOTTOM AND IT'S QUARTER 1, 2016. THAT IS WHERE YOU SEE THE CHANGE. THEY MADE PROGRESS ON THEIR COUNTS. THEY HIT A PLATEAU, BY GOING THE QUARTERLY REPORTS MONTHLY THEY NOW HAVE A HIGHER PERCENTAGE OF HOSPITALS HAVING THEIR SPECIMENS ARRIVE WITHIN TWO DAYS OF COLLECTION. SO THAT'S WHAT THEY'RE MEASURING THERE. VIRGINIA MADE A LOT OF CHANGES. AND SO THEY HAVE MORE HOSPITALS TO THEIR ROUTE, THEY ADDED A SUNDAY COURIER SO THAT YOU ARE' SIX DAYS FROM FIVE TO SIX DAYS. THEY ALSO GAVE REPORT CARDS BUT THEIRS WERE QUARTERLY, DOING EDUCATION EFFORTS WITH SOME OF THE POOR PERFORMING SITES. THEY PUT INFORMATION IN THE REPORT CARDS, ABOUT CHANGES TO HIGHLIGHT SUCCESS STORIES TO LET PEOPLE KNOW WHAT CAN BE DONE AND THEN WORKING TO CAPTURE THE DATA WE NEED TO TRACK. SO THIS IS THREE MONTH PRIOR TO JOINING COIIN -- SORRY JOINING 360. COIIN ON STEROIDS. BUT THEY -- ONES IN RED ARE TAKING OVER THREE DAYS TO GET SPECIMENS SCHEDULED TO HOSPITALS, ONES IN GREEN MAKE OUR TWO DAY MARK AND ONES IN YELLOW ARE THE COMING IN THE RIGHT DIRECTION. THIS IS THREE MONTHS AFTER JOINING COIIN. IN THAT TIME, SIX MONTHS, THEY HAD CHANGES FROM SIX BIRTHING HOSPITALS, THREE WERE ABLE TO COME DOWN AND MORE IMPORTANTLY THREE WERE WITHIN TWO DAYS. SO THEIR EFFORTS THEY'RE ALREADY SEEING A CHANGE QUICKLY. SO WISCONSIN IS ANOTHER PROGRAM THAT WAS FUNDED BY 360, THEY'RE FOCUSED ON GETTING RESULTS TO PROVIDER FASTERS. SO INSTEAD OF MAIL THEY ARE MOVING TO FAXING AND THE GOAL IS BY DECEMBER, TO HAVE 80% PROVIDERS RECEIVING FAXES. WHAT HAPPENINGS, THEY HAVE 95% OF THE RESULTS VERIFIED BY THE SEVEN DAYS OF LIFE BUT WHEN YOU MADE THOSE RESULTS YOU'RE ADDING ANOTHER THREE DAYS FOR THAT REPORT TO GET IN THE HANDS OF SOMEONE WHO CAN DO SOMETHING ABOUT IT. BY FAXING YOU'RE ADDING A FEW HOURS. TO GET RESULTS INTO THE HANDS OF SOMEONE WHO CAN DO SOMETHING ABOUT IT. FAXING IS NOT EASY, IT TAKE TIME ABOUT THINGS HAVE TO HAPPEN BUT HERE IS A SUCCESS THEY'RE SEEING AS A RESULT OF THE CHANGING THEY'RE MAKING SO LESS THAN 10% BEING IN THE HANDS WITHIN SEVEN DAYS AND NOW THEY'RE OVER 50%. SO IN A FEW MONTHS THEY HAVE MADE A BIG CHANGE BY FAXING TO THE ONES THEY CAN REACH AND AS THEY ADD MORE PROVIDERS THAT NUMBER WILL GROW. FINALLY, THE -- WE HAVE VIRAL PARTNERS ONE FEDERAL PARTNER IS BABY'S FIRST TEST. ONE THING THEY DID FOR US FOR 360 IS THEY CONDUCTED A FOCUS GROUP DURING THE A 1 MEETING. SONATA SHAH LED THE FOCUS GROUP, THERE WAS 14 PEOPLE, EIGHT STATES REPRESENTED SO HERE ARE SOME OF THE FINDINGS, IT REALLY REINFORCE WHAT IS WE HEARD. FROM COIIN STATES AND ALSO PROVIDES SOME NEW INSIGHT. GETTING A BLOOD SPECIMEN ISN'T AS EASY, WE HEARD YESTERDAY FROM JACKIE BUT MIDWAYING STRUGGLE WITH THAT EQUIPMENT, HOW TO DO IT WELL. WE HAVE THE DIFFERENT SHIFTS HAVING DIFFERENT INFORMATION WHICH IS WE HAVE GOT THE ONE STATE DOING THE POINT OF CARE EDUCATION SO THAT MIGHT BE A SOLUTION FOR THAT ONE. HOW TO FIT NEWBORN SCREENING INTO THE WORK FLOW. YOU NEED TO WORK WITH THE SYSTEM AND OUR STATES HAVE A LOT OF SUCCESS BY ASKING THEM HOW TO MAKE IT FIT IN. GETTING THAT BUY IN, SOME OF THAT IS SENT IN A REPORT TO YOU, BUT EDUCATION IN HOSPITALS WHY IT MATTERS. LOTS OF THINGS ARE HAPPENING, WE HAVE LOTS OF COMPETING PRIORITIES SO TRYING TO GET BACK TO THAT AND FINDING THE CHAMPION THEN TO SHARE THE PERSONAL STORIES, WHEN WE DO REVIEWS WE SHARE THE REPORTS WHICH ARE GREAT BUT EVEN AS WE KNOW HERE, THOSE PERSONAL STORIES ARE TOUCHING AND SOMETIMES YOU HAVE TO REMIND PEOPLE WHO THEY SAVE AS A RESULT AND MAYBE WHEN THINGS DIDN'T GO WELL SO THEY UNDERSTAND THE IMPORTANCE BECAUSE THAT GETS LOST IN THE DAY TO DAY ROUTINE. SO THEY'RE GOING TO SUBMIT AN ABSTRACT TO THEY WANT TO SHARE FINDINGS, QUALITATIVE SENSE, CHECKING AND SHARING WITH OTHER MEMBERS AND HAVE A PUBLICATION. SO WE ARE DOING GREAT THINGS. WE ARE NOT THERE YET. I LOOK FORWARD TO GIVING AN UPDATE AGAIN ON 360 ACTIVITIES. AS FOR EVERYTHING EVERYONE HAS SAID, IT TAKE AS VILLAGE TO DO THIS. THIS IS OUR NEW STEPS TEAM WHO HELPED WITH COIN AND HELPING WITH 360. THESE WERE AMAZING STATES THAT WERE PART OF COIIN, I CANNOT THANK THEM ENOUGH FOR LEARNING WAS, THEY GOT TO START FROM THE GROUND UP. THANK YOU. [APPLAUSE] TIME FOR QUESTIONS. >> YES YOU HAVE TIME. YVONNE, THANK YOU VERY MUCH, EXCELLENT PRESENTATION THAT SHOWS REALLY A REMARKABLE WONDERFUL APPROACH AND EXCELLENT RESULTS IN A RELATIVELY SHORT PERIOD OF TIME. VERY GOOD. LOOK FORWARD TO ANOTHER REPORT, IN ANOTHER 18 MONTHS. LET'S START WITH DISCUSSION. STEVE. >> I WANT TO THANK YOU FOR AN ABSOLUTELY OUTSTANDING PRESENTATION AND I WANT TO THANK YOU SO MUCH FOR THE IMPORTANT WORK THAT YOU ARE DOING, YOU'RE BENEFITING MANY CHILDREN. I HAVE ONLY BE INVOLVED WITH THIS COMMITTEE FIVE YEARS, MANY HAVE BEEN HERE LONGER BUT FIVE YEARS AGO WHEN I FIRST CAME HERE THE FIRST ADVOCATES THAT I MET WERE THE PARENTS OF CHILDREN WHO DIED BECAUSE TESTING HAD NOT BEEN PERFORMED ADEQUATELY. AND TO SEE FIVE YEARS LATER THE PROCESS OF MILWAUKEE SENTINEL NEWSPAPER DOING BRILLIANT REPORTING THEY DID, CONGRESS SEEMS LIKE YOU DO NOTHING GOT INVOLVED AND ASSISTED OUR ECONOMY IN PUBLIC HEALTH LAB PEOPLE WHO ARE WORKING ON THIS ISSUE, OUR COMMITTEE MADE SOME RECOMMENDATIONS YEAR AND A HALF AGO ALSO HAVE TO COMPLIMENT IOWA WHO DOES OUR NORTH DAKOTA TESTING AND STAN BERBIGDE AND THE LEADERSHIP HE PROVIDED FOR THE BENCHMARK FOR THE STATES TO GET UP TO AND JUST TO SEE THE RAPID PROGRESS THAT'S BEING MADE, IN RESOLVING THIS ISSUE, IT'S JUST SO IMPRESSIVE. GOING BACK WHERE WE WERE FIVE YEARS AGO AND PARENTS COMING TO OUR COMMITTEE WHERE WE'RE AT RIGHT NOW I WANT TO THANK YOU SO MUCH FOR THE WORK WE OTHER DOING, YOU DO AN EXCELLENT JOB PRESENTING THAT INFORMATION AS WELL. I HAVE ONE QUESTION YEAR AND A HALF AGO FEBRUARY 2015 WE VOTED ON THIS ISSUE, SET AN OBJECTIVE OF STATES ENCOURAGED STATES THAT HAVE 95% TIME RESULTS CRITICAL FIVE DAYS AND ALL REPORTS IN SEVEN DAYS, THERE WAS SUPPOSED TO BE A DATABASE SET UP BUT STATES WERE ENCOURAGED TO REPORT RESULTS. AND QUESTION I HAVE IS I GUESS FOR THE SERVICES BRANCH OR MCH, WHAT PROGRESS IS BEING MADE AND WHO ARE THEY GOING TO BE REPORTING THAT INFORMATION? TO? THANKS SO MUCH FOR WHAT YOU HAVE DONE. >> I THINK A DATABASE MIGHT BE A NEW STEPS NOT ALL STATES HAVE MOU BUT MANY STATES. I DON'T REMEMBER THE NUMBER. MARCY? 30. SO 31 STATES HAVE MOUs ENTERING DATA AND WE JUST DID A REPORT FOR THE GAO AND STATES -- SOME WERE UNABLE TO SUBMIT DATA TO US VIA EXCEL FILE SO WE HAVE SOME OF THAT DATA. >> THANK YOU. Q. THAT WAS EXCELLENT. QUICK QUESTION. THE TERM COURIER I FIND IS USED LOOSELY. NOT BY YOU. IT IS MY UNDERSTANDING IT IS A TRANSPORT SYSTEM SO EVEN THE MAIL IS TECHNICALLY A COURIER. -- MY UNDERSTANDING ALSO FROM DR. BURBRIGDE WHO EDUCATED ME ON THIS TOPIC, OUTSIDE THE MAIL YOU THEN HAVE SCHEDULED COURIERS WHICH THEY ARE RUNNING ROUTES AND YOU ARE BASICALLY CONTRACTING THEM AND EITHER THEY HAVE YOUR ROUTE ON THE ROUTE OR THEY DON'T AND WILL TELL YOU WHAT TIME THEY CAN PICK UP, TEN TO THE UPS FEDEX OR CONTRACT WITH THE COURIER, YOU CAN DESIGN WITH A COMPUTER LIKE I WOULD LIKE YOU TO BE HERE, ET CETERA, ET CETERA. DO YOU HAVE A SENSE OF THE DISTRIBUTION OF THOSE TYPES OF COURIERS AMONG THE STATES IN >> I CAN'T TELL YOU FOR ALL THE STATES, I CAN TELL YOU FOR THE COIIN STATES THEY WERE MOSTLY USING STATE RUN COURIER SYSTEMS. SO NOT -- SOME WERE USING -- THERE WAS ONLY A FEW. SO -- AND I DON'T HAVE AN EXACT DISTRIBUTION BUT THE ONES THAT BROUGHT IT ON BROUGHT ON STATES RUN COURIER SYSTEMS. >> SO STATES DECIDE WHEN THE PICK UP COMES AND WHEN THE DROP OFF HAPPENS. >> >> THEY HAVE CONTRACTS WITH THOSE COURIERS. I DON'T KNOW HOW THEY'RE NEGOTIATED. BULSES -- SO WE HAD A DISCUSSION RECENTLY FOR 360, TALKING ABOUT THAT CHANGE IN LAB HOURS AND SOMEONE POINTED OUT IF YOU HAVE A CONTRACT WITH A COURIER, EITHER CHANGE THAT CONTRACT -- IT'S EASIER TO CHANGE THE CONTRACT THAN CHANGE THE WORK FORCE AND DEAL WITH THE UNION. SO BUT I CAN'T -- THAT'S ALL I CAN TELL YOU. I DON'T KNOW -- AGAIN I'M LOOKING AT MY NEW STEPS VILLAGE, DOES ANYONE OUT THERE HAVE A -- I KNOW WHEN WE COLLECT THE DATA ON COURIER IT IS UP TO THEM TO DEFINE COURIER AND THAT IS A DISCUSSION WE HAD TWO DAYS AGO ABOUT TRYING TO DEFINE THAT BETTER. >> OTHER QUESTIONS, COMMENTS? >> THANK YOU FOR A GREAT PRESENTATION. WONDERFUL WORK. YESTERDAY IF I UNDERSTOOD CORRECTLY WE LEARNED AT LEAST FROM SOME OF THE STATES THEY'RE ADDING NEW TESTS GENETIC AND GENOMIC TESTS, MAYBE HARDER TO MEET THE DEADLINES IN FIVE TO SEVEN DAYS. IS THERE A MECHANISM FIGURING THAT OUT OR CHANGING THE DEADLINES OR DATA VISUALIZATION, HOW DOES THAT CHANGE? >> WE HAVEN'T HAD TO DEAL WITH THAT YET. BUT UNDER THE NEW DISORDERS GRANT, WE WILL. AND WE'RE NOT LOOKING AT TIMELINESS READINESS PER SE SO WE'RE TRYING TO TRACK THE TIME THESE STEPS TAKE, RIGHT NOW WHEN PEOPLE FILL OUT ABOUT IMPACT HOW LONG IT TAKES, IT'S A GUESS. SO WE'RE GOING TO COLLECT REAL TIME DATA TO GET A SENSE WHEN IT STARTS AND HOW LONG IT TAKES. THERE'S VARIATION DEPENDING ON THE TYPE OF TEST. SO MY ANSWER IS I DON'T KNOW YET BUT ASK ME AGAIN. >> I CAN COMMENT BECAUSE OF RIGHT NOW FOR -- INTERESTING, WHAT WE'RE DOING IS WHEN WE HAVE TEST RESULTS AVAILABLE EXCEPT CFTR MUTATION, WE SENT PRELIMINARY REPORT AND TELL THEM CS MUTATION TEST PENNING. WHEN YOU HAVE CS MUTATION AVAILABLE WE SEND ANOTHER REPORT SO WE TRY -- ANOTHER THING IT HAS AN EFFECT OVERALL 95% OF THEM BECAUSE ALL YOU HAVE TOP 4% ON THE GO. SO BRINGING FORWARD THAT'S THE ISSUE WE NEED TO THINK ABOUT LIKE THE STUDY YESTERDAY. >> SO AGAIN, THANK YOU FOR A GREAT REPORT. THAT SEEMS LIKE A GREAT SOLUTION, ANYBODY PAYING ATTENTION KNOWS YOU HAVE JUST TOLD THEM IRT WAS ABNORMAL BECAUSE IF THE CFTR IS PENDING AND YOU'RE ONLY DOING ON THE TOP 4%, YOU JUST TOLD THEM CIRT WAS NORMAL AND YOU KNOW WHAT YOU'RE DOING. >> EXCEPT I DON'T THINK MOST PHYSICIANS KNOW ANY OF THE TESTS. I AGREE WITH YOU. >> RIGHT. >> WILL KNOW AND PATHOLOGY WILL KNOW AND THE QUESTION IS, YOU KNOW, YOU CAN SEE A PARENT ASKING QUESTIONS. >> WE DID -- WE'RE WORKING ON THIS IN MICHIGAN BECAUSE AT MICHIGAN THEY WERE GIVING OUT GIVING OUT THE REPORT POSITIVE BUT NO MUTATION DATA. THE PHYSICIANS DIDN'T KNOW TO ASK FOR THE MUTATION DATA. IN ADDITION, WHEN WE SURVEYED THE STATE, THE PHYSICIANS IN THE STATE PRIMARY CARE PHYSICIANS, MANY GOT IT WRONG. UPWARDS OF 40% WHEN WE ASKED THEM IF THE SCREEN HAD TWO MUTATIONS HOW LIKELY THE CHILD WAS -- SO I THINK THAT THE -- I AGREE WITH YOU. I'M SUSPICIOUS THE PRIMARY CARE PHYSICIANS EVEN IF IT'S THERE THE COMPLICATION OF THE IMPLICATIONS UNLESS FLAT OUT TOLD THEY PICK UP ON THAT. >> I AGREE. WHAT'S INTERESTED IN IS FARTHER DOWN THE LINE DEPENDING ON STATE'S CRITERIA. IF YOU DECIDE TO CALL IT NEGATIVE, MUTATION NEGATIVE AND SOMEBODY STARTS QUESTIONING WELL, THE CIRT WAS POSITIVE IN ANOTHER STATE WOULD HAVE DONE A SWEAT TEST. SO IT'S JUST -- I MEAN, TO SAY IT'S PENDING, I HAVE HAD EXPERIENCE WITH ANOTHER STATE THAT CHANGED THE NEWBORN SCREENING FORM AND DIDN'T REAL IZE THEY WERE CONVEYING INFORMATION THEY HADN'T INTENDED TO CONVEY SO IT DOES CONVEY ADDITIONAL INFORMATION TO ANYONE WHO KNOWS WHAT TO LOOK FOR WHICH COULD BE A LAWYER LATER. >> GETTING BACK TO YOUR POINT BY SAYING PENDING IT'S STILL NOT ALL RESULTS SO NOT WITHIN THE SEVEN DAYS SO THAT'S SOMETHING WE NEED TO WORK ON. >> (INDISCERNIBLE) CF SCREENING ALGORITHM IS A TWO STEP, EVEN YOU HAVE A TOP 4%, I WOULDN'T CON VINCE THIS WAS -- IF SCREENING POSITIVE, THAT'S NOT OUR STATE EDUCATED PEOPLE BECAUSE LARGELY PEOPLE IN THIS CENTER IS NORMAL. THE REASON IS BECAUSE YOU HAVE A SECOND STEP YOU ALLOW YOURSELF A LITTLE BIT -- SO WE WOULDN'T THINKING BECAUSE POTENTIALLY -- >> NATASHA. >> QUICK, THANK YOU. YVONNE, THANK YOU, WHAT A GREAT PRESENTATION PARTICIPATING IN THE EXPANDED -- AND FORGIVE ME FOR TEXTING DURING YOUR PRESENTATION BECAUSE I WAS MESSAGING AMY BECAUSE I CLEARLY (INAUDIBLE) STATE OF MINNESOTA. ARE WE AT SIX DAYS OR SEVEN DAYS? I LEGITIMATELY DIDN'T KNOW AND I WANTED TO HEAR WHAT OUR HURDLES WERE. SO IT SEEMED FROM YOUR PRESENTATION THAT THIS 24 HOUR BENCHMARK, CORRECT ME IF I'M WRONG, IT SEEMED ALMOST UNACHIEVABLE IN SOME WAYS. I DON'T SAY THAT OFTEN BECAUSE WE'RE IN A WORLD TODAY WHERE I CAN CLICK ON AMAZON NOW AND GET 40 PACKS OF TOILET PAPER DELIVERED TO MY HOUSE ON CHRISTMAS DAY WITHIN AN HOUR. SO I THINK THE -- TO YOUR QUESTION WHAT'S THE DEFINITION OF COURIER, WE HAVE SO MANY INNOVATIVE NEW OPTIONS AVAILABLE TO ALL OF US INCLUDING THE PUBLIC SECTOR, THAT MIGHT REQUIRE EXPLORATION, IT COULD BE LIKE ON OUR NEW CCATA CALL WHERE WE PROVIDE NEW RECOMMENDATIONS HOW YOU CAN DO THIS BETTER. IT'S THAT WHOLE IDEA OF WHAT TRANSFER LOOKS LIKE. AND HOW I KNOW MECHANISMS AVAILABLE TODAY COST TO PUBLIC HEALTH SYSTEM BE AVAILABLE TO HOSPITALS. THAT WAS ONE THING. BUT I WONDERED ABOUT THE STANDARD AND WHAT THEY OR MAY NOT CHANGE AS A RESULT OF THIS NEWLY FUNDED WORK. >> THE RECOMMENDATION CHANGE WE HAVE A NEW BENCHMARK FOR 360, WE WILL USE THE RECOMMENDATION VERSUS THE OTHER. SO I DON'T -- I DON'T KNOW, ME NOT SPEAKING FOR ANYONE OTHER THAN ME, I REPRESENT NO AGENCY, I DO WONDER IF IT'S AABLE BUT ALSO I WONDER DOES IT MATTER. IF -- IF IT'S ACHIEVABLE. THAT'S THE ONE THAT REALLY MATTERS TO ME AND IF THAT'S WHAT'S BEING MET, THAT'S THE REST THAT GOES INTO IT, SO WE USE THIS AS WAY TO KIND OF SAY HOW -- WHERE IS THERE ROOM IN SYSTEM TO IMPROVE BUT 48 HOURS IS THE ROOM IN THE SYSTEM TO IMPROVE THAT'S OKAY, THEN AS LONG AS WE MEET 5 TO 7 THAT'S THE IMPORTANT BENCHMARK TO ME. >> I WANT TO QUICKLY -- THE 5 TO 7 IS THE METRIC WE'RE MEETING AND I'M GOING TO TALK ABOUT THIS TOO BUT THAT METRIC WAS DEFINED BY THIS COMMITTEE. THAT -- I WANT TO POINT OUT, THAT'S A METRIC WE DEFINED AND IF WE END UP WITH A CHILD THAT COULD HAVE BEEN DETECTED ON A DAY 4 AND WE CREATED A SYSTEM, ALL WE HAVE TO DO IS GET TO FIVE AS FAST AS WE CAN WITHIN REASON AND COST, WE ARE MAKING OURSELVES PLAYING AN ARBITRARY METRIC. I'M SAYING BEING SATISFIED WITH FIVE, IF FOUR IS ACHIEVABLE OR THREE, IT'S NOT NECESSARILY ACCEPTABLE. >> CLEARLY WITH THE TIMELINESS WORK GROUP, THEY TURNED AROUND THE QUESTION AND SAID WHAT DO WE WANT TO ACHIEVE. THE ACHIEVEMENT WAS SEVEN DAYS RESULTS WITH FIVE DAYS FOR TIME CRITICAL ILLNESSES OR SEVEN DAYS TIME CRITICAL. SO THEN THEY WORKED BACKWARDS AS TO WHAT WOULD BE NEEDED TOE MAKE THAT HAPPEN. SO WE'RE STILL WITHIN THAT TIME FRAME IS ONE THING BUT I DON'T THINK WE'RE AT THIS POINT READY TO CHANGE ANY GUIDANCE. NATASHA. >> JUST TO ADD TO WHAT -- I THINK THAT'S REALLY IMPORTANT BECAUSE AS YOU KNOW, SO MUCH OF WHAT STARTED THIS REALLY IMPORTANT WORK THOSE ARTICLES THAT CAME OUT, SOME OF THOSE STORIES IN THOSE ARTICLES UNDER ALL THIS GREAT WORK WE HAVE DONE SKA IT WOULDN'T CHANGE THE OUTCOME FOR THE CHILDREN. THIS WAS BY A PUBLIC IF YOU WILL MEDIA PUSH. AND THAT'S SOMETHING TO KEEP IN MIND BECAUSE WHERE ARE WE NOW VERSUS WHERE WE WERE THEN. I WANT TO ADD TO THE FOCUS GROUPS THAT WE DID WITH THOSE NURSES, WE REALLY TARGETED NURSE S WHO WERE LEADs OR FELT RESPONSIBLE. WHAT WE FOUND FROM THAT, IT WASN'T NECESSARILY SOMEONE WITH AN OFFICIAL TITLE BUT IT WAS SOMEONE WHO -- OH, I'M THE PERSON THAT BRINGS THE EDUCATIONAL MATERIALS BACK TO MY UNIT OR I'M THE -- THINGS THAT WE WOULD NEVER HAVE KNOWN WITHOUT ACTUALLY HAVING THOSE CONVERSATIONS. SO I REALLY COMMEND THE STRUCTURE AND REALLY CAN'T WAIT TO SEE HOW THIS STRUCTURE REALLY BEING COLLABORATIVE AND SPEAKING TO THE PEOPLE WHO WERE ALL IN THE FRONT LINE IN DIFFERENT WAYS. BUT THEY ARE REALLY ON THE FRONT LINE IN A SPECIFIC WAY. AND EVEN JUST GETTING INFORMATION BACK, NO ONE EVEN ASKED US THE USER INS ABOUT NEWBORN SCREENING. NO ONE ASKED US ABOUT OUR EXPERIENCE AROUND IT AND HOW IMPORTANT IT WAS AND HOW EMPOWERED TO HAVE A FOCUS TO SAY I'M GOING BACK TO THINK ABOUT THIS. PEOPLE TALK TO US ABOUT ALL SORTS OF ISSUES, NO ONE REALLY TALKS NEWBORN SCREENING EVEN DOWN TO NO ONE TALKED TO US ABOUT CHANGING THE FILTER PAPER AND THE INFORMATION ON IT AND THAT'S COMPLETELY KIND OF MESSED UP OUR FLOW AND ALL THOSE THINGS SO I THIS I THIS IS A REALLY IMPORTANT WORK AND THERE'S EVEN MORE IMPORTANT WORK THAT CAN BE DONE. >> COMMENTS. >> ANN COMO, MASSACHUSETTS. IBILITY THANK YOU FOR A VERY MICE PRESENTATION WHICH I THINK GAVE COMPLEXITY OF THE SITUATION AND VERY NICELY SHOWED A VARIETY OF COOPERATIVE SOLUTIONS. AND QUITE A VARIETY THAT SAID WHEN I COMES TO EVALUATION, I'M GOING TO REALLY ADVOCATE FOR MUCH LESS VARIETY AND VERY CAREFUL DEFINITIONS OF WHAT IT IS WE'RE LOOKING FOR. WHEN WE ARE LOOKING FOR WHAT WHAT IS OUR BENCHMARK FOR REPORTING A NEWBORN SCREEN. WHAT IS A SCREENING RESULT? IS IT A SCREENING RESULT ALL ENCOMPASSING OR IS IT A SCREENING RESULT PLUS SUPPLEMENTAL INFORMATION? VERY DIFFERENT TIME LINES, YOU'RE GOING TO GET FROM PEOPLE. SHOULDN'T WE BE ALSO LOOKING FOR TIME CRITICAL RESULTS? SO DESPITE THE KINDS OF VARIETY THAT YOU DISPLAY, I THINK I'M HOPEFUL, AND I THINK THAT MOST NEWBORN SCREENING PROGRAMS WOULD BE ABLE TO SAY WHEN THEY HAVE A OUT OF RANGE METABOLIC RESULT OR OUT OF RANGE ANY KIND OF RESULT THAT GETS OUT THE DOOR PROBABLY THE SAME DAY IT COMES IN. THAT WOULD BE A GOOD MEASURE BUT WE HAVE TO CAREFULLY DEFINE WHAT IT IS THAT WE ARE GOING TO REQUIRE NEWBORN SCREENING PROGRAMS TO AIM FOR. I WILL ALSO ADVOCATE THE GUIDELINES THE FIVE AND SEVEN DAYS WERE GOOD PLACES TO START BUT IN ORDER TO MAKE A DIFFERENCE TO THE SICK KIDS WE NEED TO FIND, WE NEED TO BE ABLE TO STANDARDIZE THE REPORTS SO ALL OF US CAN UNDERSTAND WHERE WE CAN HAVE IMPROVEMENTS. IF WE DON'T STANDARDIZE IT, WE WON'T KNOW WHERE TO GO. THANK YOU. >> THANK YOU. >> THANK YOU VERY MUCH FOR YOUR PRESENTATION, THANKS FOR THE DISCUSSION. NEXT ON THE AGENDA IS ROBERT WOOD JOHNSON PROJECT ON NEWBORN SCREENING TIMELINESS AND THE COMMITTEE MEMBER WILL BE JOINED BY AMY COCHRAN, RESEARCH ASSISTANT PROFESSOR UNIVERSITY OF MICHIGAN. DR. COCHRAN IS THE TH RESEARCH PROFESSOR IN THE MATHEMATICS DEPARTMENT UNIVERSITY OF MICHIGAN, HE RESEARCH INTERESTS ARE MATHEMATICAL BIOLOGY, ESPECIALLY COMPUTATIONAL PSYCHIATRY. SHE FOCUSED ON PSYCHIATRIC DISORDERS BIPOLAR DISORDERS AN ON DESCRIBING MATHEMATICALLY THE VOLATILITY OF THOSE CHARACTERISTICS OF THIS DISORDER. WELCOME. >> THANK YOU. I'M GOING THE START THE PRESENTATION AND THEN DR. COCHRAN WILL TAKE IT HOME. I WANT TO THANK YOU ALL FOR HAVING ME PRESENT TODAY ON OUR PRELIMINARY FINDINGS ON THE PROJECT IN GENERAL. I WANT TO THANK MY TEAM WHICH IS LARGER THAN THE TWO OF US AND THANKS TO THE MICHIGAN DEPARTMENT OF HEALTH WHO HELPED US WITH THIS PROJECT. AND WHO IS ON THE LINE, MY TEAM MEMBER, MARY KLINE AND LOIS TURBINE, SO THEY MAYBE ABLE TO ANSWER ADDITIONAL QUESTIONS IF I'M UNABLE TO AND/OR ADD PERSPECTIVE. SO THIS IS A PROJECT FUNDED BY ROBERT JOHNSON FOUNDATION THROUGH THE PUBLIC HEALTH SERVICES AND SYSTEMS RESEARCH NETWORK. AND THE TITLE IS IMPROVING EFFICIENCY OF NEWBORN SCREENING FROM COLLECTION TO RESULTS. THIS IS OUR RESEARCH TEAM, YOU CAN SEE THE NAMES HERE, DR. SONTAG IS ON IT, VERY HELPFUL AS A CONSULTANT. AND THE TEAM HERE IS VERY MULTI-DISCIPLINARY, IT'S ONE OF MY TAKE HOME POINTS, THIS IS A COMPLEX PROCESS INVOLVING MULTIPLE STAKEHOLDERS AND THEREFORE AS WE HAVE SEEN IN YVONNE'S PRESENTATION, INVOLVES THE MELDING OF MULTIPLE EXPERTS TO GET IT DONE. AND THAT'S WHAT WE HAVE DONE HERE IS HEALTH SERVICES RESEARCHERS, APPLY MATHEMATICIAN, QUALITY IMPROVEMENT EXPERT HEALTHCARE OPERATIONS ENGINEER, NEWBORN SCREENING AND HEALTH ECONOMISTS. THIS IS OUR ADVISORY COMMITTEE. NOT EVERYONE CAN SIT ON THE RESEARCH TEAM. SO WE MEET ON A REGULAR BASIS. ON THIS COMMITTEE YOU CAN SEE WE HAVE REPRESENTATION FROM SEVERAL STATES NEWBORN SCREENING PROGRAMS, HOSPITAL HEALTH ASSOCIATION, WORKING WITH NEW STEPS CLOSELY. AND THIS HAS BEEN INCREDIBLE AN INCREDIBLE RESOURCE IN TERMS OF SIFTING THROUGH THE DATA AND THINKING ABOUT THINGS IN WAYS THAT HOW THINGS RUN ON THE GROUND. SO WE'LL PRESENT PROJECT DESIGN AND GOALS, PRELIMINARY GOALS AN DISCUSS NEXT STEPS FOR THE PROJECT. SO THIS IS TO GET AGREEMENT AND BUY IN, WHICH I THIS THINK WE HAVE THIS IS A COMPLEX PROCESS, REQUIRES COORDINATED AND TIMELY COLLABORATION THROUGH MULTIPLE STAKEHOLDERS. YVONNE DEMONSTRATED THIS NICELY, WITHIN AND BETWEEN CLINICAL MEDICINE AND PUBLIC HEALTH. AND THERE ARE DIFFERENT WAYS TO ORGANIZE AND DELIVER NEWBORN SCREENING. EACH STATE PROGRAM, WE KNOW, DESIGNED ITS OWN PROCESS, I WANT TO BE CLEAR DIFFERENT DESIGNS ARE EQUALLY AFFECTED. DIFFERENT IS NOT BAD AS LONG AS ONTIVES CAN BE ACHIEVED. IN A COST CONTAIN MANNER THAT THE PROGRAMS CAN AFFORD. SO DIFFERENT IS NOT BAD. DIFFERENT MAKES IT DIFFICULT TO ASSESS THE PROCESSES ACROSS STATES WHERE LEVERAGE POINTS MIGHT BE USEFUL. SO THIS IS A PLUG FOR HEALTH SERVICES RESEARCH SLIDE. THIS PROBLEM IS WELL SUITED FOR HEALTH SERVICES RESEARCH, IT TALKS SYSTEM FACTORS, MANY THAT AFFECT QUALITY OF CARE THAT AFFECT HEALTH OF NEWBORNS. AND CAN BE ALL LEVELS FROM THE POPULATION DOWN TO INDIVIDUAL. THIS IS IN GENERAL A APPROACH WE TOOK TO EDUCATE THOSE IN OUTSIDE -- IN PUBLIC HEALTH NEW IMPORTANT SCREENING ABOUT THE GENERAL STEPS GOING ON. WE TALK ABOUT IN OUR GROUP, THE COLLECTION, THE TRANSPORT AND THE PROCESSING. THREE MAJOR STEPS AND WHAT IS HAPPENING WITHIN EACH OF THOSE STEPS WHERE THEY'RE HAPPENING AND WHAT'S HAPPENING FROM A TIMING, FROM A STAFFING, FROM A FREQUENCY AND AVAILABILITY PIECE. AS WE JUST NOTED, THE GOAL RIGHT NOW IS FIVE TO SEVEN DAYS DEPENDING ON RESULTS. SO WHY DID WE DO THIS PROJECT IF COIIN EXISTS AND NEW STEPS 360 EXIST? WELL, THIS PROJECT WAS MOTIVATED BY MY BEING PART OF THE COMMITTEE AS LIAISON, IS THERE A ROLE FOR TAKING A BROADER PERSPECTIVE OF THIS PROCESS? TO PERFORM A SYSTEMATIC ANALYSIS OF THE BROAD PROCESS AND IDENTIFY LEVERAGE POINTS TO INTERVENE AND IMPROVE PROCESS EFFICIENCY. HERE IS AN EXAMPLE. WE CAN FOCUS VERY TIGHTLY ON AREAS IN THE PROCESS WHEN WE KNOW THERE'S A PROBLEM. WE CAN FOCUS VERY TIGHTLY ON HOSPITALS AND THE COURIER. WE CAN GET THEM WE CAN MAKE INCREMENTAL PROGRESS ON EACH STEP I SHOWED YOU IN MAKING THEM -- MAKE LENGTH OF TIME SHORTER. BUT, I'M NOT SAYING THIS IS BAD, BUT THIS IS PART OF THE MOTIVATION OF THIS PROJECT. AT SOME POINT IT DOESN'T MATTER HOW FAST YOU ARE IN THE HOSPITAL IF WAITING FOR THE COURIER TO PICK YOU UP SO YOU EXHAUSTED YOUR ABILITY TO MAKE IT SHORTER. FROM THE SUBWAY ANALOGY, IT'S THE RED LINE IS COMING IN AT NOON, IT DOESN'T MATTER IF YOU GET THERE AT 11:59 OR 11:30. YOU'RE GOING TO GET ON THE BUS. SO THE QUESTION IS, THIS IS WHERE THE PERSPECTIVE COMES IN OF THE TOTAL PROCESS AND WHERE POTENTIAL LEVERAGE POINTS ARE THAT LEAD TO THE TOTAL PROCESS BECOMING MORE EFFICIENT. SO THE GOAL OF THE PROJECT WAS INNOVATIVE DYNAMIC SIMULATION MODELING, DR. COCHRAN HERE TECHNIQUES TO SYSTEMATICALLY IDENTIFY POTENTIAL PROCESS IMPROVEMENT STRATEGIES FROM REDUCING TIME FROM COLLECTION TO TEST RESULTS. AND THEN ASSESS THE TRADES OFF BETWEEN TIMELINESS AND COST FOR STRATEGIES IDENTIFY. NOT YOU DON'T WANT TO BUILD A PORSCH IF YOU DON'T HAVE THE MONEY IF YOU CAN BUILD A CIVIC AND GET THERE JUST AS FAST SO YOU MUST HAVE AN ASSESSMENT WHAT IS THE INCREMENTAL COST OF CHANGING THE SYSTEM AND WHAT YOU'RE GETTING FROM WHAT YOU'RE INVESTING. COST NOT JUST DOLLARS BUT RESOURCES. SO SIMULATION MODELING, THIS IS A TYPICAL METHOD FOR IDENTIFYING THE STEPS IN THE PROCESS THAT CAN BE MODIFIED. AND THE IMPLICATIONS ARE BY RUNNING MULTIPLE SIMULATIONS WITH DATA END POINT, THE IMPLICATION ARE IDENTIFYING A PROCESS AND IDENTIFYING THE STEPS IN THE PROCESS TO LINKED TO SIGNIFICANT TIMELINESS LEVERAGE POINTS AND TAILORED TO STATE SPECIFIC PROCESS. I THINK THE POTENTIAL HERE IS IT CAN GET US OUT OF THE WEEDS FOR A MOMENT. MANY STATES KNOW WHERE THEIR -- WHERE SOME OF THEIR PROBLEMS ARE. THERE MAYBE OTHER POINTS IN THE PROCESS WHICH ARE NOT SEEN BUT CAN BE LIFTED TO THE FOREFRONT WITH MODELING ANALYSIS THAT LOOKS AT THE ENTIRE PROCESS. EARLY CHALLENGES AND BARRIERS WHICH WE ALREADY DISCUSSED, YOU ARE AWARE OF, THIS IS A COMPLEX PROCESS, NOT ONLY IS EACH PROGRAM DIFFERENT EACH HOSPITAL IS DIFFERENT POTENTIALLY SO NOW YOU HAVE 83 AGENTS COLLECTING SPECIMENS IN POTENTIALLY DIFFERENT WAYS GOING TO A NEWBORN SCREENING LAB. AND YOU HAVE TO UNDERSTAND HOW THE PROCESSES WORK. YVONNE DEMONSTRATED IT'S NOT EASY. IMPLEMENTATION AT THE PROGRAM HOSPITAL LEVEL. WHO COLLECTS NATASHA SAID, WHO DOES WHAT JOB AND WHAT THEIR TITLE IS DEPENDS ON THE HOSPITAL. THAT YOU'RE TALKING ABOUT AND THE AVAILABILITY OF DATA IS DIFFICULT BECAUSE NOT EVERYONE IS COLLECTING ALL THE DATA USEFUL FOR THIS TYPE OF MODEL. AS I MENTIONED A FEW MINUTES AGO, WHAT IS THE HEALTH OUTCOME GAIN OF LESS THAN FIVE DAYS SO THE END OF THE DAY I CAN TELL YOU COST TO GET INCREMENTALLY HOURS BELOW OR TO FIVE DAYS BUT ULTIMATELY ANYONE WOULD ASK ME THE LAST PIECE ON THE HEALTH SERVICES MODEL WHICH IS SO HOW MANY LIES DID YOU -- LIVES DID YOU SAVE. THE DATA, IT IS DIFFICULT TO ACTUALLY I THINK GIVE AN ASSESSMENT OF HOW MANY BABIES PRESENT LESS THAN FIVE DAYS. I DON'T KNOW, PEEP -- THIS IS ALSO A PLUG FOR THOSE IN THE AUDIENCE THAT HAVE THAT SYSTEMATIC DATA THAT WILL BE HELPFUL. IF WE KNEW WHAT PERCENTAGE OF N CATS PRESENT AT THREE OR FOUR DAYS, THAT COULD BE BUILT INTO THIS MODEL. THIS IS DIFFICULT DATA TO GET BECAUSE IT MAY NOT BE SAM MATICALLY COLLECT -- SYSTEMATICALLY COLLECTED. SO I'LL TURN IT TO DR. COCHRAN TO PRESENT PRELIMINARY MODEL RESULT US. >> THANK YOU. I'M GOING TO FOCUS ON THE DATA ANALYSIS THAT WE DID AND I'M PARTICULARLY FOCUSED ON THE PART OF THE PROCESS THAT STARTS AT BIRTH. AND ENDS WHEN THE LAB STARTS PROCESSING AND THE ISSUE -- THEY ISSUE RECEIPT OF THE STARTING OF A PROCESS. THE DATA LOOKED AT IS COLLECTED FROM MICHIGAN NEWBORN SCREENING PROGRAM, SO THIS IS RUN BY STATE OF MICHIGAN. WE HAVE NEARLY 100,000 NDS SPECIMENS CHECKED OVER A YEAR ACROSS STATE OF MICHIGAN SO 83 BIRTHING HOSPITALS. I'M GOING TO PARTICULARLY FOCUS ON THOSE NEWBORNS NOT BORN TO A NICSCU OR SPECIAL CARE -- NICU OR SPECIAL CARE UNIT. WE HAVE SEVERAL CHARACTERISTICS THAT WE'LL LOOK MORE CLOSELY, HOSPITAL ID, THE TIME AND DATE OF BIRTH COLLECTION AND THE RECEIPT OF THE ARRIVAL, AS WELL AS MILEAGE AND PICK UP SCHEDULES IS THE LAB HOURS OF THE STATE. SO KIND OF THE FIRST THING WE DO IS ALWAYS TAKE A LOOK AT THE DATA, TO GET A BETTER INSIGHT INTO WHAT WE HAVE. THESE ARE THE DISTRIBUTIONS, TOP LEFT DISTRIBUTION OF BIRTHS ACROSS THE DAYS OF THE WEEK. SO VERY -- THIS IS AVERAGED OVERALL THE SPECIMENS THAT WE HAVE. AND WHAT WE FIND IS THAT -- AND THIS MAYBE NO SURPRISE, DURING WEEKDAYS FIRST ARE MORE COMMON THAN THEY ARE ON THE WEEKEND. IN ADDITION THROUGHOUT THE DAY BIRTHS ARE MORE COMMON AROUND 8 AND THAT SLOWLY DECLINES UNTIL AT NIGHT THEY'RE LESS COMMON. THIS IS AVERAGED OVER THE HOSPITAL BUT WE SEE SIMILAR PATTERNS BETWEEN HOSPITALS. SO WHAT DOES THAT MEAN AS FAR AS TIMELINESS? IF BIRTHS ARE MORE COMMON ON THE WEEKENDS, OR WEEKDAYS AND GIVEN THAT IN MICHIGAN YOU WAIT 24 HOURS BEFORE YOU COLLECT, COLLECTION IS GOING TO BE MORE COMMON ONE DAY SHIFTED OVER. SO YOU CAN SEE THIS IN THE DATA, FROM TUESDAY TO SATURDAY, IS MORE COMMON FROM SUNDAY TO MONDAY. SO THINKING STAFFING OR HOW MUCH EFFORT TO PUT INTO COLLECTION YOU HAVE TO THINK ABOUT THE FACT IT WILL BE MORE COMMON FROM TUESDAY TO SATURDAY. THE HOURS OF COLLECTION ARE -- DON'T HAVE SUCH A NICE TREND. WE'LL LOOK AT MORE CAREFULLY WHY PERHAPS THAT MIGHT BE. SO FROM THERE WE JUST TOOK THE PROCESS AND SPLIT UP INTO TWO PARTS. SO FIRST THE PART FROM BIRTH TO COLLECTION. AND THEN COLLECTION TO ARRIVAL OF THE LAB AND STARTING OF THE PROCESSING. AS FAR AS BIRTH COLLECTION WAIT 24 HOURS BEFORE THEY START COLLECTION BUT NEARLY 70% SPECIMENS ARE COLLECTED 24 TO 26 HOUSE IN THE STATE OF MICHIGAN. SO IT'S VERY TIGHTLY CONTROLLED, DOING A GREAT JOB GETTING COLLECTION. AND IN FACT OVER 99% OF THE SPECIMENS ARE COLLECTED WITHIN 36 HOURS. NOW, WHAT THAT MEANS IS IF YOU THEN GO STEP DOWN AND LOOK AT THE COLLECTION TO LAB TIME, YOU CAN IMMEDIATELY SEE A GREATER VARIABILITY IN THE TIME. SO FROM A SYSTEMS PERSPECTIVE, PERHAPS OUR RESOURCES ARE BETTER SPENT IN THE COLLECTION TO LAB TIME TRYING TO PROVE -- IMPROVE THAT BECAUSE OF THE HIGHER VARIABILITY. SO ONE THING THAT WILL DOCUMENT UP AGAIN IS THIS PICK UP AND WE TALKED ABOUT IT BEFORE BUT THIS COURIER PICK UP. SO THIS IS ALL THE HOSPITALS WHEN THE COURIERS ARE TYPICALLY PICKED UP. IN MICHIGAN THEY'RE TYPICALLY PICKED UP SIX DAYS A WEEK. SO EVERY WEEKDAY AND AROUND ABOUT 6 P.M. IS TYPICAL WEEKDAY TICK UP AND THEY'LL HAVE A PICK UP ONER SATURDAY OR SUNDAY SO MICHIGAN IN THE UPPER PENINSULA, THEY'RE TYPICALLY PICKING UP SPECIMENS ON SATURDAY. THEN BECAUSE THAT'S FARTHER AWAY FROM THE STATE LABORATORY BUT THEN OTHER HOSPITALS PICK UP 6 P.M. SUNDAY SO IN MICHIGAN IT'S IMPORTANT TO NOTE THERE'S A FIXED COURIER ROUTE. SO THEY PICK UP SPECIMENS, TRAVEL NOT DIRECTLY TO THE LAB BUT PERHAPS OTHER HOSPITALS BEFORE ARRIVING TO THE LAB. WE HAVE TWO HOSPITALS THAT ARE HAVE THEIR OWN COURIER TIME OR COURIER AND THEY GO DIRECTLY TO THE LAB, ABLE TO REALLY CUT THAT TIME BY ABOUT SEVEN HOURS. IN FACT, THE RESULTS THAT I PRESENT HOW LONG IT TAKES IS AN IMPORTANT FACTOR. FROM HERE WE WANTED TO UNDERSTAND A LITTLE BIT BETTER WHERE THAT VARIABILITY COMES IN THE COLLECTION TO LAB TIME. NOTICE THERE'S KIND OF THREE PEAKS, THEY'RE ALL SEPARATED BY DAY. WE DID A SIMPLE YOU CAN GET, A LINEAR MODEL, LINEAR REGRESSION TO SEE WHAT FACTORS ARE IMPORTANT TO THAT COLLECTION TO LAB ARRIVAL. ONE THING WE LOOKED AT WAS HOSPITAL VOLUME. AND THIS IS JUST IN TERMS OF HOW MANY BIRTHS THEY'RE HANDLING. THIS WASN'T SIGNIFICANT. IN FACT, THE SIZE IS NOT EVEN MAKING IT FASTER. WHAT WAS IMPORTANT WAS TIME OF COLLECTION. SO YOU CAN STAIR AT TUESDAY COLLECTION AND LOOK AT THE ESTIMATE. THAT'S IN HOURS SO WHAT THIS IS SAYING IS A TUESDAY COLLECTION ON AVERAGE IS ABOUT 12 HOURS FASTER THAN A SATURDAY COLLECTION. THIS IS ALL RELATIVE TO SATURDAY. IN FACT, THE FRIDAY COLLECTION IS ABOUT THREE HOURS LONGER THAN A SATURDAY COLLECTION. WE CAN LOOK AT TIME OF DAY, THIS ALSO CONTRIBUTES TO THE TIMELINESS. SO EARLY MORNING. COLLECTION ARE THREE HOURS FASTER ON AVERAGE THAN EVENING COLLECTION. MILEAGE TO LABORATORY, THIS HAS -- VERY INTUITIVE THAT THAT CONTRIBUTES TO THE TIMELINESS. THAT NUMBER, MEANS ABOUT TWO MINUTES PER MILE. TO DO THE RECIPROCAL. SO THAT MAKES SENSE. SINCE COLLECTION TIME THROUGHOUT THE DAY AS WELL AS ACROSS THE WEEK IS AN IMPORTANT FACTOR. YOU MAY ASK WHY, WHY IS FRIDAY AND SATURDAY SO MUCH SLOWER THAN THE OTHER DAYS OF THE WEEK? IN MICHIGAN WE HAVE A SIX DAY SCHEDULE FOR THE LAB. SO ON SUNDAY, IT'S CLOSE. IF YOU PICK UP SPECIMENS ON SATURDAY ARRIVING TO THE LAB SUNDAY, IT'S GOING WAIT UNTIL MONDAY BEFORE PROCESSING BEGINS SO THAT PERHAPS COULD BE CONTRIBUTING TO THE DELAY. THIS IS WHERE SIMULATION COMES IN. CAN WE EXPLORE THIS HYPOTHESIS FURTHER? COULD COLLECTION TIME BE SO IMPORTANT FOR THE TIMELINESS THROUGH ITS RELATIONSHIP BOTH TO COURIER SCHEDULE THAT WE TALK AS WELL AS LAB HOURS. SO WE USE THE DATA TO CREATE REALISTIC SIMULATION TO CAPTURE ALL THE PARTS OF THE SYSTEM FROM BIRTH TO LAB ARRIVAL. SO WE REPRODUCE PATTERNS OF BIRTH, INCLUDING UNCERTAINTY SO THERE'S RANDOMNESS INVOLVED MANY THE SIMULATION. THEN WE KIND OF TOOK THOSE BIRTH TO COLLECTION TIMES TO ALSO GENERATE SORT -- SOME SORT OF TIME, PICK UP ALLOWING FOUR HOURS OF DRYING IN SIMULATION. AND FIXED TRANSIT TIME. WE ASSUME PROCESSING BEGINS ONCE THE LABS OPEN SO ONCE THE SPECIMEN ARRIVED AND THE LAB IS OPEN. AS FAR AS THE TEN HOUR FIXED TRANSIT TIME, THIS IS JUST CAPTURING THE EXPERIENCE FROM THE PEOPLE WE WORK WITH IN MICHIGAN WHO SAY GIVEN ABOUT A TYPICAL PICK UP OF 6 P.M. THOSE SPECIMENS ARRIVED BETWEEN 3 AND 4 A.M. SO I WILL ASSUME A TEN HOUR SCHEDULE. SO THE RESULTS WILL DEPEND ON THE FIXED TRANSIT TIME AND WE HAVE LOOKED AT OTHER SHORTER TRANSIT TIMES AND HOW THAT MIGHT AFFECT THINGS. SO WITH SIMULATION WE CAN START TO EXPLORE WHAT YOU MIGHT WANT TO IMPLEMENT WERE YOU IMPLEMENT IT. SO IT'S A NICE WHAT IF SCENARIO TO SEE WHAT THE TRADE OFFS ARE. SO WE'LL PARTICULARLY FOCUS ON WHAT HAPPENS WHEN WE CHANGE THE LAB HOURS AS WELL AS WHAT HAPPENS WHEN WE CHANGE THE PAY OFF SCHEDULE. SO LAB HOURS ON THE BOTTOM FOR THE STATE OF MICHIGAN. FOR POINT OF REFERENCE. HERE LET'S FIX LAB HOURS AND SEE WHAT HAPPENS WHEN WE CHANGE THE PICK UP SCHEDULE. SO OUR BASELINE IS GOING TO BE THIS TYPICAL 6 P.M. SUNDAY TO FRIDAY PICK UP. THIS IS WHAT'S RETURNED FROM SIMULATION AND IT LOOKS SIMILAR TO WHAT THE ACTUAL DATA IS. WE HAVE THREE PEAKS AND THIS WIDE VARYN 'T. -- VARIABILITY. YOU MIGHT SAY WE HAVE SIX DAYS, WHAT IF WE SWITCH OUR SUNDAY PICK UP TO A SATURDAY PICK UP. SO THE UPPER RIGHT WE'RE LOOKING AT THAT SCENARIO. AND WHAT YOU FIND IS YOU HAVE LOOKED OUT TOWARD THE 86 HOURS, IN THE 6 P.M. SUNDAY THROUGH FRIDAY, YOU HAVE A LOT LESS SPECIMENS THAT ARE COLLECTED AT THAT VERY DELAYED TIME. WHEN YOU COMPARE TO THE 6 P.M. MONDAY THROUGH SATURDAY. AGAIN, THAT MAKES SENSE. IF YOU SWITCH YOUR PICK UP TO SATURDAY, THEY PICK UP ON SATURDAY BUT THEN WAIT ON SUNDAY UNTIL THE LAB OPENS SO SWITCHING THAT WOULD DELAY THE PROCESS FURTHER. THERE'S OTHER THINGS WE CAN DO, SO RATHER THAN SWITCHING THE DAY YOU CAN SAY DELAY PICK UP BY HOURS, SO THAT'S 12 A.M. MONDAY THROUGH SATURDAY PICK UP. IF YOU COMPARE THE TWO -- THE UPPER LEFT TO BOTTOM LEFT YOU CAN SEE THAT THE CURVE IS SHIFTED TO THE LEFT. SO YOU IMPROVED TIMELINESS FOR THE MAJORITY OF THOSE SPECIMENS. SO WE CAN LOOK AT THAT A LITTLE BIT MORE CAREFULLY WITH NUMBERS. SO WE RAN A SIMULATION, WE TRIED 35 DIFFERENT SIMPLE PICK UP SCHEDULES, THESE ARE SIX DAY SCHEDULES, 12 A.M. 6 P.M. 12 P.M. 6 P.M. 9 P.M. I WANT TO SAY WE ALSO TRIED SEVEN DAY SCHEDULES BUT BECAUSE THE LAB ISN'T OPEN ON ONE OF THOSE DAYS, YOU ACTUALLY DON'T GRADE IMPROVEMENT SO IMPORTANT THING TO KNOW. IF YOU GO SIX DAY ONE DAY A WEEK TO SEVEN DAY ONE DAY A WEEK. SO WE HAVE A RANKING SYSTEM AND YOU CAN SEE THE 6 P.M. SUNDAY THROUGH FRIDAY OUR BASELINE AGAIN. SO IF WE SWITCH FROM 6 P.M. SUNDAY FRIDAY TO 12 A.M. MONDAY TO SATURDAY, YOU GET ON AVERAGE A FOUR HOUR IMPROVEMENT. YOU CAN ALSO AT REALLY LONG DELAYED AND YES, PROCESSES. SO WE CAN LOOK AT THOSE SPECIMENS THAT TAKE LONGER THAN 60 HOURS TO GO FROM BIRTH TO WHEN THEY ARE ISSUED A RECEIPT. AND YOU CAN SEE REDUCTION FROM ABOUT 14.6% TO 3 #% SO 14% LESS SPECIMENS ARE COLLECTED AFTER -- ARRIVE IN THE LAB AFTER 60 HOURS OF BIRTH. SO AGAIN, THIS IS JUST WAYS TO COMPARE BEFOREHAND WHAT WOULD HAPPEN IF YOU CHANGE YOUR COURIER SCHEDULE. I DO WANT TO SAY THIS IS A CAVEAT, I'M ONLY FOCUS CANNING ON TIMELINESS. AND SO IN FACT WHEN YOU GO FROM A 6 P.M. 12 A.M. THAT MIGHT AFFECT OTHER THINGS. SO THE 10 A.M. TRAP SIT TIME, THOSE 12, IF YOU PICKED SCHEDULES, PICK THEM UP AT 12 A.M., THEY'RE ARRIVING TO THE LAB AT 10 A.M. HOWEVER OUR LAB OPENS UP AT 7 SO THEY'RE STARTING THEIR PROCESSING LATER. AS CONSEQUENCE BY THE TIME THEY FINISH PROCESSING IT MIGHT BE TOO LATE IN THE AFTERNOON TO CONTACT YOUR PRIMARY CARE PROVIDER. THAT'S A BIG CONCERN THAT MICHIGAN IS FOCUSING ON. SO YOU COULD SAY -- ACTUALLY DO A 9 P.M. IN BY CASE THOSE ARRIVE AT 7 A.M. RIGHT WHEN THE LAB OPENS. AND YOU GET SIMILAR RESULTS TO THE 12 A.M. SO THESE ARE THINGS YOU CAN EXPLORE APRIORI BEFORE YOU ACTUALLY CHANGE IT IN THE SYSTEM. WE LOOKED AT CHANGING LAB HOURS. A LOT OF STATES LOOK AT WHAT HAPPENS FROM A FIVE DAY TO SIX DAY SCHEDULE, WHAT HAPPENS WHEN I GO FROM A 6 DAY TO SEVEN DAY SCHEDULE. THAT'S THE UPPER ONE IS CURRENT ONE IN MICHIGAN, SO THAT'S KIND OF THE BASELINE. WE CONSIDERED ALL THESE THINGS, WE CONSIDERED SHIFTING THE LAB HOURS, SO MICHIGAN IS SHIFTING EARLIER FOR THE REASON OF TRYING TO GET TO THE PRIMARY CARE PROVIDER EARLY ENOUGH. WHEN WE DO THAT WE CAN SEE LITTLE CHANGES, SO SECOND TO LAST ROW 5 A.M. TO 3 P.M. HAS SIMILAR RESULTS TO THE CURRENT LAB SCHEDULE SO BENEFICIAL FROM PERSPECTIVE OF CONTACTING A PRIMARY CARE PROVIDER. SO KIND OF BROAD CONCLUSIONS. BECAUSE MICHIGAN IS DOING SUCH A GOOD JOB WITH THE COLLECTION, THE BOTTLENECK NOW IS THAT TIME FROM COLLECTION TO LAB ARRIVAL. WE NARROWED THROUGH SIMULATION AND REGRESSION ON THE PICK UP SCHEDULES AS WELL AS THE LAB HOURS. HOW WE CAN ADJUST THEM AND GENERAL GUIDELINES IS FIRST RECOGNIZE THERE ARE PATTERNS OF BIRTH SO YOU MIGHT WANT TO CONSIDER A SYSTEM THAT TAKES THAT INTO ACCOUNT AS WELL AS WHEN THE LAB IS OPEN. SO IF SPECIMENS ARE SITTING THERE WAITING FOR THE LAB TO BE OPENED THAT'S NOT IMPROVING THE PROCESS. SIMULATION, I'M A MODELER SO I ALWAYS PLUG SIMULATION. CAN GIVE US SOME IDEAS BEFORE WE ACTUALLY CHANGE. OF COURSE THIS IS NOT CAPTURING EVERYTHING. AND WE ALSO DIDN'T REALLY FOCUS ON THE LAB PROCESSING. WHICH WE'LL ALSO HAVE PROBABLY OTHER BOTH NECKS TO CONSIDER. SO TURN IT BACK OVER TO BETH TARINI. >> FROM THIS IS THEIR CHANCE TO MODEL GREAT DATA THEY HAVE TO GET A MODEL RUNNING TO SEE HOW IT WORKS SO THE NEXT STEP IS TO REFINE THE MODEL WITH ADDITIONAL DATA FROM SURVEYS OF OTHER HOSPITALS AND STATE NEWBORN SCREENING PROGRAMS AND OF COURSE I ENSURE YOU'RE ALL THINKING THAT'S GREAT, I CAN CHANGE AND OPEN MY LAB BUT WHAT WILL I DO AND HOW MUCH WILL IT COST ME. THAT IS ANOTHER GOAL OF OURS TO GET DATA. OF COURSE WE KNOW FROM THE PRELIMINARY DISCUSSIONS WE HAVE HAD WITH THE COST THOSE ARE NO EASY FEATS TO FIND THAT DATA AND WHO IS PAYING FOR IT IS A WHOLE 'NOTHER PIECE. BEFORE I END I HAVE ON THE LINE MARY KLINE WHO WAS EPIDEMIOLOGIST AT MICHIGAN AND WHO IS ONE OF THE NEWBORN SCREENING NURSE COORDINATORS, SO I WANT TO GIVE THEM A CHANCE IF THEY HAVE ANY COMMENTS BEFORE WE GO INTO THE QUESTION PERIOD. I THINK THEIR LINES ARE OPEN. >> THIS IS MARY, CAN YOU HEAR ME? >> YES WE CAN. >> PERFECT. I DON'T THINK (INAUDIBLE) PRESENTATION REALLY INTERESTING, SEE ALL THIS SIMULATION MODEL SO I JUST WANT TO HEAR IF ANYBODY HAS ANY SPECIFIC QUESTIONS ABOUT OUR PROCESS, I'M HAPPY TO ANSWER THEM. >> THANKS, MARY. AND LOIS? >> I JUST HAVE ONE COMMENT WORKING WITH HOSPITALS (INAUDIBLE) SO COURIER PICK UP TIME LATER IN THE DAY THERE MAYBE NO HOSPITAL PERSONNEL TO ACTUALLY PACKAGE THE SPECIMENS TO THE COURIERS. >> IMPORTANT THING TO REMIND ME, GETS BACK TO DR. COCHRAN'S POINT THAT YOU CAN'T TAKE THE MODEL AND GO. WITH ANY DATA YOU HAVE TO TAKE THE MODEL AND SAY THIS IS WHAT WE MIGHT DO. AND THEN CONSIDER GIVEN THE CONTENT EXPERTISE AROUND THE TABLE, WHAT ARE THE OTHER OPPORTUNITIES COSTS THAT WE' RUN INTO OR OTHER PROBLEMS WE'RE GOING TO CREATE. LIKE WHEN YOU DO SOMETHING, SEE WHAT THE COLLATERAL DAMAGE COULD BE FROM YOUR INTERVENTION. YOU CAN'T PRESUME IT'S NULL. THANK YOU, LOIS. NOW THE DOOR IS OPEN TO QUESTION. >> PETER. >> GREAT WORK, GREAT PRESENTATION. THANK YOU VERY MUCH. I SIT HERE AND WONDER WHETHER ONE SHOULD REVISIT THE ISSUE WHEN THE SAMPLE IS COLLECTED. SO IN THE NEW STEPS WE HEARD BECAUSE CALIFORNIA IS PART OF IT, ONE LOOKS AT 12 TO 48 HOURS AS COLLECTION. IN MICHIGAN YOU LOOK AT STATUS QUO. >> 24 TO 36. >> THE DATA SHOW THEY'RE TRYING TO MEET THE 24 HOURS AT LEAST. >> YES. >> WHAT COULD YOU MODEL FOR 12 HOURS? AGAIN, LOOKING HOW THE OBs ARE DELIVERING THE BABIES. I ASSUME IT'S BIOLOGY THAT DICTATE THE WEEKENDS. SO -- AND THEN DELIVER MOSTLY IN THE MORNING SO 12 HOUR COLLECTION WOULD MEAN THEY COLLECT IN THE EVENING. ED ON WOULD THAT MAKE ANY DIFFERENCE, THAT WOULD BE INTERESTING TO KNOW. MAYBE YOU ADD CALIFORNIA TO YOUR STATE YOU WANT TO LOOK AT. THAT MIGHT BE WORTHWHILE. >> WE CAN CERTAINLY MODEL THAT. ONE COLLATERAL PIECE TO LOOK AT, I KNOW A FEW COMMENTS HERE THIS TIME BUT THE PAPER THAT WAS PUBLISHED OUT OF CALIFORNIA TALKS ABOUT WHAT HAPPENS WHEN YOU GET THOSE 12 HOURS, YOU DON'T SEE A SIGNIFICANT SHIFT IN THE METABOLIC BUT YOU DO SEE INCREASE IN THE FALSE POSITIVE RATE OF THE HORMONE TEST SO YOU PICK UP ANOTHER PIECE YOU MIGHT HAVE TO LOOK AT. >> AND AS I SAID LAST TIME, (INDISCERNIBLE) IS LOOKING AT THIS WITH CLEAR AND CAN ADJUST THE RESULTS. BY BIRTH WEIGHT PER HOUR. >> IF WE CAN DO THAT WE CAN ADDRESS THAT PROBLEM THAT WOULD BE CREATED BY THE CURRENT STATE OF AFFAIRS SO THAT'S HELPFUL. THANK YOU, DEAR. >> THE ONE COMMENT THAT I WAS GOING TO MAKE WAS THE -- >> TOOK THE WORDS OUT OF MY MOUTH. >> IN YOU DID, CAH AND THYROID FALSE POSITIVE RATE GOES UP DRAMATICALLY. THE OTHER THING I WANTED TO REINFORCE, FIRST OF ALL, THAT WAS FABULOUS. CLEARLY A WAY THAT WE HOPEFULLY -- I WOULD BE INTERESTED TO KNOW HOW MUCH IT ACTUALLY COSTS TO DO THAT KIND OF SIMULATION BECAUSE THAT IS THE RIGHT WAY TO BE GOING ABOUT SOLVING EVERY PROBLEM. AND HOW MUCH CAN THAT APPROACH, CAN IT BE SCALED TO BE USED BY PEOPLE WITHOUT A GRANT FROM THE ROBERT WOOD JOHNSON FOUNDATION. SO THAT'S ONE QUESTION. THE OTHER IS DEALING WITH OTHER LABORATORIES, SOMETIMES INTRODUCING OLD FAGGED TECHNOLOGY, IF YOU WANT THE COURIER TO GO TO BIRTHING CENTER TO PICK UP THE SAMPLE, YOU'RE ACTUALLY PUTTING YOUR HOSPITAL LAB IN A MAJOR RISK UNLESS THERE'S A SOLUTION INTRODUCED THERE, BECAUSE THAT'S NOT JACO OR CAPPED BECAUSE THEY HAVE TO HAVE THE SPECIMEN ASESSION AND YOU CAN'T HAVE SATELLITE LABORATORIES ANY MORE. CDC COULD TELL US MORE ABOUT THAT. BUT ONE SOLUTION USED IN ONE PLACE IS SEND SAMPLE OUT DIRECTLY AND MAKE A COPY AND THE LAB CAN SESSION BASED ON THE COPY WHILE THE SAMPLE GOES DIRECTLY. SO THERE ARE SOLUTIONS BUT CAN'T ALWAYS BYPASS THE LABORATORY. >> FIRST QUESTION ABOUT THE COST, THIS IS THE BEAUTY OF HEALTH SERVICES. YOU ARE NOT BUYING A MACHINE, THOUGH THAT'S SOME COST. YOU'RE BUYING THE EXPERTISE INDIVIDUAL WHO MAY HAVE OTHER EXPERTISE AS WELL. SO THERE'S NO REASON, AM I RIGHT? SO FOR A GRANT I GET BEST OF THE BEST. TO FIGHT FOR THE DOLLARS. NOW, THIS MODELING PRACTICE, I BELIEVE, CORRECT ME IF I'M WRONG, THERE CAN BE WAYS TO DO THIS, I'M IN IOWA, DR. COCHRAN IS IN MICHIGAN. SHE WORKS THE DATA REMOTELY FOR ME. SO ACTUALLY DOESN'T HAVE TO BE IN MY LAB. I UTILIZE HER TIME AND PAY HER FOR THAT TIME. SO THERE IS AN ACCESS AND COST ISSUE THAT I THINK POTENTIALLY COULD BE FELT, LET ME QUALIFY, I'M SAYING IT'S NOT -- THERE CAN BE CREATIVE SOLUTIONS TO EMORY'S POINT TO GET AT THIS UTILIZATION SKILL. YOUR SECOND POINT ABOUT COURIER, I'M NOT SURE I UNDERSTAND. IF THEY ARE ON HOSPITAL GROUNDS IS A PROBLEM? >> IS IT THE PHYSICAL SAMPLE ISN'T IN THE LABORATORY, THE LABORATORY IT IS MY UPPING HOSPITAL LABORATORY -- WE USED TO HAVE SATELLITE LABORATORIES ALL OVER HOSPITALS AND THEY REALLY STOPPED THAT. IT'S RELATED TO JACO AND CAT AND THE LABORATORY HAS TO HAVE CONTROL OVER ALL SPECIMENS, IT HAS TO BE A SESSION. AND THE LABORATORY CAN CHANGE WORK FLOW WITHIN THE LABORATORY BUT THE SAMPLE NEVER GOT TO THE LABORATORY, IT WAS PICKED UP FROM THE BIRTHING CENTER. >> EXACTLY. >> I'M GOING TO ASK LOIS WHO IS ON THE LINE, THERE ARE -- ARE THERE HOSPITALS IN MICHIGAN, THE SMALLER HOSPITALS WHERE THEY GO TO THE LABORATORY? OR ACCESSIONED ON THE FLOOR OR DO YOU KNOW? >> THERE ARE MANY HOSPITALS (INAUDIBLE) ON THE FLOORS AND THEY HAVE THEIR HONE -- SOME DO SOME DON'T THIS WAS THE FIRST TIME I HEARD THIS AS A COB EARN IS SO WHEN WE HAVE OUR (INAUDIBLE) IT'S DEFINITELY A QUESTION I WOULD ASK THEM. >> GREAT EXCHANGE OF INFORMATION SO THIS GETS TO THE OTHER ISSUE WHICH IS, IT'S A MIX SO WHEN I GO TO DESIGN A -- THIS IS WHERE IT'S THE PEOPLE ON THE GROUND UP TO 30,000-FOOT VIEW OF THE MODELER. I HAVE TO UNDERSTAND WHAT EACH PROCESS OR PROGRAM, UNDERSTAND WHAT EACH PROCESS IS ON EACH HOSPITAL. ONCE THOSE PROCESSES THE FIRST OF THE THREE SECTIONS IS TIGHTENED YOU CAN LOOK AT THESE OTHER PIECES TO YOUR POINT ABOUT DOS THAT'S AN IMPORTANT PIECE TO LOOK AT, I HAVE DR. BURBRIDGE HAS DONE WHEN IOWA, CORRECT ME IF I'M WRONG, IOWA DID A COST ANALYSIS BEFORE THIS HAPPENED, THEY WENT SEVEN DAY AS WEEK 24 HOURS A DAY. MY UNDERSTANDING IS THE ARGUMENT MADE TO THE -- TO THE PUBLIC HEALTH DEPARTMENT WHAT THEY HAVE BEEN, IS IN DOLLARS ONLY, NOT TALKING THE ACTUAL ABILITY TO HAVE SOMEONE RUN THE LAB ON A -- SUNDAY OR HIGHER PEOPLE OR HAVE THEM RUN AT NIGHT BUT IN TERMS OF DOLLARS, IT'S ON PAR POTENTIALLY WITH ADDING A NEW DISORDER. SO WHEN WE -- I PUT THIS OUT THERE AS A THOUGHT. WE DON'T THINK TOO MUCH, WE DON'T DISCUSS IN THIS COMMITTEE EXPLICITLY DOLLARS SPENT WHEN ADD A DISORDER. BUT WE TALK DOLLARS SPENT WHEN WE ARE RUNNING THE LAB AND TIMELINESS. NOT SAYING RIGHT OR WRONG, JUST POINTING OUT. SO IF WE ARE HAVING DOLLAR CONVERSATIONS ABOUT TIMELINESS WHICH AFFECT ALL DISORDERS, WHY ARE WE NOT HAVING THOSE CONVERSATIONS ABOUT ADDING A DIGS ORDER IF THOSE ARE COMPARABLE COSTS. ONE AFFECTS ONE DISORDER AND ONE AFFECTS 50. >> JOHN BAILEY, THANKS SO MUCH FOR A GREAT PRESENTATION. I LOVE SOPHISTICATION OF THE ANALYSES. A COUPLE OF OBSERVATIONS AND A QUESTION. SO IN REGARD TO COST, YOU HAVE GOT KEY -- A KEY COST IS GETTING THE ACTUAL RAW DATA TO TO BEGIN WITH, MICHIGAN SEEMS TO HAVE VERY GOOD SYSTEM OF TIMELINESS ESSENTIALLY, I DON'T KNOW HOW MANY OTHER STATES HAVE THAT LEVEL OF DATA. I CAN SEE FROM YVONNE'S PRESENTATION UNDERSTANDING AND END POINT OR THE OUTPUT OF THIS BUT TO ACTUALLY HAVE THE TIMING OF THIS, IS ONLY WAY YOU CAN DO THE MODELING IN ANY COST EFFECTIVE WAY. >> YOU HAVE TO TRACK SPECIMENS MORE THAN HOW MANY GOT HERE, X PERCENT ARRIVED AT THE TIME, THAT'S CORRECT. >> THEN I WANT TO ASK DR. COCHRAN -- THIS IS JUST A QUESTION FROM MY OWN INTEREST, IS THERE A DIFFERENCE BETWEEN MATHEMATICAL MODELING AND SIMULATION AND WHAT PEOPLE WOULD DO IN OPERATIONS, CONCERN FOR OPERATIONS BIAS? I THINK THAT'S -- YOURS IS A VERY SOPHISTICATED MATHEMATICAL LEVEL, MY UNDERSTANDING OPERATIONS RESEARCHERS IS TAKING AND SAYING OKAY, WE HAVE THE SYSTEM, WE HAVE 27 ELEVATORS, HOW WE PROGRAM THOSE ELEVATORS IN A WAY THAT MAXIMIZE EFFICIENCY. ARE YOU IN A PROGRAM THAT DOES THAT KIND OF WORK? I WANT TO ASK MARY KLINE, BECAUSE SHE'LL KNOW, HOW DIFFICULT IS IT TO TIME STAMP THE DATA, HOW DIFFICULT DID YOU FIND DO THAT AND WHAT WERE BARRIERS AND CHALLENGES? >> THIS IS AMY. (INAUDIBLE) DESIGN TIME STAMP BEFORE. SO IN TERMS OF -- COLLECTION DATE AND TIME. SO KNOB LABORATORY WE ENTER THAT INTO THE SYSTEM, THAT WAS ALREADY AVAILABLE. TRACKING LABORATORY RECEIPT DATE TIMES, WHAT HAPPENS IS WHEN THE SCIENTISTS OR TECHNICIAN LOGS TO THE COMPUTER IN THE MORNING, WHICH IS A TEST TOOL MARK SCANNER (INAUDIBLE) SCAN THEN THAT TIME -- THAT DATE AND TIME STAMP IS AUTOMATICALLY ADDED TO OUR SECTION NUMBER UNIQUE IDENTIFIER WE USE TO TRACK THE SAMPLE THROUGHOUT THE WHOLE TESTING PROCESS IN THE LABORATORY. SO ALL OF OUR DATE AND TIME STAMPS ARE COLLECTED AND TRACKED IN OUR SOFTWARE. >> SO YOU MAKE A GOOD POINT. NOT EVERY STATE CAN DO THIS NOW. BUT I THINK IT'S REASONABLE TO CONSIDER, THE ABILITY TO ASPIRE TO IT, SPECIALLY WITH OTHER POTENTIAL DOWNSTREAM. IT ALREADY EXISTS. AND YOU CAN HAVE DATA. MICHIGAN, OFTENTIMES WE SAY OH, WELL, WE CAN ASK SO AND SO BECAUSE THEY'RE COLLECTING DATA SO IT'S BASICALLY EXPONENTIAL PIECE. DR. COCHRAN. >> THAT WHOLE DESCRIPTION -- >> HE'S BY THE WAY POTENTIALLY HIREABLE SO SHOULD ANYBODY BE LOOKING FOR -- >> I WANT TO SAY SO ONE OF OUR COLLABORATORS, WHO IS ACTUALLY MY HUSBAND IS OPERATIONS RESEARCH. SO WE WORKED VERY CLOSELY WITH EVERYONE ON THE TEAM AND THE TWO OF US WERE THE ONES SITTING AT THE COMPUTER DOING THIS CLOSELY TOGETHER. SO WE HAD MORE EXPERTISE MODELING THE PROCESS NOT MY BACKGROUND BUT HE WAS GUIDING MAKING SURE THAT WAS THE PROPER WAY. >> REALLY OBSERVATION, I LOVE THE SYSTEMS APPROACH AS OPPOSED TOK LOUING AT EACH INDIVIDUAL PIECE. AND IT HELPS SEE ANALOGY RIGHT WAY IS A VERY GOOD ONE, TAKING DATA AND MAKING CHANGES ASSUMES IN THE EASIEST WAY WITH CONTROL OVER EACH STEP OF THAT PROCESS AND DIFFERENT PEOPLE HAVE DIFFERENT -- HAVE CONTROLS OVER DIFFERENT STEPS OF THE SYSTEM. >> CORRECT. >> FROM THE LAB DOESN'T REALLY CONTROL WHAT HAPPENS IN THE HOSPITAL. HOSPITAL DOESN'T CONTROL WHAT THE COURIER DOES, ET CETERA. SO I LOVE THE SYSTEMS APPROACH. I THINK MODIFYING A WHOLE SYSTEM IS DIFFERENT FROM MODIFYING ONE PIECE OF THE SYSTEM. >> AGREE. AND I WOULD COUNTER THAT WE MODIFY A WHOLE SYSTEM WHEN WE ADD A NEW DISORDER. SO IT IS NOT NEW TO US TO MODIFY THE SYSTEM. BUT WHEN IT COMES TO CERTAIN THINGS BUT THIS IS AN AREA THE PROGRAMS YOU SAW FROM YVONNE WHO KNOWS WHERE THE CONTRACT IS, WHO KNOWS WHAT THE CONTRACT IS MODIFIABLE. THESE PIECES, I HAVE FOUND IN NEWBORN SCREENING IN GENERAL, IT IS NOT BAD, IT JUST IS WHEN WE HAVE DONE SURVEYS FROM MY TEAM, THERE ARE MANY PEOPLE INVOLVED AND VERY GOOD AT WHAT THEY DO. SOMETIMES THE INFORMATION UNDERSTANDING THEY ALL KNOW WHO DOES WHAT, BUT TRYING TO UNDERSTAND AND CONNECT THOSE PEOPLE AND UNDERSTAND THE LARGER PICTURE IS DIFFICULT. WHICH MAKES ACTUALLY KNOWING IF YOU CAN AFFECT IT ONE STEP AND ACTUALLY AFFECTING CHANGE A SECOND -- SO I THINK YOU'RE RIGHT. THIS SHOWS YOU THE POTENTIAL LEVERAGE POINT, THEN YOU MUST GO INTO THE REALITY, ARE THEY COST EFFECTIVE AND REWARDING AND HOW MANY DO YOU GET -- HOW MUCH JUICE YOU GET FOR THE SQUEEZE AND CAN YOU ACTUALLY DO IT. >> I GIVE YOU THE LAST COMMENT. >> I HAVE A QUESTION. AGAIN, I LIKE THIS BECAUSE I FEEL WE ARE GOING TO AT THIS BASE -- ONE THING I WAS WONDERING ALL THIS MONITORING CAN BECOME -- BECAUSE EACH STATE, YOU BASE ON THE MICHIGAN DATA, WE HAVE ALL THE DATA, DATA POINTS BUT IT'S DIFFERENT LIKE A COURIER -- SO OFFICE YES. >> SO THE GOAL AND THE WAY THIS WAS POSED WAS THIS WOULD BUILD A PROCESS MODEL IN THE SENSE OF PROCESS MODEL THAT CAN BE MANIPULATED, YOU CAN PUT IN DIFFERENT STEPS INTO IT AND THEN THEY INPUT THE DATA SO THE TECHNIQUE CAN BE TWEAKED FOR THE STATES AND THAT IS THE OPPORTUNITY FOR -- TO EFFECT CHANGE, IT'S A TOOL THAT CAN BE MODIFIED AND USED. IF YOU WANT TO COMMENT MORE. ON THE SOPHISTICATION. >> I THINK REALLY WHAT IT SEEMS LIKE THE NEXT STEP IS TO TAKE THIS AND TURN IT INTO SOME SORT OF -- WHERE SOMEONE COULD INPUT THEIR OWN DATA. YOU HAVE THE MONEY TO DO IT. RIGHT? THIS IS THE -- SO A DIFFERENT STATE COULD PUT IN THEIR OWN DATA AND MODEL THE WHOLE PROCESS, I THINK THAT WOULD ACTUALLY BE PRETTY STRAIGHT FORWARD AND NORMALLY DONE FROM MY PERSPECTIVE. >> GOOD WORK. >> SO I HOPE A NEW STEP CAN BE PLACED -- >> I WILL CALL YOU. BECAUSE -- >> HAPPY TO. >> WE'LL DISCUSS. AFTER. >> ALL RIGHT. THANK YOU VERY MUCH. BOTH BETH AND AMY FOR THE PRESENTATION. WE REALLY APPRECIATE IT AND LOOK FORWARD TO THE NEXT PHASE OF YOUR STUDY. SO WE ARE JUST A FEW -- FIVE MINUTES BEHIND. WE'RE GOING TO COME BACK AT 10 MINUTES TO 11:00. SHORT BREAK. AND THEN BE BACK FOR THE NEXT SPEAKER. THANK YOU. >> LET'S WELCOME BACK FROM THE BREAK. WE'RE READY TO START THE NEXT PRESENTATION AND NEXT TWO PRESENTATIONS ARE GOING TO BE BY INDIVIDUALS WHO ARE PRESENTING TO US BY TELEPHONE. THE FIRST IS DR. SHARMINI ROGERS. DR. ROGERS IS THE CHIEF OF THE BUREAU OF GENETICS -- BUREAU OF GENETICS AND HEALTHY CHILDHOOD FOR MISSOURI DEPARTMENT OF HEALTH AND SENIOR SERVICES. SHE WILL TALK TO US TODAY ABOUT MISSOURI'S EXPERIENCE IMPLEMENTINGLYZE -- IMPLEMENTING LYSOSOMAL STORAGE TALKING ABOUT FOLLOW-UP FOR POMPE GO CHEER FABRE AND KRABBE DISORDER. DR. SHARMINI IS WITH THE MISSOURI DEPARTMENT OF HEALTH AND SENIOR SERVICE WAS FOR A NUMBER OF YEARS. SHE HAS OVERALL RESPONSIBILITY FOR THE NEWBORN SCREENING PROGRAM, GENETIC PROGRAM SUCH AS CYSTIC FIBROFIBROSIS, SICKLE CELL AND FOR INDIVIDUALS IDENTIFIED WITH METABOLIC DISORDER. DR. ROGERS SCHEDULE MADE IT IMPOSSIBLE FOR HER TO TRAVEL BUT SHE KIND AGREED TO SHARE HER EXPERIENCE BY PHONE. SO DR. ROGERS. WE'RE READY WHEN YOU ARE. >> THANK YOU, GOOD MORNING TO ALL OF YOU I WOULD LIKE TO THANK DR. (INAUDIBLE). ALL THE INFORMATION I SHARE THIS MORNING IS THE HARD WORK OF MANY PEOPLE IN THE STATE LAB, MY FOLLOW-UP STAFF AND (INAUDIBLE). WITHOUT THEM WE HAVE HAVE NO STORY THE TELL YOU THIS MORNING. NEXT SLIDE, PLEASE. MY GOAL FOR TODAY ARE REALLY TO PROVIDE THE LEGISLATIVE BACKGROUND, PROCESS, (INAUDIBLE) HOW WE IMPLEMENT SHORT TERM FOLLOW-UP AND CON TERMTORY RESULTS ALSO WANT TO TAKE THIS OPPORTUNITY TO THANK (INAUDIBLE) CHALLENGES WE CONTINUE TO FACE AND LESSONS LEARNED. NEXT SLIDE, PLEASE. AS MANY OF YOU KNOW (INAUDIBLE) BEHIND ILLINOIS LEGISLATION AND FOLLOW THE SAME LANGUAGE. (INAUDIBLE) OVER 267 SADLY NOT READY. IN PAST IN 2009 AND HE HAD UNTIL JULY 2012 TO START SCREENING. (INAUDIBLE) THEY DON'T SPECIFY WHICH (INAUDIBLE) SCREENING THEY GAVE US AN OPTION TO ADD OTHER (INAUDIBLE), NEXT SLIDE PLEASE. CAME TO (INAUDIBLE) NEXT SLIDE PLEASE. WHEN THE LAW PASSED, (INAUDIBLE) SCREEN AND FINALLY DECIDED TO USE MICROFLUIDICS METHOD. WHEN TIME CAME FOR IMPLEMENTATION WE DISCOVERED WE WERE NOT REALLY READY (INAUDIBLE) AND GETTING PRESSURE TO START SCREENING. SO WE CONTRACTED WITH NEW YORK TO DO A SCREEN (INAUDIBLE) ESPECIALLY TO DR. (INAUDIBLE) A TASK FORCE WAS CREATED IN EARLY 2012 TO DEVELOP FOLLOW-UP GUIDELINES. WE MEET REGULAR I BY CONFERENCE CALL AND (INAUDIBLE) BEGAN SCREENING IN 2012 AND POPULATION BASED POMPE GOECHER (INAUDIBLE) WITH INTENTION OF SCREENING IN HOUSE WHICH WE BEGAN (INAUDIBLE). NEXT SLIDE PLEASE. A LITTLE BACKGROUND (INAUDIBLE) 78,000 SCREENS 92,000 SAMPLES GIVEN THE SPECIMEN (INAUDIBLE). ARCH 375 SPECIMENS DAILY. TWO FULL TIME EMPLOYEES DEDICATED TO SCREENING FOR MSD AND THE FOLLOW-UP STAFF HAVE (INAUDIBLE) DATES DOWN TO TWO STAFF WORKING ON THE FOLLOW-UP SO THAT WE HAVE (INAUDIBLE). THE OTHER (INAUDIBLE) PROVIDE DATA. AND THEN FOUR CONTRACT CENTERS TO PROVIDE THE FOLLOW-UP IN CON -- CONFIRMATORY TESTING. NEXT SLIDE. SO WE WANTED TO SHOW YOU THE PLATFORM (INAUDIBLE). SO WE HAVE STAFF NOT SURE WHICH OF THE EIGHT IS IN WHITE. YOU CAN SEE THE SMALL PLATFORMS THAT DO NOT TAKE UP MUCH SPACE AND EASILY HANDLED (INAUDIBLE). NEXT SLIDE PLEASE. THIS IS THE SELECTED ITEMS TO SHOW WHAT SCREENING USING (INAUDIBLE) MICROFLUIDICS. THE FLOWCHART YOU CAN SEE THE TESTING DID NOT TAKE VERY LONG, ALL IN ALL FOR ONE IT TAKES ABOUT FIVE HOURS. THESE ARE (INAUDIBLE) OVER THE COURSE OF THREE YEARS THAT WE HAVE BEEN SCREENING WE HAVE CHANGED OUR COLORS SEVERAL TIMES. THEY ARE HOPTORRED (INAUDIBLE) WITH INPUT FROM OUR TASK FORCE GENETIC SYMPTOMS. NEXT SLIDE. THIS HAS AFFECT OF (INAUDIBLE) DETECTS SCREENING AND YOU CAN SEE THERE IS SOME DIFFERENCE BUT NOT VERY MUCH. NEXT SLIDE, PLEASE. (INAUDIBLE) SHOWING THAT MARKED DIFFERENCE IN THE RESULT IN (INAUDIBLE) NEXT SLIDE. SO THIS SLIDE SHOWS (INAUDIBLE) ACTIVITIES TOGETHER BY AGE OF COLLECTION, IT ALL SHOW SOME DIFFERENCES THAT YOU CAN SEE CLEARLY BASED ON THIS DATA AGE RELATED WAS DEVELOPED EARLY ON TO HELP US GET REDUCING (INAUDIBLE). NEXT SLIDE, PLEASE. AT THE (INAUDIBLE) MALE AND FEMALE AND WE DIDN'T REALLY SEE MUCH DIFFERENCE, PRETTY SIMILAR (INAUDIBLE) A LITTLE HIGHER BUT IT DIDN'T WARRANT ANY DIFFERENT (INAUDIBLE). NEXT SLIDE PLEASE. SO THIS DATA APPEARS (INAUDIBLE) MULTIPLE DISORDERS TOGETHER PROVIDE INFORMATION, IMPORTANT AND USEFUL INFORMATION. THE PROFILE HELPS READ COMPROMISED SAMPLES AND FALSE POSITIVES TO REDUCE (INAUDIBLE) THIS IS NO DIFFERENT FROM WHEN WE USED TANDEM MASS. (INAUDIBLE) SO SUMMARY OF THE LYSOSOMAL STORAGE DISORDERS, POMPE FABRY GOECHER AND KRABBE WE DID -- THE PRENATAL BABIES CAN SHOW (INAUDIBLE) LEVELS WHY THE BIG SCREEN WOULD BE USEFUL. MULTI-TESTING HAS GREAT ADVANTAGES FOR ASSESSING THE RELIABILITY. I DIDN'T SHOW ANY SLIDES BUT DIRECTLY ESPECIALLY FOR THE DOUR COURIERS NO DIFFERENT WHEN WE DO THE GALT ASSAY. (INAUDIBLE) MICROFLUIDICS METHOD (INAUDIBLE) INSTALLATION AS WELL AS SCREENING METHOD (INAUDIBLE) REALLY DIDN'T TAKE THAT LONG TO RUN -- USING THAT METHOD. NEXT SLIDE PLEASE. NOW SPECIFICALLY FOR KRABBE SCREENING, (INAUDIBLE) TO BEGIN SCREENING, 2012, (INAUDIBLE) WE DIDN'T HAVE A METHOD WAITING AT THAT POINT. AFTER ALL THE TESTING WAS COMPLETED IN MISSOURI, THE SAMPLES WERE SENT TO NEW YORK VIA OVERNIGHT FEDEX AND WE THEN TESTED THE SAMPLES (INAUDIBLE) WHICH WAS TESTING ANYTHING LESS THAN 20% OF THE (INAUDIBLE) EARLY TESTING ANALYSIS WAS LESS THAN 12%. AND THE CONTINUED DNA TESTING LESS THAN 12%. THEY NOTIFIED THE -- MUTATIONS WERE FOUND. (INAUDIBLE) SAMPLES BACK AND WE KNOWED HOW YOU (INAUDIBLE) GENETIC SAMPLE. NEXT SLIDE, PLEASE. (INAUDIBLE) 266,189 SAMPLES FROM AUGUST 2012 TO JULY 2016. THEY REPORTED 42 (INAUDIBLE) AFTER THEM AND (INAUDIBLE). THEY WERE ALSO TESTED FOR -- THEY DETECTED SIX GENOTYPES OF UNKNOWN SIGNIFICANCE, THREE GENOTYPES OF UNKNOWN ONSET AND 42 WITH ONE KNOWN KRABBE MUTATION. WE HAD THREE THAT RECEIVED ANY FOLLOW-UP. IN APRIL OF 2014 WHEN WE BEGAN VALIDATION FOR IN HOUSE SCREENING FOR KRABBE USING THE METRIC PINCH UPDATE. (INAUDIBLE) ANALYSIS FOR (INAUDIBLE). THE SCREENING WAS DONE IN TANDEM IN NEW YORK. WE HAVE 34 (INAUDIBLE) AS WELL, ONE MUTATION AND EXCEPT FOR ONE CARRIER OF THE (INAUDIBLE) FOLLOW-UPS ONLY IN THAT FLAG IS ABNORMAL, IT TESTED POSITIVE SAMPLE PROVIDED BY NEW YORK AND THEY WERE ABLE TO FLAG THAT AS WELL. NEXT SLIDE, PLEASE. SO I WANT TO SHOW YOU THE EQUIPMENT USED, YOU CAN SEE THE SMALL EQUIPMENT ON THE TABLE. NEXT SLIDE, PLEASE. YOU CAN SEE THE PROCESS (INAUDIBLE) IT CAN BE DONE IN 24 HOURS. THE LAB HAS A FIXED (INAUDIBLE) FOR THE DNA AND ONLY LOOK FOR THE RELATION, WE ALSO HAVE LOWER LEVEL (INAUDIBLE) CONSENSUS ALSO WHEN WE REQUEST (INAUDIBLE) LOW LEVELS WITHOUT GOING TO BE DNA TESTING, THIS IS WHEN YOU GET A RESULT INCONCLUSIVE AND THIS IS -- THAT ALSO AND YOU CANNOT PROVIDE A RESULT BECAUSE IT'S A PREMATURE INFANT, TRANSFUSED BABY OR DETRIMENTAL -- SPECIMEN WAS COLLECTED EARLY. FINALLY (INAUDIBLE) BROAD LINE RANGE BUT NOT LOW ENOUGH TO MEET OUR DNA (INAUDIBLE). NEXT SLIDE, PLEASE. I WANTED TO SHOW YOU (INAUDIBLE) TESTING WITH NEW YORK AND REALLY INCREDIBLE HOW WELL THEY MATCH DIFFERENT METHODOLOGIES. NEXT SLIDE, PLEASE. NEXT SLIDE, PLEASE. (INAUDIBLE) CONTEXT AS I SAID EARLIER GENETIC (INAUDIBLE) PROVIDE TESTING AS WELL AS IDENTIFY (INAUDIBLE). THIS BEGAN CONTRACTING IN 2005 AFTER WE EXPANDED SCREENING TO TWO TANDEM MASS. NEXT SLIDE, PLEASE. (INAUDIBLE) DIVIDED FOR COVERAGE, WE WERE ON THE (INAUDIBLE) LEADING TO IS CENTRAL AREA AND -- WE HAVE TWO CENTERS ON THE EAST END SIDE OF THE STATE AND WE DIVIDED BY GIVING (INAUDIBLE) LAST NAMES THAT BEGIN A TO M. AND WITH LAST NAME BEGINNING (INAUDIBLE) WHILE YOU MAY ASK? NOTHING SCIENTIFIC ABOUT THAT. EVEN DISTRIBUTION USING TO DETERMINE WHERE IT STANDS IN THAT REGION. NEXT SLIDE, PLEASE. PROVIDE RESULTS ON THE (INAUDIBLE) WE JUST FOLD AND (INAUDIBLE) THE CENTER AND THEN CONTACT THE PRIMARY CARE PROVIDER TO COORDINATE THE DARE WITH THE FAMILY. AT THE CONCLUSION OF THE IMPLEMENTATION STAGE, AND (INAUDIBLE) INFANT (INAUDIBLE) ALONG WITH THE (INAUDIBLE) NEGATIVE RESULTS FOR OUR RANDOM NEWBORN SCREENING PROTOCOL. NEXT SLIDE PLEASE. I WANTED TO SHOW YOU (INAUDIBLE) (INAUDIBLE) FOLLOW IT UP. NEXT SLIDE. SO FOR (INAUDIBLE) TASK FORCE DEVELOPED GUIDELINES FOR DIAGNOSTIC TESTING AND WE REQUIRE THEM TO REPORT RESULT BACK TO THE STATE. (INAUDIBLE) FOR POMPE TIPPEDLY OCCURRED -- TYPICALLY OCCURS WITHIN 24 HOURS, GENETIC EXAMINATION AND (INAUDIBLE) PROVIDED (INAUDIBLE) NEWBORN SCREENING AND POMPE DISEASE. AND THE INFORMATION THAT RESULTED BACK TO THE STATE AT DATE OF INITIAL CLINIC VISIT, YOU CAN G ARKANSAS ACTIVITY RESULT IN THE DATE THAT WAS SELECTED. KNOB USE ANY REFERENCE RANGES, AND THE FOLLOWING TEST REQUIRED FOR POMPE. FINALLY LET US KNOW WHAT THE DIAGNOSIS IS WHAT IS THE DATE THEY CONFIRM DIAGNOSIS AND THE TREATMENT AND THE FOLLOW-UP PLAN. NEXT SLIDE, PLEASE. FORGO -- FOR THE GOECHER, (INAUDIBLE) ANALYSIS DONE HANDLED BY THE SAME AS THE NON-URGENT REFERRAL ASSESSMENT AND TESTING. PHYSICAL EXAM WITH GENETICIST, BABIES WITH TYPE 2 AND 3 GOECHER (INAUDIBLE) THE INFORMATION REPORTED BACK, THAT ACTIVITY TO CONFIRM THAT LABORATORY REFERENCE RESULT OF MUTATIONAL ANALYSIS DIAGNOSIS, DATES, TREATMENT AND FOLLOW-UP PLAN. NEXT SLIDE, PLEASE. THE CONSERVATORY TESTING FORGO CHEER IS (INAUDIBLE) FOR MALES, IF IT IS LOW DNA IS (INAUDIBLE) AND DNA AT THE SAME TIME. THE BABIES GENETIC EVALUATION AND GENETIC CLOWNLING. THE MOTHER -- COUNSELING. THE MOTHER IS THEN TESTED ALONG WITH OTHER FAMILY MEMBERS. THE INFORMATION (INAUDIBLE) INITIAL -- INITIALICALLY SICK VISIT, RESULT CAN BE COLLECTED, THE MUTATION DIAGNOSIS, DIAGNOSIS TREATMENT AND FOLLOW-UP PLAN. NEXT SLIDE, PLEASE. (INAUDIBLE) TESTING WITH THE (INAUDIBLE) DNA ANALYSIS IT'S CONSISTENT THEN MUTATIONAL TESTING WHAT I ALREADY (INAUDIBLE). NEXT SLIDE, PLEASE. CONFIRMATORY RESULTS (INAUDIBLE) ARE SEND TO A CONFIRMATORY LAB AND NEWBORN SCREENING (INAUDIBLE) ENZYME MUTATION, BABIES SEEN WITHIN 24 HOURS BY GENETIC AND NEUROLOGY, IF NEUROLOGY IS NORMAL THEN (INAUDIBLE) MRI IDENTIFIED WITH (INAUDIBLE) GENETICS NEUROLOGY IS NOT (INAUDIBLE) BASICALLY WHATEVER TESTS THEY HAVE DONE AND DIAGNOSIS TREATMENT AND FOLLOW-UP. NEXT SLIDE. (INAUDIBLE) NEWBORN SCREENING WITHIN 24 HOURS (INAUDIBLE) A LONG WITH -- BABY (INAUDIBLE) NEXT SLIDE, PLEASE. (INAUDIBLE) THROUGH THIS PRESENTATION WE HAD TWO MORE CONFIRMED CASES, AFTER THREE YEARS OF SCREENING FOR LYSOSOMAL STORAGE DISORDERS AND SCREENING FOR 276,000, WE HAVE 141 INFANTS CONFIRMED WITH ABNORMAL LYSOSOMAL STORAGE DISORDER. THE TWO ADDITIONAL CONFIRMATION, POMPE AND ONE LATE ONSET POMPE BRINGING THE POMPE TO 36 INSTEAD OF 34 GOECHER ARE THE FIVE CONFIRMED, FABRY 86 CONFIRMED, (INAUDIBLE) ONE, THREE CONFIRM CONFIRMED, KRABBE (INAUDIBLE) AND NO INFANT WILL CONFIRM WITH MULTIPLE -- NEXT SLIDE. SO I THINK WE HAVE 36 CONFIRMED POSITIVE POMPE CASES, WE NOW HAVE 8 INFANT AND 20 LATE ONSET POMPE. SIX ARE CONFIRMED CLASSICAL INFANT AND TWO (INAUDIBLE) OF WHICH (INAUDIBLE) AND WE HAVE ONE NEGATIVE BABY. STOP CO-DON, THEY HAD THIS BABY AT ERP AS WELL AS IMMUNE SEPARATION. THE BABY IS NOW TWO AND A HALF MONTHS OLD AND IT'S DOING WELL. THE REMAINING FIVE (INAUDIBLE) CASES WAS STARTED ON EARLY BI WEEKLY INFUSION, ONE WITH BP, ANY FOLLOW-UP (INAUDIBLE). TWO NON-CLASSICAL INFANTILE SIBLINGS WERE PUT ON TREATMENT BUT YOUNGER SIBLING DID NOT TOLERATE THE INFUSION SO THE INFUSIONS CONSIDER STOPPED. (INAUDIBLE) NORTH CAROLINA AND FOLLOWED UP AFTER A YEAR, THEY HAVE RECENTLY MOVED BACK TO MISSOURI AND CONTINUED CARE AT ONE OF OUR CENTERS. THE LATE ONSET HAVE BEEN FOLLOWED UP REGULARLY AND (INAUDIBLE). NEXT SLIDE. FORGO -- FOR, GOECHER (INAUDIBLE) THREE DEVELOPED AT 16 MONTHS AND WAS STARTED ON INFUSION EVERY OTHER WEEK. THE OTHER TWO (INAUDIBLE) NOT ON TREATMENT. ONE DIAGNOSIS OF GOECHER (INAUDIBLE) THROMBOCYTOPENIA AS WELL AS LONG FAMILY HISTORY (INAUDIBLE) REPEAT INFUSION. THE FIRST WAS (INAUDIBLE) SIGNIFICANT AND THEREFORE IS NOT ON TREATMENT BUT IT WAS FOLLOWED UP ANNUALLY. ALL THESE CASES ARE (INAUDIBLE). NEXT SLIDE. FOR FABRY, 86 CONFIRMED POSITIVE, 83 DIAGNOSE WITH FABRY THE OTHER THREE DIAGNOSED WITH GENOTYPE OF UNKNOWN SIGNIFICANCE (INAUDIBLE) THROMBOCYTOPENIA, AS WELL AS (INAUDIBLE) CLASSICAL FABRY HAVING THE SAME GENETIC MUTATION AS THE MOTHER WHO IS CURRENTLY FABRY PATIENT. (INAUDIBLE) ALLELE HAS BEEN ASSOCIATEDDED WITH NON-CLASSICAL FABRY DISEASE AND APPEARS COMMON IN THE GENE FOUND IN 61% OF THE CASES. STILL HAVE BEEN RAISED (INAUDIBLE) GIVEN (INAUDIBLE). NEWBORN SCREENING, NUMEROUS IDENTIFY FABRY AND WE NOW HAVE FOUR MEMBERS PUT ON TREATMENT BECAUSE OF NEWBORN SCREENING AND (INAUDIBLE). NEXT SLIDE, PLEASE. FOR (INAUDIBLE) TWO CONFIRMED CASES, THE FIRST HAD MULTIPLE ABNORMALITIES (INAUDIBLE) COMPLICATIONS FROM BONE MARROW TRANSPLANTS. THE SECOND BABY UNDERWENT (INAUDIBLE) AND IS DOING WELL. NEXT SLIDE, PLEASE. TO DATE WE HAVE NOT SEEN AN INFANT (INAUDIBLE) FOLLOWING UP ON THE UNKNOWN ONSET AND GENOTYPE OF UNKNOWN SIGNIFICANCE (INAUDIBLE) THE INCIDENCE I LISTED HERE ARE JUST FOR CONFIRMED DISORDERS, KNOWN DISEASE WITH MUTATION EXCEPT FOR FABRY, ALWAYS ONSET. POMPE AND UNKNOWN SIGNIFICANCE FROM THE TIME I THINK THE PRESENTATION FOR POMPE, 39,000 THAT I SHOW HERE, BECAUSE OF ADDITIONAL 80 THAT WAS IDENTIFIED, CONFIRMED INCIDENCE IS NOW (INAUDIBLE) IN 500. FOR FABRY ONLY 3,300. HOWEVER IF YOU COMBINE FABRY WITH GENOTYPE OF UNKNOWN SIGNIFICANCE (INAUDIBLE) 200, JUST LOOK AT MALES, IT'S ONE IN 1,800, IF YOU LOOK AT THE FEMALES, IT'S 1 IN 20,000. FOR POMPE IT'S ONE IN 34,500 FOR INFANTILE, FOR THE LATE ONSET ONE IN 15,000, IF YOU COMBINE THE INFANTILE AND LATE ONSET, IT'S ONE IN 9,800. LOOK AT GENE TYPE OF UNKNOWN SIGNIFICANCE OR YOU CAN KNOWN ONSET, IT'S ONE IN 35,000. IF YOU COMBINE IT'S ONE IN 7,600. FOR GOECHER, ONE IN 69,000, IF YOU LOOK AT THE GENOTYPE OF UNKNOWN SIGNIFICANCE, IT'S ONE IN 236,000. IF YOU COMBINE THEM, IT'S ONE IN 55,000. (INAUDIBLE) AND IF YOU COMBINE IT, IT'S ONE IN 92,000. FOR KRABBE, IT'S ZERO. AND GENOTYPE OF UNKNOWN SIGNIFICANCE OR ALONG WITH THE UNKNOWN ONSET IS ONE IN 31,000. SO IF YOU CAN SEE, (INAUDIBLE) MANY MORE CASES THAN EXPECTED FROM THE PUBLISHED (INAUDIBLE). NEXT SLIDE PLEASE. SO I WANT TO TELL YOU OUR CHALLENGES. (INAUDIBLE) OOH OVERWHELMED WITH THE PATIENT VOLUME. MORE THAN WHAT WAS EXPECTED. THEY FELT THEY NEEDED PERCENTAGE FOLLOW-UP ON THE INFANT (INAUDIBLE) TO ENSURE THE ASSESSMENT AND CLINICAL EVALUATION. WHEN PLANNING TO IMPLEMENT, THEY THOUGHT THEY NEEDED TO DEVELOP A GOOD (INAUDIBLE) AND TO EDUCATE OTHER SPECIALISTS AS WELL AND GET THEM (INAUDIBLE). NEXT SLIDE, PLEASE. (INAUDIBLE) CONFIRMATORY TESTING, LARGE (INAUDIBLE) NEWBORN SCREENINGS SPECIALLY DUE THE DIFFERENTS BETWEEN -- INTERPRET IT. GENOTYPE CORRELATION, IF YOU CAN SEE AT THE TABLE I SHOWED PREVIOUSLY, WE HAD MANY UNKNOWN SIGNIFICANCE AND GENOTYPE OF UNKNOWN ONSET (INAUDIBLE) TO DECIDE WHAT TO DO WITH THEM. VERY EXCITING NUMBER EFFICIENCY. 3% AMOPPING AFRICAN AMERICAN POPULATION AND PERFORM (INAUDIBLE) 4% ASIAN POPULATION (INAUDIBLE). NEXT SLIDE PLEASE. (INAUDIBLE) LYSOSOMAL STORAGE DISEASE RESULTED IN SCREENING PATIENTS, THERE ARE NO GUIDELINES FOLLOWING UP ASYMPTOMATIC PATIENTS. THE QUESTION IS DO WE HAVE SUFFICIENT EVIDENCE TO SAY THAT THE PATIENT IS DEFICIENCY WOULD NOT DEVELOP (INAUDIBLE). MAKING ASYMPTOMATIC CHILDREN. (INAUDIBLE) IN FRONT OF ALL THESE UNKNOWNS, CONFIRM DIAGNOSIS, PREFER (INAUDIBLE) DIAGNOSTICS. THE CLINICIAN THAT THE FAMILY WOULD BE LOST TO FOLLOW-UP IF (INAUDIBLE) LATE AT NIGHT AND GENETIC, IN THE THREE YEARS THAT WE HAVE LOST FAMILY THAT WE HAVE NOT SEEN NEWBORN SCREENING. FOR POMPE (INAUDIBLE) FAMILY MEMBERS THE MEMBERS HAVE (INAUDIBLE) THAT POPULATION. 61% HAVE (INAUDIBLE) ALLELE. WE HAVE IDENTIFIED A LOT OF ADULT -- BUT HAVE SEEN SYMPTOMATIC -- INTERESTING FOR THE CLINICIAN FOLLOWING (INAUDIBLE) TEST ASYMPTOMATIC (INAUDIBLE), ALSO WE (INAUDIBLE) GENETIC COUNSELOR -- FABRY NUMBER OF PATIENTS OF FABRY ARE PRETTY OVERWHELMING. NEXT SLIDE, PLEASE. I THINK MANY OF THE QUESTIONS HAVE BEEN HEARD WITH THE ETHICAL DILEMMA OF SCREENING BECAUSE OF IDENTIFICATION OF A LOT OF CARRIERS (INAUDIBLE) THAT MAYBE THE POSSIBILITY OF USING THE (INAUDIBLE) BUT I THINK WE CAN ADDRESS THESE WITH EDUCATION. LICENSURE AND LONG TERM CARE INSURANCE AND DISABILITY INSURANCE IS DEFINITELY BARRIER AND WE NEED FIND A WAY TO BRIDGE THIS. BUT THE ONE THING THAT IS VERY IMPORTANT TO ADDRESS IS (INAUDIBLE), NEWLY DIAGNOSED ONSET (INAUDIBLE) SUPPORT GROUP BECAUSE THE FIRST TO IDENTIFY ALL THESE INFANTS. NOT ONLY (INAUDIBLE) LATE ONSET DIAGNOSIS, PARTS HAVE NOBODY TO TALK TO SEE WHAT TO EXPECT OR WHAT NOT TO WORRY ABOUT. BE THINKING ABOUT THAT. NEXT SLIDE, PLEASE. THESE ARE SOME OF THE LESSONS LEARNED IT IS REALLY IMPORTANT THAT WE CREATE A TASK FORCE TO HELP US, WE FOLLOW GUIDELINES BUT NEED TO BE FLEXIBLE WE HAVE LEARNED QUITE A BIT, (INAUDIBLE) FOR OVER THREE YEARS. FOLLOW UP SO FAR (INAUDIBLE) SMOOTHLY. NO MAJOR HICCUPS WE HAVE SEEN TO DATE. NEXT SLIDE, PLEASE. OVERALL WE HAVE BEEN VERY PLEASED THE NETWORK (INAUDIBLE) INCIDENCE MUCH HIGHER THAN THE PUBLISHED INCIDENCES OF FALSE POSITIVES NEWBORN SCREENING TEST WE HAVE 141 CASES TO DO (INAUDIBLE) UNDETECTED CASE PRESENTED IN THE NEWBORN (INAUDIBLE) REALLY STILL (INAUDIBLE) UNKNOWN BUT I THINK WE HAVE MADE GREAT STRIDES IN UPPING THE CHILDREN IDENTIFIED IN NEWBORN TREATMENT (INAUDIBLE). NEXT SLIDE, PLEASE. (INAUDIBLE) HARD WORK ESPECIALLY THE LAST GENETIC CENTER ADVISORY COMMITTEE. I WOULD LIKE TO TAKE OPPORTUNITY TO THANK ALL OF THEM AND MOST IMPORTANTLY THE PATIENTS AND FAMILIES THAT WE HAVE IDENTIFIED THANK YOU. QUESTIONS. [APPLAUSE] >> THANK YOU FOR THAT GREAT PRESENTATION, CLEARLY YOU'RE BREAKING GROUND WITH THIS -- WITH YOUR WORK. LET'S OPEN THIS TO THE COMMITTEE. >> THANK YOU VERY MUCH FOR THAT PRESENTATION. IT'S A LITTLE SOBERING WHEN YOU'RE PART OF THE COMMITTEE AND YOU APPROVE THESE CONDITIONS THEN YOU SEE WHAT HAPPENS AND THAT THERE'S POTENTIAL HARM THAT CAN BE THERE, IT'S MOSTLY DRIP BY FALSE POSITIVES AND THE CARRIERS THE PSEUDODEFICIENCIES AND -- HAVE NO GENOTYPES CONSISTENT OF DISEASE ARE REALLY FALSE POSITIVES. I WOULD LIKE TO MAYBE SOMETHING FOR THE LAB STANDARDS COMMITTEE TO ADDRESS FIRST IS TO REALLY DEFINE WHAT A TRUE POSITIVE IS. AND SEE WHETHER WE CAN IDENTIFY MEANS TO REDUCE IT. THERE'S NEXT GENERATION SEQUENCING, ALL THIS STUFF BUT WE SEE CLEARLY THE GENOTYPES OF UNCERTAIN VARYIONS ARE NOT HELPFUL. NOW I CAN DISCLOSE THAT I HAVE PROBABLY A CONFLICT OF INTEREST THOUGH I DON'T THINK I MAKE ANY MORE MONEY BECAUSE I HAVE A LAB THAT OFFERS SECOND GEOTESTING FOR SOME OF THESE CONDITIONS. WE ARE SCREENING KENTUCKY BABIES FOR THREE LYSE SOLVAL STORAGE DISORDERS INCLUDING KRABBE DISEASE BECAUSE THAT'S ON THEIR LAWN, THEY ASKED IF WE COULD DO IT AND WE SAID YES, WE ALSO SCREEN FOR POMPE AN MPS 1. WE HAVE BEEN DOING THIS SINCE -- ACTUAL HI THE SECRETARY SENT LETTER OUT SHE ENDORSED MPS 1 AND DALD. 6 MONTHS, 25,000 BABIES. FIRST WEEK WE SCREENED WE IDENTIFIED ONE MPS 1. THE SECOND WEEK WE HAD A FALSE POSITIVE FOR MPS ONE AND WE DIDN'T HAVE A SINGLE ONE SINCE BECAUSE WE ADD AD SECOND TIER TEST FOR HEPARIN SULFATE SO WE DON'T REPORT ANYONE WITH A LOW ACTIVITY NORMAL IMMUNOGLYCANS. FOR KRABBE WE USE SYCRASINE, I'M SURPRISED WE STILL DON'T TALK ABOUT IT IN AT LEAST IN TERMS OF FOLLOW-UP NOT EVERYTHING IS CLOSED WITH RESPECT TO HOW USEFUL SYCOSI INNE MEASUREMENT IS IN FOLLOW-UP OF PATIENTS BUT SO FAR I CAN THINK ANY SYMPTOMATIC PATIENT WITH KRABBE DISEASE AT ANY AGE WILL HAVE ELEVATED SYCOSINE SO WOULD BE HELPFUL TO IDENTIFY EARLY NEWBORN CASES IN THE NEWBORN -- IT'S DIFFICULT TO OVERCOME AS WE HER HERE, AND AS WE KNOW FROM NEW YORK. BUT WE SHALL CONSIDER WHETHER LATE ONSET ARE SECONDARY TARGETS. FOR POMPE DISEASE WE'RE WORKING ON A SECONDS TIER TASK IF IT WORKS OUT WILL BE EASY FOR ANY LAB TO IMPLEMENT. SO I THINK WE SHOULD ADDRESS IT AND HELP THE STATE LABS AND PARTICULARLY, ALSO OUR FOLLOW-UP PEOPLE AND THE PATIENTS TO BETTER DEFINE WHAT THE GOAL OF THE SCREENING PROGRAMS ARE. >> THANK YOU, ADDITIONAL QUESTIONS, COMMENTS FROM THE COMMITTEE? IF NOT, NATASHA THEN CAROL GREEN. >> GENERAL TUCK ALLIANCE. THANK YOU FOR THAT PRESENTATION. I THINK IT'S A LOT OF DATA AND INFORMATION FOR US TO MULL OVER. FIRST I REALLY APPRECIATE YOU TALKING ABOUT WHERE THIS PUTS FAMILIES IN TERMS OF THIS IS A NEW EXPERIENCE IN TERMS OF BEING IDENTIFIED WITH THESE CONDITIONS. BUT THEY'RE NEWBORN SCREENING AND IT'S SOMETHING THAT BEOF COURSE ARE VERY INTERESTED IN. YOU SAID THEY DON'T NECESSARY NECESSARILY FIT INTO THE ESTABLISHED ADVOCACY OR SUPPORT GROUPS THAT ARE OUT THERE. HAVE YOU SEEN THEM DOCUMENT TOGETHER IN ANYWAY? BECAUSE WHAT KINDS OF SUPPORTS ARE THEY GIVEN OR IF YOU CAN ELABORATE ON THAT A LITTLE BIT MORE. I WILL SAY IT'S SOMETHING THAT WE'RE HOPING TO BE ABLE TO LEARN A LITTLE BIT MORE ABOUT THROUGH THE NEW CONDITIONS PROGRAM THAT WAS RECENTLY AWARDED TO APA CHALLENGE BEING A PARTNER WITH THEM ON THAT BUT BE GREAT TO HEAR WHAT YOU HEARD IN TERMS OF WHAT FAMILIES ARE DOING BECAUSE THIS IS NEW TERRITORY EVEN ON THAT ADVOCACY SUPPORT GROUP PAGE. >> NATASHA, I THINK THE CLINICIANS OUR CENTERS COULD GIVE (INAUDIBLE) PICTURES FROM WHAT I HAVE HEARD IS THAT THERE ARE ONLY A FEW BABIES THAT HAVE BEEN IDENTIFIED TO HAVE THE INFANTILE DISEASE (INAUDIBLE) NEWBORN SCREENING SO FAMILIES DON'T HAVE THEY'RE NOT NECESSARILY NOT NECESSARILY BROUGHT TOGETHER TO KNOW WHO THE OTHER FAMILY ARE AND TO BE ABLE TO TALK TO EACH OTHER. CASES OUT THERE THAT YOU HAVE POMPE SUPPORT GROUP AND A GOECHER SUPPORT GROUP, THESE ARE ALL PEOPLE IDENTIFY IN (INAUDIBLE). AND I WAS TALKING ABOUT THOSE THAT WERE IDENTIFIED WITH LATE ONSET, THOSE ARE FAMILIES THAT HAVE HEALTHY CHILDREN AND VISIBLY HEALTHY FOR MANY YEARS TO COME BUT YET I THINK THE LAST FEW CONNECTED SOME WAY TO OTHERS WHO ALSO HAVE THE (INAUDIBLE) AND THE APPREHENSION I GUESS TO COME TOGETHER. >> THANK YOU FOR THAT PRESENTATION. MY QUESTION IS ABOUT FABRY DISEASE AND DEFINITION OF CARRIERS. I WAS STRUCK BY THE FACT THAT IT DIDN'T APPEAR THAT YOU WOULD IDENTIFIED ANY FEMALE CARRIERS OF THE CONDITION THOUGH THIS IS AN EXCELLENT CONDITION. I WONDER IF YOU'RE DEFINING CARRIER DIFFERENTLY THAN WHAT I WAS EXPECTING GIVEN THIS HETEROGENEITY WITH REGARD TO PRESENTATION WITH SYMPTOMS FOR THOSE WHO DO HAPPEN TO CARRY MUTATIONS IN THE GENE. CAN YOU GIVE MORE FEEDBACK ON THAT? >> WITH THE KNOWLEDGE THAT I HAVE BASICALLY THE CENTERS ARE SENDING (INAUDIBLE) FABRY DISEASE. ANDREA, I KNOW YOU'RE ON THE PHONE HERE, DO YOU WANT TO SAY SOMETHING? >> SHE'S COMING TO THE MICROPHONE. THERE IS NO MICROPHONE. (OFF MIC) >> I HAVE TO START THIS OFF BY SAYING I UNFORTUNATELY AM NO LONGER AT CHILDREN'S MERCY OF GENETIC COUNSELOR AND I WORK FOR SHIRE NOW, GOING BACK AND SPEAKING WITH -- ABOUT HOW THE CENTERS IN MISSOURI WHERE IT'S DUE TO FOLLOW-UP FOR FABRY DISEASE, WE DIDN'T USE THE TERMINOLOGY CARRIER FOR FABRY DISEASE, THEY WERE HETEROZYGOTE OR FABRY MALE, THE FEMALES IDENTIFIED AS HETEROZYGOTE OR FABRY DISEASE WERE CLASSIFIED AS FABRY DISEASE, KNOWING WITH X LINKED DISORDERS WHEN FEMALES ARE CONCERNED WE WON'T PICK UP EVERY FEMALE THROUGH A HETEROZYGOTE SCREEN SO THERE'S A FAIR NUMBER OF GIRLS OUT THERE THAT ARE CARRIERS FABRY DISEASE WITH NORMAL ENZYME FUNCTION SO THEREFORE NOT REFERRED THROUGH NEWBORN SCREENING TO BE IDENTIFIED. >> CAROL GREEN SIMD. I HAD TWO QUESTIONS FOR FOLLOW-UP BUT TO CONTINUE ON THAT THEME, THIS MAYBE COMING BACK TO WHAT DR. MATERN SAID A MOMENT AGO NEEDING TO HAVE A COMMON LANGUAGE AND IT WOULD MAKE SENSE THAT IF SOMEBODY'S ENZYME ACTIVITY WAS LOW ENOUGH THAT YOU MIGHT CALL IT FABRY DISEASE RECOGNIZING YOU DIDN'T PICK UP OTHER HETEROZYGOTES BUT BECAUSE YOUR BLOOD LEVEL IS LOW DOESN'T MEAN YOU'LL HAVE LOW ENOUGH LEVEL TO GET SYMPTOMATIC. SO I THINK THAT BRINGS BACK TO WHAT DR. MATERN SAID ABOUT NEEDING A COMMON LANGUAGE. SO AGAIN I WANT TO ADD MY THANKS FOR A GREAT PRESENTATION. I HAVE TWO QUESTIONS THAT HAVE TO DO WITH THE FOLLOW-UP EARLIER YOU MENTIONED SOME ANECDOTES THAT THE FAMILIES WERE GRATEFUL TO HAVE THE INFORMATION I THINK YOU WERE REFERRING TO THOSE WHO WERE SYMPTOMATIC AND LATER MENTIONED THAT MANY OF THE FAMILIES WHO ARE THE TERM OF ART NOW SEEMS TO BE PATIENTS IN WAIT ING, THAT ARE ANXIOUS. ONE QUESTION IS THERE ANY FORMAL EVALUATION OF THE PSYCHOSOCIAL IMPACT ON THOSE FAMILIES. SO THINKING ABOUT FINDING THEM SUPPORT IS GREAT. I WONDER IF THERE'S FORMAL E VALUE WEIGHS IMPACT ON FORMAL FAMILIES. SECOND QUESTION, SPEAKING AS A CLINICIAN AND GET POSITIVE NEWBORN SCREEN AND WHAT TESTING NEEDS TO BE DONE AND THE INSURANCE DOESN'T COVER IT, WE COMPROMISE A LOT, CAN'T GET A SAMPLE TO THE RIGHT LABORATORY. DO YOU HAVE INFORMATION ABOUT THE EXPERIENCE OF THE CLINICAL CENTERS ACTUALLY GETTING THIS TESTING DONE? >> YOUR FIRST QUESTION ABOUT THE EVALUATION WE HAVE THE FORMAL EVALUATION BUT THE CENTERS TOLD US THAT FAMILIES THAT HAVE INFANTS WHO HAVE BEEN CONFIRMED WITH A DISEASE (INAUDIBLE) BUT THOSE WITH CLINICAL ONSET ARE ALSO GRATEFUL BECAUSE ONCE THE EDUCATION IS GIVEN TO THEM AND THE UNDERSTAND -- THEY UNDERSTAND WHAT THE DISEASE IS, THEY TRULY ARE GRATEFUL THAT THEY KNOW AND DON'T HAVE TO WORRY DOWN THE ROAD AT SOME POINT THE CHILD GETS SICK AND HAVE TO GO FROM DOCTOR TO DOCTOR TO FIND OUT WHAT THAT CHILD (INAUDIBLE) THEY ARE GRATEFUL AND GOING TO HAVE SOME BALANCE THAT REALLY ARE UPSET. YOUR SECOND QUESTION, (INAUDIBLE) IMI FORGOT WHAT THE QUESTION WAS. CAN YOU REPEAT THE SECOND QUESTION, PLEASE? >> WHAT TEASE CENTERS EXPERIENCE ACTUALLY GETTING INSURANCE APPROVALS FOR -- TO SEE THE NEUROLOGIST TO HAVE THE SPINAL TAP, TO HAVE THE DNA TESTING DONE, TO HAVE ENZYME ASSAYS DONE? >> FROM WHAT I KNOW WE HAVE NO PROBLEMS WITH MEDICAID BECAUSE AS LONG AS NEWBORN -- THERE'S A NEWBORN SCREENING IS DONE FOR THAT PARTICULAR DISORDERS THEY WILL APPROVE ALL THE TESTS. TRICARE HAS HAD SOME PROBLEMS WITH APPROVING OR PAYING FOR CERTAIN TESTS, WE AS A STATE HAVE GIVEN EACH CENTERS SOME SEED MONEY NOT REALLY MUCH TO HELP IF INSURANCE DOESN'T COVER IT AND NEW (INAUDIBLE) WHAT INSURANCE DOESN'T COVER, TESTING SO THE CHILDREN'S HOSPITAL FIND THAT DIFFICULT TO GET TESTING DONE SO WE PROVIDED FUNDING FOR THAT. I WANTED TO ALSO SAY THAT ALL OUR CENTERS ARE SENDING MEMBERS FOR (INAUDIBLE) (INAUDIBLE) IN THE BEGINNING BECAUSE AT THAT TIME IT WAS A RESEARCH PROJECT BUT NOW ALL ARE SENDING (INAUDIBLE). NOT SOMETHING INITIALLY CREATED IN OUR FORM TO REPORT BACK. >> THANK YOU VERY MUCH FOR YOUR PRESENTATION AND YOUR COMMENTS. WE APPRECIATE IT. WE'RE NOW GOING TO GO ON TO THE NEXT PRESENTATION. THIS IS BY JENNIFER KWON. DR. KWON IS ALSO ON THE PHONE. SHE'S ASSOCIATE PROFESSOR OF NEUROLOGY, PEDIATRIC, PATHOLOGY AND LABORATORY MEDICINE AT THE GOLISANO CHILDREN'S HOSPITAL, UNIVERSITY OF ROCHESTER. DR. KWON IS A CHILD NEUROLOGIST WITH STRONG INTEREST IN IMPROVING CLINICAL OUTCOMES IN CHILDREN DIAGNOSED WITH RARE DISORDERS BY NEWBORN SCREENING, A MEMBER OF THE EVIDENCE REVIEW COMMITTEE AND THE REGISTRY COMMITTEE IN THE AMERICAN ACADEMY OF NEUROLOGY. SHE'S GOING TO TALK TO US ABOUT LONG TERM FOLLOW-UP FOR POMPE DISEASE. DR. KWON. YOUR SLIDES. >> THANK YOU. I HOPE YOU CAN HEAR ME. I'M IN A CANADIAN HOLIDAY TOWN AND IN ORDER TO ENSURE STREAMING OF HIGH DEFINITION VIDEO AND CLEAR SIGNAL I FOUND AN IDEAL LOCATION EXCEPT IT'S NEAR A BAR SO IF YOU HEAR SOME BACKGROUND NOISE THAT'S WHAT THAT IS. >> SURE. WE CAN HEAR YOU. GO AHEAD. >> OKAY. >> SO THE NEXT SLIDE. IN TERMS OF DISCLOSURES, I ANNOTATE CONSULTANTS FOR GEM SO I'M AND I'M SITE PI FOR THE GENZYME REGISTRY, I'M NOT A POMPE DISEASE CLINICAL EXPERT. I'M A CLINICIAN INTERESTED IN IMPROVING LONG TERM CLINICAL OUTCOMES. AND THERE IS NO (INAUDIBLE) NEWBORN SCREENING. MY -- I'M NOT QUITE SURE WHY THE CLINICAL EXPERT IN POMPE DISEASE ARE AREN'T GIVING THIS TALK INSTEAD OF ME BUT I HAVE A FEW SLIDES, NEXT SLIDE, WITH AMY YOUR AND MIKE WATSON OF NSTRN SLASH ACMG AS WELL AS (INAUDIBLE) AND I REALLY THANK THEM FOR THEIR ASSISTANCE WITH THESE SLIDES. I AM SURE THEY WILL APPRECIATE IT, ANY OF THESE VIEW IS EXPRESS ARE MY OWN. YOU SHOULD BE AWARE THAT THOSE CLINICIANS WHO ARE FOLLOWING CHILDREN IDENTIFIED BY POMPE DISEASE NEWBORN SCREENING CONVERTS REGULARLY WITH LSD EXPERTS AND POMPE DISEASE EXPERTS. NEXT SLIDE. SO THE BACKGROUND OF LONG TERM FOLLOW-UP POMPE DISEASE, IN FACT EVEN THOUGH NEWBORN SCREENING WAS ADDED TO THE -- TO IMPROVE OUTCOMES OF THOSE BE INFANT AND CHILD POMPE DISEASE BY ALLOWING EARLY TREATMENT, WE HAVE ALWAYS KNOWN THAT NEWBORN SCREENING IS LIKELY TO JUST -- TO IDENTIFY FAR MORE INCIDENCE WITH LATE ONSET POMPE DISEASE. ANY TIME FROM EARLY CHILDHOOD TO ADULTHOOD. FROM BASED ON THAT EVIDENCE REVIEW, I DIDN'T PUT ON THE SLIDE BUT THE ONE CONDUCTED FOR THE ADVISORY COMMITTEE TWO YEARS AGO FOR POMPE DISEASE WE PREDICTED ANNUALLY WE IDENTIFY 40 CASES OF INFANTILE POMPE DISEASE IN THE U.S. AND ABOUT 90 PLUS CASES OF LATE ONSET DISEASE. SO WHAT I -- NEXT SLIDE, PLEASE. SO THE PREPARATION FOR HOW BEST TO FOLLOW THESE POMPE DISEASE PATIENTS, WE LOOK AT THE LANDSCAPE OF INFORMATION THAT WE ALREADY HAVE (INAUDIBLE) AND THIS IS A LISTING PROVIDED BY THE (INAUDIBLE) THAT JUST HIGHLIGHTS NICHD AND NDSPRS FOR THE SCREENING OF POMPE DISEASE NEW YORK, THERE'S ALSO CARRIERS RESOURCES TO HAVE MONTHLY CALLS OF THE PILOT CENTER AS WELL AS STATE NEWBORN SCREENING LABS IN STATES INTERESTED IN IMPLEMENTING LSD SCREENING. AND OTHER STATES JOINED ON TO OUR SPEAKING OF BECOMING INTERESTED LATER. RECENTLY WE DID CLINICIAN FOCUSED CALLS TO DEAL WITH LONG TERM FOLLOW-UP ISSUES. IN ADDITION, IT SPONSORS A COLLABORATORY AN ANALYTICAL TOOL THROUGH ALL THOSE PROJECTS AS WELL AS LONG TERM FOLLOW-UP TOOL AND DATA SETS WHICH IS REALLY SUPPOSED TO BE THE HEART OF LONG TERM FOLLOW-UP REGISTRY DATA DESIGN. NEXT SLIDE, PLEASE. THE FOLLOWING SLIDES I'LL GO THROUGH RELATIVELY QUICKLY IN THE INTEREST OF TIME BUT THIS IS TO SHOW THE WEALTH OF GUIDELINES THAT WERE AVAILABLE BEFORE NEWBORN SCREENING BEGAN. FIRST WE HAVE THE ACMG ACT GUIDELINES SHEET WHICH REALLY GAVE EARLY INFORMATION ABOUT HOW TO DIAGNOSE THE CONDITION ONE REFERRAL IS MADE AS WELL AS EMERGENCY MANAGEMENT GUIDELINES. SECTION NEXT SLIDE, PLEASE. THE NEXT GUIDELINE WAS AN ACMG PRACTICE GUIDELINE, SPECIFICALLY FOR POMPE DISEASE PUBLISHED IN MAY OF 2006. THERE IS A NEWBORN SCREENING FOLLOW-UP NOT PUBLISHED YET. THEN THE MAY 2006 PUBLISHED GENETICS AND MEDICINE. FOLLOWING ACMG STANDARDS GUIDELINE MORE BROADLY FOR LYSOSOMAL DISEASE IN GENERAL, INCLUDING PRESYMPTOMATIC MANAGEMENT OF A VARIETY OF LYSOSOMAL DISEASE INCLUDING POMPE DISEASE. AND THAT CAME OUT IN GENETICS MEDICINE IN MAY OF 2011. THE FINAL GUIDELINE WHICH WE'RE NOT AN ACMG PRODUCT BUT A PRODUCT OF NEUROLOGISTS, INTERESTED IN ESTABLISHING SOME SORT OF CARE GUIDELINE SPECIFICALLY FOR LATE ONSET POMPE DISEASE PATIENTS. AND THIS IS LOCATED IN MARCH OF 2012. RESEARCH SHOWING Y'ALL THESE, NEXT SLIDE PLEASE. I'M SORRY WE'LL GO THROUGH THIS SLIDE LYSOSOMAL AND THEN THE CONSENSUS GUIDELINES IN MUSCLE AND THEN THE NEXT SLIDE NUMBER TEN IS MY WAY OF SAYING GUIDELINES ARE HELPFUL AND USEFUL TO GIVE YOU THE GENERAL GIST OF WHAT WE'RE TRYING TO A VOID OR TRY TO PREVENT IN DISEASE. THEY ARE NOT NECESSARILY BELL SUIT FOR ON GOING CLINICAL ACTS THAT TAKE PLACE BETWEEN DOCTOR AN PEOPLE WHO ARE IDENTIFIED BEING AT RISK FOR POMPE DISEASE. TO THAT END WE HAVE RECOGNIZED PROVIDERS SEEING POMPE DISEASE, ESPECIALLY THOSE THOUGHT TO BE LAID OFF, SUPPOSED TO HAVE THEIR PRESENTATION LATER IN LIFE BUT WE DON'T KNOW NECESSARILY HOW MUCH LATER IN LIFE. THERE HAVE BEEN A NUMBER OF DISCUSSIONS AMONG THOSE PROVIDERS. SO AS WE SAID BEFORE IN ESTRN RECENTLY STARTED SPONSORING PROVIDER CALLS. I THINK I MAYBE -- UNLESS THERE IS A TIME LAG, I MAYBE A SLIDE AHEAD. NEXT SLIDE, CLINICAL FOLLOW-UP INITIATIVE. BEGAN RECENTLY IN JUNE. IN STATES SUCH AS MISSOURI THEY HAVE PROVIDER BASED CALLS, REGULARLY TO TALK ABOUT ISSUES WITH THEIR WHOLE LYSOSOMAL PROGRAM AND REALLY SO WE LOOKED AT THE POMPE DISEASE GUIDELINES FOR EXAMPLE PRODUCED BY MISSOURI. IN ADDITION, THERE ARE GENZYME RESPONDSORRED WORKSHOPS THAT AS YOU ARE AWARE THE TREATMENT THERAPY, THE ERT PRODUCED BY GENZYME HAVE A NUMBER OF HELPFUL WORKSHOPS FORKEDLY ANYTHINGS FOLLOWING PRE-SYMPTOMATIC PATIENTS FOR POMPE DISEASE. AS YOU CAN IMAGINE DISCUSSION TEND TO BE EXPERTS AND STANDARDIZE APPROACHES ARE REALLY NOT PRESENT YET BUT THEY ALL INVOLVE. SO NEXT SLIDE PLEASE. SO THIS TABLE IS REALLY MEANT TO GIVE YOU A SENSE OF THE NEW YORK STATE POMPE DISEASE GUIDELINES, AND JUST A QUICK AND DIRTY FORM, BASICALLY ONLY MANDATED TESTING FOR CLINICAL DIAGNOSTICS AT DIAGNOSIS OR TIME OF REFERRAL. FOR LATE ONSET PATIENTS, IN OTHER WORDS PATIENTS WHO APPEAR TO BE ASYMPTOMATIC, FOLLOW-UP REALLY DEPENDS ON THE EYE OF THE (INAUDIBLE) THERE ARE A LOT OF THINGS (INAUDIBLE) CLINICALLY INDICATED. AND ANY ONE OF THOSE FOLLOW-UPS HAPPEN TO BE ABNORMAL THAT'S TRIGGER FOR CONSIDERING INITIATING THE RT. SO IN NEW YORK THIS IS DATA THAT (INAUDIBLE) ABOUT 400,000 INSTANCES. OF THOSE WE THINK WE IDENTIFY TWO, POSSIBLY THREE INFANTILE CASES. AND POSSIBLY 28 LATE ONSET CASES IT'S NOT MEANT TO BE A STATISTIC TO CARRY A WISH, (INAUDIBLE) THE ISSUE OF LATE ONSET DISEASE FOLLOW-UP. NEXT SLIDE, PLEASE. SO THE QUESTION TO ASK IS HOW DO WE ACTUALLY FOLLOW LATE ONSET PATIENT? DO WE ASK PEOPLE LIKE TO COME WHEN WORRIED ABOUT THEIR CHILD, MEET REGULARLY AND DECIDE IF WE'RE WORDED ABOUT THE CHILD OR THERE'S BEEN A LITTLE (INAUDIBLE) HAVING A MORE STANDARDIZED CLEARLY DEFINED PROTOCOL FOR FOLLOW-UP. AND THE QUESTIONS THAT ARISE ARE WHAT MIGHT BE OPTIMAL SURVEILLANCE AND TESTING AND WHAT POINT SHOULD WE THINK PULLING THE TRIGGER AND STARTING A PATIENT ON PATIENT THERAPY KNOWING THAT WHEN A CHILD IS STARTED ON ENZYME REPLACEMENT THERAPY, WHICH IS AS THE OTHER BEAK INFUSION, IT IS VERY LIKELY THIS TREATMENT WILL BE CONTINUED FOR THEIR LIFETIME. TO THAT END, THERE ARE POMPE DISEASE NEWBORN SCREENING REGISTRY EFFORTS UNDERWAY TO AT LEAST (INAUDIBLE) CLINICAL PRACTICE WE CAN HOPEFULLY LOCK BACK AND EVALUATE. THESE TAKE PLACE BETWEEN (INAUDIBLE) AND ALSO GENZYME HAS BEEN COLLECTING NEWBORN SCREENING DATA. NEXT SLIDE. >> WE HAVE LOST THE SOUND. >> HELLO? >> WE CAN HEAR YOU NOW. WE LOST YOU A LITTLE WHILE. >> I'M SORRY. SO I JUST ACTUALLY ONLY HAVE -- HAD A FEW SLIDES. SO WE WERE TALK ABOUT THE NEW YORK STAY POMPE DISEASE OUTLINE. AND DID YOU HEAR ANY TALK ABOUT THE NUMBERS OF INFANTILE LATE ONSET PATIENTS. >> WE DID. >> OKAY. SO THEN THE NEXT SLIDE AFTER THAT, -- >> WE'RE ON THE SLIDE RECENT QUESTIONS RAISED ABOUT LONG TERM FOLLOW-UP. >> OKAY. I'M SORRY. I'M LOOKING AT THE VIDEO, THERE IS A LAG SO THANK YOU VERY MUCH. SO THE RECENT (INAUDIBLE) QUESTIONS THAT HAVE COME UP ON -- ABOUT LONG TERM FOLLOW-UP AND CLINICIANS KEEP ASKING IS FIRST HOW FREQUENTLY SHOULD WE BE FOLLOWING THESE (INAUDIBLE) AND THEN WHEN ISOLATED, HOW SHOULD THEY BE ADDRESSED? MANY OF US ARE SEEING INFANT WHOSE HAVE (INAUDIBLE). MANY OF US ARE SEEING INFANTS AND YOUNG CHILDREN OR SIBLINGS OF INFANTS IDENTIFIED WITH LATE ONSET POMPE DISEASE WHO HAVE FATIGUE, WEAKNESS, HEADACHE OR PAIN. AND THERE ARE ALSO INFANTS WHO HAVE MORE INVOLVED FOLLOW-UP IN THEY HAVE PERHAPS MINOR ABNORMALITIES, NOT ABNORMALITIES THAT (INAUDIBLE) BUT POSSIBLY SOME THINGS THAT MAY SUGGEST INVOLVEMENT. THE OTHER QUESTION THAT ARYES, AS YOU KNOW IN CERTAIN POPULATIONS -- AS I RISES, IN CERTAIN THERE'S (INAUDIBLE) WHICH IS THOUGHT TO LEAD TO MORE BENIGN PHENOTYPE. SOME OF US HAVE SEEN PATIENTS HOMOZYGOUS FOR THIS MUTATION WE WANT TO FOLLOW LESS FREQUENTLY BECAUSE THE DATA SUGGEST THESE PATIENTS SHOULD DO BETTER. SO THAT ANSWERS ONE OF THE QUESTIONS THAT WAS ALSO RAISED TO GIVE YOU AN EXAMPLE. SO WE ARE OUR LAST PROVIDER CALL, NEXT SLIDE, WHICH IS ENTITLED WHEN PUBLIC HEALTH MEETS RARE DISEASE CARE, OUR LAST CALL IT WAS SUGGESTED THAT HE FOR POMPE DISEASE NEWBORN SCREENING AND CLINICAL FOLLOW-UP REGISTRY, WE CONSIDER WHAT THE FOUNDATION REGISTRY DOES AND HOW THEY WORK. AND SO -- AND I HAVE LONG BEEN A PROPONENT OF USING THIS DISEASE FOUNDATION AND REGISTRY, THE MODEL FOR IMPROVING CLINICAL OUTCOMES OF RARE DISEASE. AND THEN WE (INAUDIBLE) SUCCESSES WE THINK ARE DUE TO THE FACT THAT THEY HAVE A SYSTEM OF ONGOING EVALUATION, CLINICAL OUTCOMES, USING SPECIALIZED NATIONAL REGISTRY. THAT THE REGISTRY THAT CONDUCTED BY ADVOCACY ORGANIZATION, WHOSE MEMBERS ARE REALLY COMMITTED TO CLINICAL QUALITY IMPROVEMENT OVER THE LIFETIME OF A PATIENT. SO THESE AREN'T RESEARCHERS NECESSARILY, THESE PEOPLE WHO WANT TO MAKE A LOT OF PATIENT WITH CS BETTER OVER THE COURSE OF THEIR LIVES AND OF COURSE, IN DOING SO, THEY'RE RAISING RESEARCH QUESTIONS, THEY'RE GENERATING (INAUDIBLE) FOR CLINICAL TRIALS. BUT THE ONE THING THAT WE OFTEN FORGET ABOUT THE FOUNDATION AND REGISTRY IS THAT THEIR REGISTRY HAS ACCESS TO SOURCES OF FUNDING THAT ARE UNHEARD OF. IN THE REST OF RARE DISEASE CARE. IT ALMOST MAKES IT A NON-STARTER, THE QUALITY -- I MEAN THE QUANTITY OF MONEY THAT'S AVAILABLE FOR THIS ONE DISEASE SPECIFIC REGISTRY. AND THAT'S WHAT MAKES IT SO DIFFICULT TO REPLICATE THIS, FOR OTHER RARE DISEASES. EVEN RARE DISEASE LIKE POMPE DISEASE WE SHOULD HAVE AS MANY AS YOU KNOW 30 FINAL SLIDE, OUR LONG TERM FOLLOW-UP EFFORTS ARE WANTED TO DEVELOP A REGISTRY BECAUSE WE UNDERSTAND ALREADY THAT WE HAVE NO IDEA WHAT WE'RE DOING WHEN IT COMES TO FOLLOWING PATIENTS WITH POMPE DISEASE, I SHOULD SAY WE DO KNOW THE CONSEQUENCES ARE TRYING TO EXTEND -- TRYING TO PREVENT. WE DO KNOW WE DON'T WANT PEOPLE TO BE WEAKENED AND HAVE END STAGE MUSCLE DAMAGE. BUT WE'RE NOT REALLY SURE OF THAT TIME THE THIRD PIECE THAT PRESENTS THAT. SO EVEN THOUGH WE HAVE THESE GUIDELINES ABOUT NOT STARTING ERT TOO LATE, WE ARE NOT NECESSARILY SURE WHEN THE OPTIMAL TIMING IS. WE KNOW ABOUT THE GENOTYPE PHENOTYPE CORRELATIONS, THE GA 8 GENE, THERE ARE RESOURCES IN (INAUDIBLE) LIKE CLIMGEM AND OTHER RESOURCES TO DEVELOP BETTER BIOMARKERS. FOR ME, JUST AT A PRACTICAL LEVEL, I FIND THE MDS SPONSOR PROVIDER CALLS, RESOURCE FOR CLINICIAN -- AIR THEIR IMMEDIATE CONCERNS BUT HOPEFULLY WE'LL FIND THESE QUESTIONS LEAD TO MORE TARGETED REGISTRY WORK. AND THAT'S (INAUDIBLE) THANK YOU. [APPLAUSE] >> JENNIFER, THANK YOU VERY MUCH. WE APPRECIATE YOUR PRESENTATION. CAN YOU GIVE US AN IDEA OF HOW MANY PROVIDERS ARE ON THE NEWBORN SCREENING TRANSLATIONAL RESEARCH NET WORK CALLS? >> SO I THINK THE FIRST CALL THERE WAS A PRETTY IMPRESSIVE ATTENDANCE. WE'RE GOING TO MAKE THE CALLS QUARTERLY. I WOULD THINK THAT OVERALL, THE CALLS ARE NOT JUST SCHOOL PROVIDERS, THEY'RE OTHER NEWBORN SCREEN STAKEHOLDERS SO IT'S HARD FOR ME TO GIVE YOU A NUMBER OF THE PROVIDERS BUT I DO THINK THAT WE ALL MANAGE TO STAY IN TOUCH WITH EACH OTHER ESPECIALLY AS THESE COMMON QUESTIONS ARISE. >> THANK YOU. ANY QUESTIONS OR COMMENTS STARTING WITH THE COMMITTEE? JEFF. >> I THINK WE HEARD -- THESE JEFF ROC -- ROSCO. CERTAIN ETHICS ISSUES COME UP WITH NEWBORN SCREENING THAT WE CAN ANTICIPATE. AARON GOALBERG AND I WITH NBSTRN GROUP ARE PUTTING TOGETHER A PAPER, SORT OF LAYS OUT COMMON ETHICS ISSUES THAT COME UP, FOR PROBABLY ANY CANDIDATE CONDITION, PROBABLY SHOULD BE THOUGHT ABOUT BEFORE WE GET TOO FAR. SO WE DON'T END UP SCREENING FOR CONDITIONS SAYING OH, WE FOUND THIS, NOW WHAT DO WE DO? IN TRYING TO THINK WHAT THE APPROACH MIGHT BE BEFORE WE GET TO THAT STAGE. HOPEFULLY WE'LL SHARE THAT WITH YOU AT SUBSEQUENT MEETINGS. >> THANK YOU. OTHER QUESTIONS, COMMENTS FOR THE COMMITTEE? JOAN. >> THIS IS JOAN SCOTT. THANK YOU FOR THAT REALLY GOOD OVERVIEW. JENNIFER, DO YOU HAPPEN TO KNOW IF THE CF REGISTRY IS DONE ONLY UNDER INFORMED CONSENT? IS IT -- IT'S -- IS IT A PATIENT ENTERED REGISTRY AND DATA OR IS IT CLINICIAN ENTERED? >> IT IS CLINICIAN ENTERED REGISTRY PROGRAM, ALL PATIENTS WHO DATA ARE ENTERED, THEY DO CONSENT. >> AND DO WE HAVE A SENSE OF WHERE THERE SCREENING FOR POMPE WHAT CLINICIANS DO TO ENCOURAGE BECAUSE THE NSTRN IS DONE UNDER INFORMED CONSENT TO COLLECT DATA AND ENTER FOLLOW-UP DATA INTO THERE, IS THAT NOT CORRECT? I'M LOOKING AT MIKE WHO IS NODDING HIS HEAD. >> THE THAT'S CORRECT. >> DO YOU HAVE A SENSE THAT CLINICIANS WHO ARE NOW SEEING INDIVIDUALS WITH POMPE ARE ASKING PATIENTS FOR INFORMED CONSENT AROUND BEING PART OF THAT LONG TERM FOLLOW-UP DATABASE? >> I WOULD SAY YES, THEY ARE. FIRST I SHOULD MAKE IT CLEAR THAT RIGHT NOW, THERE ARE PLANS TO DEVELOP A POMPE DISEASE NEWBORN SCREENING LONG TERM FOLLOW-UP REGISTRY WITH NBSTRN. BUT IN ORDER TO MAKE THOSE KINDS OF REALITIES, BEWILL HAVE TO FIGURE OUT SOME WAY OF INSTITUTING SOME CONSENT PROCEDURE. BUT EVEN WITHOUT THAT, MANY CENTERS ARE ALREADY GENZYME REGISTRY SITES ARE ALREADY ENTERING NEWBORN SCREENING DATA INTO THE GENZYME PATIENT REGISTRIES AND AGAIN, THEY CAN ONLY DO THAT WITH PATIENTS. SO THERE IS STILL NO WAY AROUND THE FACT THAT THIS ACTIVITY IS AN ACTIVITY (INAUDIBLE). >> YEAH. I WASN'T QUESTIONING THAT IT SHOULDN'T BE I JUST WAS TRYING TO GET A SENSE ABOUT WHETHER OR NOT THE FAMILIES WHO ARE BEING IDENTIFIED THROUGH NEW IMPORTANT SCREENING ARE BEING TOLD OF THESE REGISTRIES AND ENCOURAGING AND PARTICIPATION, BECAUSE IT'S THE ONLY WAY TO SYSTEMATICALLY COLLECT THE DATA WE NEED. >> I THINK THAT BECAUSE -- I'LL SPEAK FOR MYSELF. SO I KNOW ABOUT THESE REGISTRY EFFORTS THAT ARE UNDERWAY BUT I HAVE TWO LATE ONSET FOLLOW-UP PATIENTS THAT I FOLLOW AND I HAVEN'T REALLY PRESENTED THE REGISTRY AS AN OPTION TO THEM YET BECAUSE I THINK THAT THIS IS STILL EARLY DAYS. IN TERMS OF THE REGISTRY PROCESS. WHEN I GO WITH THE WORK FLOW WHEN IT'S A LITTLE CLEARER AND THE STRUCTURE OF REGISTRY OVERSIGHT IS A LITTLE CLEARER, I THINK ALL CLINICIANS ACCUMULATE -- HAPPY TO ENTER INTO A REGISTRY. >> ALL RIGHT. THANK YOU. JENNIFER, THANK YOU VERY MUCH FOR YOUR PRESENTATION. I THINK THERE ARE NO OTHER QUESTIONS OR COMMENTS AT THIS POINT IN TIME SO PLEASE GO BACK AND ENJOE I THE REST OF YOUR HOLIDAY. -- ENJOY THE REST OF YOUR HOLIDAY AND YOUR LOCATION. THANK YOU. >> THANK YOU. >> SO THAT WILL CONCLUDE THE MORNING SESSION. WE HAVE UNTIL ONE O'CLOCK TO RETURN FOLLOWING LUNCH. I WOULD LIKE COMMITTEE MEMBERS BEFORE YOU HEAD FOR LUNCH TO MEET AT THE LECTERN. WE'RE GOING TO TAKE A GROUP PHOTO. SEE YOU BACK PROMPTLY AT 1:00. LET'S GO AHEAD AND DO THE ROLL CALL. WE DO HAVE SOME EARLY LEVERS AND SOME LATE RETURNERS. SO LET'S SEE WHO'S HERE. DON BAILEY, HERE. [INAUDIBLE] KELLIE KELM, HERE. DETER -- HERE. [INAUDIBLE] ANN MARIE -- HERE. [INAUDIBLE] DEBBIE -- HERE. JOSEPH BIGGIO ON THE PHONE. SUSAN TANKSLY -- >> HERE. >> GREAT. ADAM CANNIS, HERE. NATASHA BANHOME -- [INAUDIBLE] IS THAT RIGHT? >> YES. >> KATE WALSH, HERE. CAROL GREEN, HERE. >> ALL RIGHT. THANK YOU ALL. SO THIS AFTERNOON SESSION, WE HAVE FOUR REPORTS FROM WORK GROUPS. THE FIRST IS THE COST ANALYSIS WORK GROUP UPDATE. THIS WILL BE PRESENTED BY ALEX KEMPER. ALEX IS THE LEADER OF THE CONDITION REVIEW WORK GROUP. HE IS AT DUKE CLINICAL RESEARCH INSTITUTE AND DEPARTMENT OF PEDIATRICS. ALEX? >> THANK YOU VERY MUCH, DR. BOCCHINI. I KNOW RIGHT AFTER LUNCH WHAT EVERYBODY WANTS TO HEAR IS ABOUT COST ASSESSMENT METHODS SO I'LL DO MY BEST TO GO THROUGH THIS. REALLY THE KEY THINGS THAT I HOPE YOU GET OUT OF THIS PRESENTATION IS BOTH WHAT WE CAN DO AND WHAT WE CANNOT DO, BECAUSE I THINK THAT BOTH OF THOSE THINGS ARE EQUALLY IMPORTANT. SO THIS IS A LIST OF THE MEMBERSHIP OF THE WORK GROUP, I WON'T READ THROUGH ALL THE NAMES BUT I WANT TO THANK EVERYONE HERE AND ALSO POINT OUT THAT SEVERAL MEMBERS OF OUR WORK GROUP ARE NOW SITTING AT THE BIG TABLE, SO IN THE SENSE WE'RE KIND OF THE PROVING GROUND, THE WAY I LIKE TO THINK OF IT. SO OUR CHARGE WAS TO CONSIDER METHODS TO ASSESS THE COST OF NEWBORN SCREENING EXPANSION, AND I THINK AS MOST PEOPLE IN THIS ROOM KNOW, THIS IS PART OF THE NEWBORN SCREENING SAVES LIVES LEGISLATION, SO WE'RE REALLY REQUIRED TO DO THIS. SO JUST TO RECAP WHERE WE ARE WITH THIS WORK, IF YOU REMEMBER LAST TIME I TALKED ABOUT DOING A PRE-TEST TO ASSESS THE FEASIBILITY AND COST ASSESSMENT METHODS, AND WE WERE LOOKING AT TWO TARGET CONDITIONS, MPS I AND POMPE DISEASE. BOTH OF THESE TESTS CAN BE DONE ON MULTIPLE PLATFORMS AND ALSO WITH OTHER SCREENING TEST, BUT AS YOU'LL SEE I'M TRYING TO REALLY SIMPLIFY SOME OF THE DETAILS IN THE ANALYSES WE'RE DOING, SO WE'RE NOT ESTIMATING COSTS FOR EACH POSSIBLE SCREENING STRATEGY, BUT JUST TRYING TO LOOK OVERALL AT THE COST, AND OUR STRATEGY HAS BEEN TO GATHER ESTIMATES AND RANGES THAT CAN BE USEFUL FOR STATES AS WELL AS THE ADVISORY COMMITTEE, BUT AT THE SAME TIME, MINIMIZING THE BURDEN ON RESPONDENTS TO GATHER THIS INFORMATION AND OBVIOUSLY WE'RE NOT BEING PRESCRIPTIVE ABOUT HOW THESE DATA WILL ULTIMATELY BE USED. SO ONE OF THE FIRST TASKS WE DID WAS TO LOOK AT THE VARIOUS CATEGORIES FOR COST THAT PLAY INTO THE COST OF DOING THE SCREENING TESTS AND THE FOLLOW-UP. AND I'M GOING TO TALK ABOUT THE FOLLOW-UP IN MORE DETAIL IN A SECOND, BUT IF YOU JUST THINK ABOUT GENERAL CATEGORIES, THERE'S EQUIPMENT AND CONSUMABLES, AND THERE'S DIFFERENT WAYS OF GOING ABOUT GETTING THIS STUFF. YOU CAN EITHER GO AND PURCHASE IT AND PURCHASE THE SUPPLIES AND THE REAGENTS AND THAT KIND OF THING OR CAN YOU HAVE A REAGENT RENTAL AGREEMENT WHERE MATERIAL IS SUPPLIED TO THE LABORATORY. THERE'S A GROUP OF OTHER LABORATORY EXPENSES WHICH DEPENDING UPON THE PROGRAM THAT WE'RE TALKING ABOUT ARE THINGS THAT AREN'T ALREADY INCLUDED IN THE EQUIPMENT AND CONSUMABLES, THINGS LIKE MAINTENANCE, REPAIRS, INSTALLATION, OR UPDATE OF THE LABORATORY INFORMATION MANAGEMENT SYSTEM. THERE'S OBVIOUSLY LABOR AND HOW LABOR IS COSTED OUT DEPENDS UPON THE NUMBER OF PEOPLE WHO WE'RE TALKING ABOUT, THE PARTICULAR POSITION AND OF COURSE WHAT THEIR SALARY AND FRINGE BENEFIT RATE IS. AND THEN THERE'S THIS ISSUE OF CONFIRMATORY TESTING AND REFERRAL, SORT OF THE SHORT-TERM FOLLOW-UP, AND THIS IS ORGANIZED DIFFERENTLY BY DIFFERENT NEWBORN SCREENING PROGRAMS, AND SO SOME NEWBORN SCREENING PROGRAMS ARE -- DO A LOT OF WORK IN THIS SHORT-TERM FOLLOW-UP AND OTHERS DON'T, AND SO IT INTRODUCES THIS ELEMENT OF VARIABILITY WHEN YOU LOOK AT COST, AGAIN ILLUSTRATING IN MORE DETAIL IN A SECOND, AND THEN OF COURSE THERE'S ISSUES OF OVERHEAD AND INDIRECT COSTS WHICH CAN DO THINGS LIKE PAY FOR THE SPACE OR THE BUILDING AS WELL AS UTILITIES AND ALL THE OTHER THINGS THAT GO INTO OVERHEAD. SO WE AS A GROUP DEVELOPED A TEMPLATE AND THE COMPONENTS -- THIS IS THE KIND OF THING THAT WHILE WORKING WITH AN INDIVIDUAL NEWBORN SCREENING PROGRAM, WE CAN TRY TO ELICIT THIS MATERIAL. SO FACTORS TO INCLUDE ARE THE NUMBER OF SPECIMENS THAT THE NEWBORN SCREENING PROGRAM EVALUATES, AND WE REALLY FOCUSED ON SPECIMENS BECAUSE THAT'S DIFFERENT THAN THE NUMBER OF INDIVIDUALS THAT ARE SCREENED BECAUSE FOR EXAMPLE SOME STATES ARE TWO SCREEN TESTS, SOME STATES ARE ONE SCREEN TEST, AND THERE ARE SOME BABIES THAT WILL HAVE REPEAT SCREENS DONE FOR OTHER WAYS, SO EVEN IN THE SINGLE SCREEN STATE IT'S NEVER ONE PER ONE. SO ANYWAY, LOOKING AT THE NUMBER OF SPECIMENS OF THIS DATE OR THE NEWBORN SCREENING PROGRAM DOES ANNUALLY, THE PLATFORM SPECIFIC TEST THAT'S DONE, AND THEN EQUIPMENT, CONSUMABLES, OTHER LAB EXPENSES, THE LABOR WE TALKED ABOUT, ISSUES OF CONFIRMATORY TESTING AND OVERHEAD AS I JUST TALKED ABOUT BEFORE. SO WHAT I WANT TO DO IS JUST SHOW YOU AN EXAMPLE OF A SPREADSHEET THAT WE FILLED OUT AND JUST TO MINIMIZE THE NUMBER OF SLIDES THAT I'M SHOWING YOU, YOU'LL SEE I HAVE STATES COMPARED -- STATE A, STATE B IN THE NEXT SLIDE US A MIGHT GUESS, WE HAVE C AND D. THE FACT THAT THEY'RE NEXT TO EACH OTHER, I WOULD REALLY AVOID SORT OF COMPARING ACROSS THE LINES. AND THAT'S BECAUSE THE NUMBER OF SPECIMENS THAT ARE TESTED ANNUALLY MIGHT BE DIFFERENT. THE PLATFORMS ARE DIFFERENT, THE NUMBER OF TESTS THAT ARE DONE -- MULTIPLEXING THAT'S GOING TO BE DONE, BUT IF YOU JUST LOOK AT -- STATES REALLY ASKED US TO MAINTAIN CONFIDENTIALITY, BECAUSE A LOT OF THESE NUMBERS ARE REALLY PROPRIETARY IN TERMS OF HOW THEY -- CONTRACTS AND THAT KIND OF THING AND THEY DIDN'T WANT THE NAMES OF THE STATES DIVULGED, SO I WON'T BE REVEALING THAT HERE. SO HERE WE HAVE TWO STATES, ONE WITH 100,000 SPECIMENS TESTED ANNUALLY AND ANOTHER, 180,000. YOU CAN SEE DIFFERENT PLATFORMS, YOU CAN SEE ONE STATE HAS A REAGENT RENTAL AGREEMENT AND THE OTHER STATE HAS PURCHASED EQUIPMENT. THE NUMBER OF CONDITIONS TESTED USING EACH PLATFORM ARE DIFFERENT, SO ONE IS A FOUR PLEX, THE OTHER IS SIX. THE STATE THAT HAD THIS RENTAL AGREEMENT DIDN'T GIVE US ANY COST OF CONSUMABLES BUT THE ONE THAT USED MASS SPEC DID. THERE'S OTHER LABORATORY EXPENSES, I SHOULD HAVE DROPPED THAT ONE LEVEL DOWN BUT YOU CAN SEE THE BIG DIFFERENCES IN TERMS OF LABORATORY PERSONNEL, ONE STATE DID NOT PROVIDE US WITH OVERHEAD OR INDIRECT COSTS. SO IF YOU DO THIS MAP, YOU CAN FIGURE OUT WHAT THE COST PER SPECIMEN IS, AND THAT'S THE NUMBER IN THE LEFT IN EACH COLUMN, AND THEN THE COST PER SPECIMEN PER CONDITION WHERE I JUST TOOK FOR STATE A AND DIVIDED IT BY 4 AND FOR STATE B DIVIDED IT BY 6, BUT IT'S NOT LIKE THERE'S THIS LINEAR ASSOCIATION BETWEEN THE NUMBER OF THINGS THAT YOU SCREEN FOR AND COST, SO IT'S A SIMPLIFYING ASSUMPTION BUT IN REALITY, IT DOESN'T MAKE SENSE BECAUSE YOU INVEST A LOT TO GET THE SCREEN TESTS AND THERE'S A SMALL PROBABLY INCREMENTAL COST FOR EACH ADDITIONAL ONE. BUT I WANTED TO BE ABLE TO AT LEAST PUT IT ON SOME SORT OF, YOU KNOW, STANDARDIZED FRAMEWORK. AND THEN HERE IS -- YOU CAN SEE STATE C, 80,000, 98,000, YOU CAN SEE THIS STATE, THE 98,000 GAVE US THE RENTAL REAGENT AGREEMENT AND IN ADDITION, YOU KNOW, LARGE AMOUNT FOR CONSUMABLES AND OTHER LABORATORY EXPENSES AND LABOR WHICH STATE C DIDN'T, AND YOU CAN SEE WHERE THE NUMBERS WIDDLE DOWN TO IN THE END. SO THE THING I WANT TO POINT OUT FROM THIS IS NEWBORN SCREENING PROGRAMS DO A LOT OF WORK, RIGHT, AND FIGURING OUT THE EXACT COST FOR OUR PURPOSES IS NOT SOMETHING THAT'S PART OF THEIR JOB, RIGHT? SO IN A SENSE, IT'S NOT SURPRISING THAT IT'S HARD TO ELICIT THE NUMBERS AND GET THEM INTO THE BUCKETS THAT WE WANT THEM TO BE IN, SO IT JUST BRIRNTION INTO HOW ACCURATE ARE THESE NUMBERS REALLY. THE OTHER THING I WANT TO MAKE SURE THAT PEOPLE APPRECIATE IS THAT THE NUMBER OF SPECIMENS THAT ARE DONE IN A STATE HAS IMPACT ON WHAT THE OVERALL COSTS WOULD BE, SO IF YOU'RE A SMALLER STATE, YOU HAVE A FEWER NUMBER OF NEWBORNS TO TEST, YOU'RE TAKING YOUR STARTUP COSTS AND PUTTING IT OVER A FEWER NUMBER OF BABIES BEING TESTED. SO ONE WAY THAT SOME NEWBORN SCREENING PROGRAMS DO THAT IS THEY PARTNER WITH OTHER PROGRAMS AND HAVE MORE CENTRALIZED TESTING, BUT THERE IS THIS FACTOR ABOUT THE NUMBER OF SPECIMENS THAT ARE TESTED AND ULTIMATELY WHAT COSTS ARE. THE OTHER THING, AND I ALLUDED TO THIS BEFORE, IS THAT DIFFERENT NEWBORN SCREENING PROGRAMS HAVE DIFFERENT TACTS FOR LONG TERM FOLLOW-UP, GENETIC TESTING AND THAT SORT OF THING, SO THAT'S SORT OF BORNE DIFFERENTIALLY BY STATES. SO ONE OF THE SIMPLIFYING THINGS WE DID WAS REALLY FOCUS ON THE COST OF TESTING THE SPECIMEN AND NOT THOSE OTHER FOLLOW-UP COSTS, WHICH YOU KNOW, MY GUESS IS THEY PROBABLY ARE MUCH SMALLER THAN THE ACTUAL SCREENING TEST ANYWAY. WE'RE GOING TO REVISIT THIS AGAIN IN A SECOND. ONE POINT I WANT TO MAKE SURE THAT I THROW OUT TOO, DIFFERENT PLATFORMS AND DIFFERENT TESTING STRATEGIES IN GENERAL ARE GOING TO HAVE DIFFERENT NUMBERS OF FALSE POSITIVES TOO, SO THAT'S GOING TO AFFECT THIS FOLLOW-UP COST AS WELL. SO WE MADE A LOT OF SIMPLIFYING SUGGESTIONS AND DID THE BEST WE COULD. HIGHLIGHTING WHAT I SAID BEFORE, ALL STATES INCUR SOME SORT OF FOLLOW-UP COSTS BUT ONLY ONE STATE REPORTED THE FOLLOW-UP COST AND COST OF CONFIRMATORY TESTING, SO EVEN THOUGH NEWBORN SCREENING PROGRAMS MAY NOT BE BEARING A LOT OF THESE COSTS, CLEARLY THE SYSTEM SOCIETALLY DOES. MEDICAID COVERS A LOT OF FOLLOW-UP TESTING IN MOST STATES, SO IT IS SOMETHING THAT THE -- THAT'S ABSORBED SOMEWHERE. SO EVERYONE KNEW -- K.K. CAME UP WITH THIS, TO GIVE HER CREDIT BUT REALLY, I FELT LIKE WE WERE COMPARING, YOU KNOW, APPLES TO APPLES AND THERE'S JUST SO MUCH VARIATION, IF YOU LEAVE WITH NOTHING, JUST REMEMBER THE SLIDE FOR THE AMOUNT OF VARIATION. OF COURSE WHEN SHE SAID THAT WE WERE COMPARING APPLES TO APPLES, THAT'S WHAT I THOUGHT OF AT FIRST. [LAUGHTER] IT'S JUST THE PICKY PERSON INSIDE OF ME. SO ANYWAY, WE HAD A LOT OF ASSUMPTIONS THAT WE HAD TO BUILD IN, AND IT'S IMPORTANT TO UNDERSTAND THE CONTEXT. THERE'S THIS HUGE VARIATION IN STATE ANNUAL BIRTH RATES, THERE'S VARIATIONS IN THE NUMBER OF SPECIMENS PER BABY, WE TALKED ABOUT THE TWO VERSUS ONE STATES, THERE'S ISSUES OF WHO PAYS FOR WHAT, THERE'S THE ISSUE OF TIMING SO WHEN YOU FIRST START THINGS OFF, THERE'S A LOT OF UP FRONT COSTS, AND THERE'S DIFFERENT PEOPLE THAT MAY BE INVOLVED IN PAYING FOR THIS, AND THEN OVER TIME TOO, THERE HAVE BECOME SCREENING EFFICIENCIES, SO SORT OF -- WE WANTED TO HAVE THIS TWO-YEAR PROJECTION BUT I THINK REALLY, IN THE END, WE PROBABLY KNOW MORE ABOUT THE INITIAL START-UP COSTS, AND THEN THERE ARE ALL SORTS OF OTHER THINGS HAPPENING IN THE STATE THAT IMPACT LIKE WHO PAYS FOR WHAT, WHERE COSTS APPEAR. AND AGAIN, ALL THESE OTHER SOURCES OF VARIATION THAT THIS COMMITTEE THINKS A LOT ABOUT IN TERMS OF SCREENING ALGORITHM, DIFFERENT LABORATORIES, ACCESS TO SPECIALIZED SERVICES, ISSUES RELATED TO CONDITION, ONE COULD GO ON AND ON THINKING ABOUT THINGS THAT COULD CAUSE COSTS TO VARY. SO AS I THINK EVERYONE IS AWARE THAT WHEN A CONDITION DOES GO FOR REVIEW, THERE'S A NINE-MONTH PERIOD FOR THAT REVIEW TO HAPPEN SO THERE'S LIMITED TIME FOR COLLECTING DATA, AND IF YOU REALLY WANTED TO GET TO THOSE COSTS, IT WOULD REQUIRE A FAIR AMOUNT OF ATTENTION AND I REALLY THINK THAT THE NEWBORN SCREENING PROGRAMS WOULD NEED ASSISTANCE LIKE HOW THE CDC CAN GET INVOLVED WITH DOING THESE VERY CAREFUL EVALUATIONS WITHIN NEWBORN SCREENING PROGRAMS BUT IT'S JUST NOT GOING TO HAPPEN WITH THIS SHORT PERIOD OF TIME THAT WE HAVE TO GET TO THAT LEVEL OF TEE TAIL. AND AS I SAID BEFORE, THIS IS NOT WHAT THE NEWBORN SCREENING PROGRAMS SIT AROUND AND THINK ABOUT US, BUT WE JUST NEED TO WORK WITH THEM TO GET IT WHERE WE CAN, ESTIMATES WILL MOSTLY REPRESENT EARLY ADOPTERS, I MENTIONED THIS ISSUE BEFORE ABOUT COSTS BE HIGHER FOR STATES WITH LOWER TESTING VOLUMES, AND THEN ONE OF THE THINGS THAT WAS VERY INTERESTING THAT I DIDN'T APPRECIATE BEFORE WE STARTED GATHERING INFORMATION WAS THAT THE PRIVACY ISSUES THAT NEWBORN SCREENING LABORATORIES FACE IN TERMS OF DETAILS THAT THEY CAN SHARE WITH US. SO AGAIN THINKING AHEAD WHAT ARE WE GOING TO DO IF NO U.S. STATE HAS STARTED SCREENING OR IS IN THE PLANNING PROCESS OF SCREENING, SO FOR EXAMPLE THERE WAS A PILOT STUDY THAT WAS DONE IN AUSTRALIA OR SR. LIKE THAT THASOMETHING LIKETHAT THAT WAS ENOUGH TO M OVE TO EVIDENCE REVIEW, SO AT THAT POINT WE HAD TO WORK WITH VENDORS AND RESEARCHERS, BUT THAT MAY NOT REFLECT WHAT'S GOING ON, AND THEN OF COURSE THESE OTHER THINGS THAT HAPPEN IN TERMS OF THE PRICING THE EQUIPMENT, FDA APPROVAL ISSUES, NEW SCREENING TECHNOLOGY, ALL SORTS OF THINGS THAT ARE GOING TO MUDDY THE WATER. SO WHAT DO I THINK WE CAN PROVIDE? AS LONG AS THERE'S AT LEAST ONE STATE THAT'S DOING IT OR IS IN THE PLANNING STAGES AND IS WILLING TO PROVIDE CONSTANT INFORMATION, I THINK THAT WE CAN GET AT LEAST IN SORT OF A BROAD SENSE THE OVERALL ESTIMATE STARTUP SCREENING AND LABORATORY COSTS, AND THEN MAKE OTHER ESTIMATES BASED ON THE UNIQUE CHARACTERISTICS OF THE STATE OR STATES THAT WE'RE ABLE TO ACCESS. AGAIN OUR COST ASSESSMENT PLAN, HOPEFULLY WE'LL BE ABLE TO AS A PRIMARY SOURCE OF DATA GO TO STATES, IN TERMS OF THE ESTIMATES THAT WE HOPE TO GENERATE, IT WOULD BE COST PER SPECIMEN TO ADD THAT PARTICULAR CONDITION. AND ONE THING, I HAVE TO THANK ANN MARIE WHO POINTED THIS OUT IS THAT EVERYTHING I'VE TALKED ABOUT SO FAR REFLECTS TRADITIONAL DRY BLOOD SPOT SCREENING, SO WE'LL HAVE TO RECONSIDER HOW WE'RE GOING TO DO THINGS AS POINT OF CARE NEWBORN SCREENING TEST IS UNDER CONSIDERATION. SO WE WILL PUT TOGETHER A NARRATIVE DESCRIPTION, AT LEAST SUMMARIZING WHAT WE KNOW IN TERMS OF THE REQUIREMENTS FOR SCREENING, THE ASSUMPTIONS THAT WE MADE, AND THE SOURCES AND METHODS OF GETTING THE COST ESTIMATES, SO AT LEAST WHEN YOU LOOK AT THESE NUMBERS, YOU CAN UNDERSTAND WHERE THEY CAME FROM. AND SO OUR NEXT STEPS ARE GOING TO BE TO FINALIZE THIS APPROACH AND SUBMIT A REPORT AS WELL TO THE ADVISORY COMMITTEE AND THEN WE'LL BE READY TO I THINK RATE THIS AS WE'RE ABLE TO INTO THE CONDITION REVIEW PROCEDURES AND THE OVERALL TIMELINE THAT WE HAVE. NOW, I'M GOING TO GO AND OPEN UP THE FLOOR FOR QUESTIONS, BUT I'D LIKE TO INVITE SCOTT GROSS TO COME UP. SCOTT HAS BEEN INKRED PLI INCREDIBLY HELPFUL IN DOING THIS AND HE'S LIKE THE REAL CARD CARRYING ECONOMIST, I JUST PLAY ONE FOR THE ADVISORY COMMITTEE. BUT I THINK IT'S IMPORTANT TO HAVE HIM UP HERE BECAUSE IF YOU ASK A REALLY SPECIFIC ECONOMIC QUESTION, I WANT TO MAKE SURE WE'RE ABLE TO GIVE YOU A GOOD ANSWER. SO NOW THAT HE'S UP, I CAN OPEN THE FLOOR FOR QUESTIONS. >> ALL RIGHT, THANK YOU, ALEX, AND THANK THE CDC FOR LENDING SCOTT OUT TO US. I THINK THAT'S BEEN GREAT, A BIG HELP. SO I WANT TO THANK YOU AND THE MEMBERS OF THAT WORK GROUP FOR ALL THE WORK THAT YOU'VE DONE TO TRY AND STANDARDIZE THIS IN SOME WAY THAT IT'S GOING TO BE BENEFICIAL TO THE COMMITTEE AND PROVIDE THE DATA THAT WE NEED WHEN THE CONDITION COMES BACK TO US WITH ALL OF THE ANSWERS. SO LET'S OPEN THIS TO ANY QUESTIONS OR COMMENTS FROM THE COMMITTEE. JOAN. >> THANK YOU VERY MUCH. THAT WAS REALLY HELPFUL, AND IT AGAIN ILLUSTRATES HOW DIFFICULT, YOU KNOW, COSTS CAN BE, MEAN SO MANY DIFFERENT THINGS, AND THERE ARE SO MANY VARIABLES WHEN YOU DO THIS KIND OF ANALYSIS. SO WHEN IT IS DONE AND THAT INFORMATION IS PROVIDED TO THE COMMITTEE AS PART OF THAT RUBRIC ALONG WITH THE EVIDENCE REVIEW AND THE PUBLIC HEALTH IMPACT, WHAT ARE THE DANGERS THAT THE COMMITTEE SHOULD BE AWARE OF IN CONSIDERING THAT INFORMATION? DOES THAT MAKE SENSE? >> THAT TOTALLY MADE SENSE BECAUSE IT INFORMS HOW YOU WEIGH THAT. SO I HAVE A COUPLE OF OBSERVATIONS. FIRST OF ALL, WE'RE ONLY LOOKING AT ONE SIDE OF THE EQUATION, RIGHT, SO WE'RE LOOKING AT THE COST OF THE SCREENING, WE'RE ALSO NOT EVEN LOOKING AT THE DIAGNOSTIC TEST AND SO FORTH, SO JUST -- WHAT I HOPE IT GIVES YOU INSIGHT INTO WHAT THE IMPACT MIGHT BE ON WHAT THE NEWBORN SCREENING LABORATORY PROGRAM WOULD HAVE TO IP VES TO INVEST TO SCREEN FOR THOSE THINGS BUT IT'S KIND OF AN UNBALANCED EQUATION. THAT BEING SAID, THOUGH, OFTENTIMES WE HEAR THAT THE VARIOUS PREVENTIVE INTERVEPTIONS ARE COST SAVING AND THAT'S REALLY ACTUALLY THE CASE BECAUSE THESE ARE RARE CONDITIONS AND SO EVERY SPECIMEN OR EVERY BABY GETS THIS COST, BUT THE BENEFITS ARE NARROWED DOWN TO A CERTAIN NUMBER OF INDIVIDUALS, BUT THAT DOESN'T MEAN IT'S A BAD THING TO DO. SO WE WILL DO AS BEST WE CAN TO ARTICULATE WHERE WE'RE CERTAIN ABOUT THE NUMBERS AND WHERE WE'RE NOT, AND I THINK IT'S GOING TO BE MORE NOT THAN CERTAIN, BUT I'M SURE THE ADVISORY COMMITTEE, WHEN THEY FIGURE OUT HOW TO USE THESE NUMBERS, WILL BE JUST ONE TEENY BIT OF INFORMATION BUT I THINK WE NEED TO BE CAREFUL ABOUT THIS. ONE OF THE THINGS THAT WORRIES ME TOO IS THAT WHENEVER YOU PRESENT SOMETHING ON A SLIDE IN A COMMITTEE OR WHEREVER WITH ALL THE CAVEATS AROUND IT, PEOPLE LOSE TRACK OF ALL THE CAVEATS AND THE NUMBER GETS OUT THERE AND PEOPLE -- THAT JUST HAPPENS ALL THE TIME. AND SO I JUST FEEL VERY STRONGLY THAT WE NEED TO BE CAREFUL ABOUT HOW MUCHOW MUCH WEIGHT WE PUT INTO THIS AND HOW MUCH IT'S USED. THAT'S SORT OF MY 30,000-FOOT LEVEL, BUT SCOTT? >> ONE OF THE ISSUES ALEX MENTIONED WAS THE COST OF THE TEST MAY CHANGE. WITH FDA APPROVAL, COSTS MAY GO UP. SO TYPICALLY WE HAVE THE COST ESTIMATES FOR THE HOME BREW BEFORE THERE'S AN FDA APPROVED TEST SO WHO KNOWS WHAT THE COST WILL BE EVENTUALLY, THAT MOST STATES WILL HAVE TO PAY. WE DID NOT INCLUDE ANY COST TO THE STATE HEALTH DEPARTMENT FOR ORGANIZING THE PRO SE, ESTABLISHING A CONDITION, ALL THE COMMITTEE MEETINGS, ALL THE STAFF TIME THAT'S TAKEN. THE BIGGEST MISSION IS THERE'S NO COST FOR THE LONG TERM FOLLOW-UP, NOT JUST THE IB FANTS THAT ARE DIAGNOSED BUT THE INFANTS WITH LATE ONSET AND SYMPTOMATIC KIDS THAT HAVE TO BE FOLLOWED UP, WHICH WE HEARD ABOUT THIS MORNING. >> I APPRECIATE HOW MUCH YOU'VE DONE, ALSO RECOGNIZING HELPING US SEE WHAT THE OLD THING ABOUT WHAT YOU PUT IN IS WHAT YOU GET OUT. THE DATA COMING INTO THIS ARE QUITE VARIABLE. I THINK ONE DANGER MIGHT BE THAT WE COME UP WITH A COST FOR THE NEXT CONDITION, AND THEN THE NEXT CONDITION AFTER THAT, WE LOOK AT IT AND SAY WELL, THIS CONDITION IS COSTING A LOT MORE OR A LOT LESS THAN THAT ONE, WE WANT TO MAKE SURE -- THIS IS WHY WHAT YOU'RE DOING IS SO IMPORTANT, WE WANT TO MAKE SURE -- WE WERE FEELING LIKE WE WERE USING THE SAME APPROACH MAKING THAT ESTIMATE ON BOTH OF THEM. BUT IT MIGHT BE WORTH THINKING ABOUT -- I DON'T WANT TO CREATE ANOTHER MATRIX, BUT MAYBE SAYING OKAY, H BASED ON THE DATA, WE THINK THIS IS TYPICAL OF WHAT YOU WOULD EXPECT TO ADD A NEW TEST, IS IT CHEAPER THAN USUAL? THAT WOULD HELP ME MORE IN THE LONG RUN TO THINK ABOUT HOW WE MAKE A DESIX IN TH DECISION IN THE PROCESS BECAUSE THEN YOU LOOK AT $8, WHAT DOES THAT DO FOR US. >> ONE WAY TO LOOK AT IT IS TO LOOK AT SKID, SORT OF A -- FOR A STANDALONE TEST, LIKE $6 TO $8. SAME THING -- LESS THAN THAT WILL BE CONSIDERED -- >> I TOTALLY AGREE WITH THAT. IT'S JUST -- IT'S HARD BECAUSE WE'RE ALSO LOOKING AT ONE SIDE OF THE EQUATION AND NOT THE WHOLE THING. I JUST DON'T WANT THIS TO BECOME LIKE $50,000 PER, YOU KNOW, QUALITY -- YOU KNOW, SOMETHING JUST KIND OF MADE UP, YOU KNOW? >> FOLLOWING ON DON'S COMMENT, COW ALSYOU COULD ALSO WONDER TOO ABOUT GIVING US A SENSE OF HOW UNCERTAIN. THERE MIGHT BE SOME TIMES WE SAY LOOK, HERE'S WHAT THE TESTS COST, PEOPLE ARE PRETTY SURE, IT'S ADDING ONE MORE CONDITION, IT'S NOT A BIG DEAL. THERE MIGHT BE OTHERS WHERE YOU SAY WE'RE SO UNCERTAIN, YOU REALLY SHOULDN'T EVEN LOOK AT THIS NUMBER, EVEN THOUGH WE HAVE TO GIVE YOU A NUMBER, SO THAT MIGHT BE HELPFUL TOO. >> I ACTUALLY WAS GOING TO SAY SOMETHING VERY SIMILAR. THAT'S WHY GOD CREATED ASTERISKS. YOU CAN'T JUST SHOVE EVERYTHING INTO THE SAME BUCKET AND SAY THIS IS A COST ANALYSIS AND -- THE SAME THING, AND EVEN TAKING CCHD OUT OF THE EQUATION, THERE WAS A SIMILARITY BETWEEN CCHD SCREENING AND SKID IN THAT WE KNEW THERE WERE GOING TO BE SECONDARY NON-TARGET CONDITIONS PICKED UP BY A TEST GENERALLY WHERE THE COST OF THE TEST IS THE TEST, RIGHT? BUT IF WERE YOU TO EVALUATE THE COST OF CARE, SHORT TERM FOLLOW-UP, LONG TERM FOLLOW-UP, IT WASN'T A CAN OF WORMS, IT WAS JUST SIMPLY LIKE HOW ARE WE GOING TO TEM STRAIGHT BOTH POTENTIALLY COST SAVE ON THE CLINICAL SIDE THROUGH EARLIER DETECTION VERSUS EXTRA DOLLARS HAVING BEEN EXTENDED FOR ALL THESE ADDITIONAL CONDITIONS THAT ARE BEING PICKED UP THAT WEREN'T ORIGINALLY THE TARGET CONDITIONS. THERE WOULD BE SOME, I IMAGINE, THEY'LL COME BEFORE THIS COMMITTEE BETTER STRAIGHTFORWARD. HERE'S THE ASSAY, NOTHING ELSE YOU'RE GOING TO FIND, IT IS WHAT IT IS, AND YOU CAN FIND YOUR APPLE WITHIN YOUR FRUIT BASKET THERE. THANK YOU TO BOTH OF YOU FOR YOUR LEAD ARE SHIP BY TH LEADERSHIP, BY TH E WAY, I'VE LEARNED A GREAT DEAL IN YOUR WORK GROUP. >> THANK YOU BOTH VERY MUCH. WE LOOK FORWARD TO THE REPORT. ALL RIGHT. NEXT, WE HAVE A SUMMARY OF THE ACTIVITIES OF THE EDUCATION AND TRAINING WORK GROUP. NATASHA IS GOING TO GIVE THAT PRESENTATION FOR THE LEADERS OF THAT WORK GROUP. >> THANK YOU. BOTH CATHY AND BETH HAD SOME TRAVEL LIMITATIONS SO I'M HAPPY TO PRESENT FOR THIS GROUP. I'M HAPPY TO TAKE QUESTIONS BUT I DON'T PROMISE THAT I CAN ANSWER ALL OF THEM. THAT'S THEIR JOB. SO JUST IN TERMS OF OUR AGENDA, WE DID OUR TYPICAL UPDATE FROM OUR MEMBER, THEN SPOKE ABOUT THE NOMINATION AND EDUCATION PROJECT. WE ALSO REVIEWED SOME OF THE WORK GROUP PROJECTS THAT HAVE BEEN DISCUSSED HERE, THEN CLOSED THE SESSION DISCUSSING SOME ADDITIONAL EDUCATION NEEDS AND PROJECT IDEAS. SO FIRST AN UPDATE ON THE NOMINATION EDUCATION PROJECT. THIS IS SOMETHING WE'VE DISCUSSED BEFORE ABOUT REALLY THE NEED FOR PARENTS AND REALLY ANYONE THINKING OF NOMINATING A CONDITION TO HAVE A BETTER SENSE OF WHAT IS THAT PROCESS, WHAT DO THE FORMS MEAN, WHAT ARE THE STEPS, WHAT ORDER ARE THE STEPS, SO THIS IS A PROJECT THAT WE HAVE UNDERTAKEN WITH DR. KEMPER AND HIS TEAM. IN THE PAST, I'D SAY FOUR OR SO MONTHS -- SORRY, WHEN I SAY WE, I MEAN GENETIC ALLIANCE, WORKED WITH DR. KEMPER AND HIS TEAM TO REALLY CREATE BOTH THE TEXT THAT WOULD GO ALONG WITH THIS AS WELL AS A GRAPHICAL REPRESENTATION BAW BAWS WE KNOW PEOPLE ARE TYPICALLY VERY VISUAL IN WANTING TO ACTUALLY SEE WHAT IS THE PATHWAY. SO AT THE MEETING WE PRESENTED THAT AND ARE ALSO EXPLORING SOME OF THE TECHNOLOGICAL CAPABILITIES AND ISSUES IN TERMS OF PUTTING THAT UP ON THE HRSA SITE ON THE COMMITTEE'S WEBSITE. SO THE END GOAL IS TO HAVE SOMETHING VERY EASY FOR PEOPLE TO WALK THEMSELVES THROUGH IN TERMS OF THE NOMINATION PROCESS ON THE ADVISORY COMMITTEE'S WEBSITE. SO AS A REMINDER, THE PROJECT ONE FOR THIS WORK GROUP THAT WAS DISCUSSED WHEN WE DID THE REFORMATTING OF THE DIFFERENT WORK GROUPS WAS TO CREATE A TOOL THAT PROVIDES PRIMARY CARE PROVIDERS WITH GUIDANCE AND STEPS FOR DISCUSSING A POSITIVE NEWBORN SCREENING RESULT WITH PARENTS, SOMETHING THAT COULD BE USED WITH THE ACT SHEETS. AS WE ALL KNOW THE ACT SHEETS ARE REALLY USEFUL AND THEY DO A GOOD JOB OF LAYING OUT WHAT THE CONDITION IS BUT THE IDEA AROUND THIS PROJECT WAS ALMOST MORE OF A COMMUNICATIONS ACCOMPANIESMENT, HOW DO YOU TALK TO FAMILIES WHEN THEY'RE IN THAT SITUATION. SO I HAVE BEEN WORKING WITH MEMBERS OF ACMG PARTICULARLY, ALICIA KEEN AND DR. FLANNERY HAVE BEEN EXTREMELY HELPFUL IN THINKING ABOUT HOW DO WE INCORPORATE THE WORK THAT WAS STARTED A NUMBER OF YEARS AGO WITH GENETIC ALLIANCE AND DR. CAROL GREEN AROUND ISSUES OF TALKING TO FAMILIES WHO WERE EXPERIENCING A FALSE POSITIVE AND WHAT ARE THE COMMUNICATION STRATEGIES AROUND THAT AND IMIEPING THAT IN SOME WAY WITH THE ACT SHEETS, SO WE HAVE MET AND HAD A NUMBER OF EMAIL CHANGES AND HAVE IDENTIFIED PARTICULAR PEOPLE WHO HAVE EXPERIENCE WORKING ON THE ACT SHEET WORKING GROUP THROUGH ACMJ TO COME TOGETHER AND TO THINK ABOUT HOW WOULD WE CREATE SOMETHING THAT'S VERY EASY THAT COULD BE A COMPANION PIECE FOR THE ACT SHEET. SO MORE TO COME ON THAT, AND AT LATER MEETINGS, BUT THAT IS MOVING ALONG. THE COMMITTEE ALSO WILL PROBABLY HELP US IN THE WAY THAT WOULD BE MOST USEFUL WHICH IS TO REVIEW WHATEVER WE END UP COMING UP WITH, SO THE WORK WILL BE DONE BETWEEN GENETIC ALLIANCE AND ACMG AND THEN WHAT COMES OUT OF THAT WILL GO TO THE COMMITTEE FOR REVIEW AND COMMENT. THERE WAS ALSO HOW DO WE ENG RATE THIS IDEA OF THE ACTUAL COMMUNICATION STRATEGIES AND WORKING WITH FAMILIES WHO ARE GOING THROUGH THAT PROCESS, AGAIN, NOT JUST THE CONDITION ITSELF BUT HOW DO YOU TALK TO FAMILIES IN A RANGE OF DIFFERENT ARENAS, AP RESIDENT EDUCATION PROCESS, THERE'S A PEDIATRIC RESIDENT, EDUCATION CURRICULUM THAT THEY'RE LOOKING AT TO SEE HOW TO INCORPORATE MAYBE A CASE STUDY AROUND THIS, SO THERE WILL BE MORE TO COME ON THAT. THAT DISCUSSION LED TO ANOTHER DISCUSS ABOUT PARENT HANDOUTS TO BE USED AT TIME OF NOTIFICATION OF POSITIVE NEWBORN SCREENING RESULTS, THE IDEA THAT YES, YOU CAN HAVE A TOOL FOR PROVIDERS TO USE BUT WOULDN'T IT BE HELPFUL TO HAVE SOMETHING PARENTS CAN GO TO WHEN THEY ARE ABOUT TO HAVE THAT CONVERSATION. THE KEY QUESTIONS TO ASK, THINGS LIKE THAT. SO THAT'S SOMETHING WE'VE SEEN IN OTHER AREAS OF MEDICINE WHERE PEOPLE ARE GETTING RESULTS BACK AND SAYING REMEMBER TO ASK THIS. WE ALL KNOW THAT EXPERIENCE OF HAVING ZERO QUESTIONS WHEN YOU'RE GETTING THE NEWS AND THEN YOU WALK AWAY AND 15 MINUTES LATER, YOU'RE LIKE, OH, HERE ARE ALL THE QUESTIONS I WISH I HAD ASKED. SO THERE IS SOME DISCUSSION ABOUT THAT BUT IT REALLY CIRCLED BACK TO THE CHALLENGES THAT THIS INFORMATION ISN'T OUT THERE, IT'S ACTUALLY HOW DO YOU DISSEMINATE IT AND GET IT INTO THE HANDS OF PEOPLE ONCE THEY NEED IT. SO THE DISCUSSION WAS REALLY TO HOLD ON THAT ENDEAVOR, FOCUS ON THE PRACTITIONER OR RESIDENCY SIDE, BUT THEN ALSO USE THE WORK OF OTHER GROUPS TO REALLY THINK ABOUT NOT CREATING ANYTHING NEW, BUT REALLY THINKING ABOUT WHAT ARE THE CHANNELS THAT WE'VE SEEN THAT ACTUALLY GET INFORMATION INTO THE HANDS OF PARENTS SO THEY KNOW WHEN THEY'RE GETTING THAT INFORMATION, THEY KNOW TO GO HERE, WHEREVER THAT PARENT IS IS. PROJECT TWO AS A FOCUS AREA FOR THE WORK GROUP IS EDUCATIONAL OUTREACH PROJECT. AN IDEA AROUND THIS IS MAPPING OF EDUCATIONAL RESOURCES, SO THE IDEA THAT THERE'S SO MANY RESOURCES OUT THERE, THERE'S SO MANY MATERIALS THAT ARE OUT THERE AND THEY'RE ALL TARGETED TO DIFFERENT PEOPLE WHETHER THAT'S PRENATAL, NEW MOMS, NEW FAMILIES, WHETHER THAT IS GENERAL NEWBORN SCREENING, THE IDEA OF REALLY MAPPING THAT OUT AND SEEING WHAT'S AVAILABLE. THE FORMAT WOULD BE TO HAVE A MATRIX WITH CHARACTERISTICS THAT WERE SEEN AS IMPORTANT, SO THE AUDIENCE, THE LOCATION, SOMETHING THAT CAN BE USED REGIONALLY, IS IT SOMETHING THAT'S DID IT HAPPEN AT A BIRTHING CENTER OR AT HOME, SO A RANGE OF DIFFERENT PIECES. ALL GREAT IDEAS. WHEN THERE ISN'T FUNDING AVAILABLE, IT'S REALLY DIFFICULT TO KNOW WHERE TO MOVE FORWARD. SO THERE'S A LOT OF ENERGY AND PASSION, I WOULD SAY, ABOUT THIS, BUT THEY THOUGHT ABOUT HOW TO USE ORGANIZATIONAL RELATIONSHIPS TO AT LEAST PULL TOGETHER MORE OF THE MATERIALS THAT ARE OUT THERE, MAYBE TEST THE NEW BOSCH SCREENIN -- THEY'RE IN THE P ROCESS OF LAUNCHING OUR RESOURCE CENTER, SOME OF YOU ARE ASKING FOR RESOURCES TO COME IN SO THAT'S SORT OF A FIRST STEP BUT THIS OBVIOUSLY WOULD BE A MUCH BIGGER AND SEPARATE PROJECT THAT THE GROUP IS INTERESTED IN BUT IS STILL THINKING ABOUT HOW TO MOVE FORWARD ON THAT. ANOTHER DISCUSSION THAT CAME UP WAS MIDWIFE EDUCATION. THERE WAS DISCUSSION WHETHER THAT SHOULD BE A PARTICULAR PRIORITY ISSUE FOR THE TRAINING WORK TBREUP. IT WAS DECIDED THAT YOU SHOULD WAIT ON THAT A LITTLE BIT IN TERMS OF REALLY FIGURING OUT WHAT OTHER GROUPS ARE OUT THERE, WHAT OTHER GROUPS ARE DOING THIS WORK. I KNOW DIFFERENT GROUPS, DIFFERENT ORGANIZATIONS AND ALSO STATES ARE LOOKING AT HOW DO THEY REACH MIDWIVES AND THOSE DOING HOME BIRTHS SO REALLY THE DECISION WAS TO KIND OF WAIT AND SEE WHAT OTHER PROJECTS ARE OUT THERE AND THEN SEE HOW THE WORK GROUP HAD LEVERAGED THOSE EFFORTS. TIMELINESS CAME UP IN TERMS OF WHAT ARE THE OPPORTUNITIES FOR EDUCATION, THE GROUP WILL REACH OUT TO SOME HOSPITAL ASSOCIATION, REALLY TRYING TO GET THIS ON THEIR RADAR. ONE REALLY GREAT SUGGESTION THAT CAME FROM DON BAILEY WAS TO EXPLORE CONNECTIONS WITH PHLEBOTOMISTS AND THEIR ASSOCIATIONS, MAYBE EVEN INVITING THEM TO PRESENT TO US IN THE FUTURE TO SEE WHAT ARE THEIR PROCESSES, I THINK SIMILAR TO WHEN YOU'RE TALKING ABOUT -- THE WORK FLOW AND THE PROCESS OF PEOPLE ON THE GROUND WHO ARE INVOLVED TO GET A BETTER SENSE OF HOW WE CAN BE HELPFUL. I BELIEVE THAT'S THE LAST SLIDE. >> THANK YOU, NATASHA. THAT WAS A GOOD SUMMARY OF LOTS OF DIFFERENT THINGS ON THAT COMMITTEE. ANY QUESTIONS OR COMMENTS? ANNMARIE? >> THANKS, NATASHA. GOOD PRESENTATION. SO WHEN YOU SAID YOU WANT TO WAIT ON THE MIDWIFE PIECE, ARE YOU WAITING FOR INFORMATION TO COME OUT OF ANOTHER ENTITY THAT'S INVOLVED WITH THIS COMMITTEE OR ONE OF THE OTHER WORK GROUPS OR IS SOMEONE ACTUALLY TAKING A LOOK, DOING A LANDSCAPE OF WHAT HAS BEEN DONE OUT THERE? THE REAP I BRIN REASON I BRING IT UP IS OV ER THE LAST COUPLE DAYS WE'VE HEARD PRETTY CONSISTENTLY THAT THERE ARE ISSUES WITH HOME DELIVERIES AND JUST SOME DEGREE MAYBE WITH SPILLOVER INTO BIRTHING CENTERS, SO MIDWIVES BEING ABLE TO EXECUTE ON NEWBORN SCREENING IN A CONSISTENT MANNER, AND WHILE I AM ALL ABOUT WONDERFUL EDUCATION MATERIALS TO THE DEGREE THEY'RE AVAILABLE TO EVERY BABY'S FAMILY FAMILY, FOR THE LARGE PART BABIES BEING BORN IN FACILITIES AND HOSPITALS ARE GETTING THE TEST WHEN IS THEY SHOULD GET THE WHEN THEY SHOULD GET THE TESTS AND FOLLOW-UP HAS BEEN FAIRLY WELL DONE, SO THIS SEEMS LIKE ONE AREA THAT WE'RE HAVING TROUBLE NOT JUST WITH EDUCATION, WE'RE HAVING TROUBLE WITH ACTUAL EXECUTION OF SCREENING AND SCREENING CARE DELIVERY, SO I WOULD ELEVATE THAT ON THE PRIORITY LIST VERSUS PUSHING IT OFF TO A -- HOPE I CAN SAY THAT BEING NOT ON YOUR WORK GROUP. THAT'S MY COMMENT. >> I THINK THAT MAKES SENSE. AGAIN I CAN'T SPEAK FOR THE CO-CHAIRS BECAUSE I'M NOT THEM. I THINK IN TERMS OF THE CONVERSATION THAT WE HAD, I IT WAS REALLY TO SEE HOW DOES THAT WORK GROUP BEST KNOW HOW TO LEVERAGE ITS EFFORTS AND TO REALLY FOCUS IN ON WHAT WE CAN DO AND I DEFINITELY OF COURSE LOOK TO THE OTHER PEOPLE WHO WERE THERE TO CHIME IN. SO I THINK THE IDEA OF WAITING WAS IT WAS NOT SO MUCH OF A PRIORITY AS AN ASSESSMENT OF WHAT CAN THAT GROUP REALLY DO AND BE REALLY USEFUL. SO NOW I'M STEPPING OUT OF THAT HAT AND INTO THE NEWBORN SCREENING CLEARINGHOUSE HAT IN TERMS OF THE WORK THAT JACKIE PRESENTED DURING THE PUBLIC COMMENTS AND SEEING THOSE TRENDS, HEARING THAT THERE ARE OTHER TEAMS LOOKING AT THIS ISSUE AND REALLY TRYING TO THINK HOW DO WE ADDRESS THAT. I DON'T THINK THERE'S NECESSARILY A HALT ON THAT. I THINK PEOPLE ARE REALLY TRYING TO THINK OF HOW DO WE REACH IT, AND I THINK YOUR POINT IS EXACTLY RIGHT. OFTENTIMES WHEN YOU TALK ABOUT EDUCATION, WE THINK THAT MEANS THE MATERIAL OR JUST AWARENESS BUILDING, SO I THINK WHAT WE'RE REALLY SEEING IS LIKE HERE'S A TRUE GAP, WHEN WE SAY WE HAVE 98% OF BABIES SCREENED, WE'RE NOW TALKING ABOUT THAT 2% THAT MAY BE FALLING INTO THAT CATEGORY AND IT'S BECAUSE OF KIND OF THE ENVIRONMENT WHERE THEY'RE BEING BORN, SO I WOULD SAY THAT THE WAITING AGAIN WASN'T NECESSARILY -- THIS ISN'T A PRIORITY, BUT IT WAS REALLY TO GET A BETTER ASSESSMENT OF WHAT IS OUT THERE, HOW IS THIS A PRIORITY FOR THE COMMITTEE BECAUSE THE WORK GROUP PRIORITIES ARE CONNECTED TO THE COMMITTEE, OVER ALL PRIORITIES AS WELL AND TO KEEP A WATCH ON IT AND TO BE ABLE TO HAVE A FURTHER DISCUSSION ABOUT IT AT OUR NEXT MEETING. I DON'T KNOW IF THAT COVERS IT OR IF ANYBODY ELSE -- >> I APPRECIATE THAT, NATASHA. I THINK I WAS WONDERING IF YOU HAD TAKEN ON THE CHARGE OF MAKING THAT ASSESSMENT OR IF YOU NEED OUTSIDE SUPPORT FROM OTHER ENTITENTITIES AS FAR AS PUSHING TO THE NEXT PLACE AS A MIDWIFE. >> YES, ALL OF THE ABOVE, ALL OF THE ABOVE NEEDS TO HAPPEN. AND I THINK THAT THAT CAN BE SOMETHING THAT WHETHER WITHIN THE CONTEXT OF THIS GROUP OR OTHER FEDERALLY FUNDED PROJECTS OR STATE BASED PROJECTS, TO REALLY THINK ABOUT, AGAIN, LIKE YOU SAID, IT'S SOMETHING THAT'S COME UP QUITE A BIT, PARTICULARLY WITH TIMELINESS, WHAT IS THE NEXT STEP IN THAT, WHAT SHOULD BE HA HAPPENING. >> THANK YOU VERY MUCH. LET'S HEAR FROM THE FOLLOW-UP AND TREATMENT WORK GROUP. THAT UPDATE WILL BE GIVEN, SINCE DR. MCDONOUGH HAD TO LEAVE EARLY, CAROL GREEN AND ALAN WILL PRESENT AN UPDATE ON THE ACTIVITIES OF THAT WORK GROUP. >> SO WE'RE GOING TO HAND THE SLIDES BACK AND FORTH BECAUSE REALLY WE HAD A VERY LIVELY DISCUSSION AND DR. MCDONOUGH HAD PUT TOGETHER THE SLIDES SO WE'LL HAVE TO TRADE OFF JUST A LITTLE BIT. SO FIRST OF ALL IT WAS A GREAT DISCUSSION. WE HAVE TWO MAJOR PROJECTS. THE FIRST SLIDE JUST REVIEWS THE MEDICAL FOODS SUBWORK GROUP AND JUST FOR FULL DISCLOSURE, THE HEAD OF THAT SUBWORK GROUP ALSO COULD NOT BE HERE OR SUE VERRE WOULD BE STANDING UP DOING THIS PRESENTATION, SO I AM ONE OF SOME OF REMEMBER CO-CHAIRS ALONG WITH CATHY CAMP AND CHRISTINE BROWN. SO THE CHARGE FROM THIS COMMITTEE, HOPEFULLY SAVE ME FROM NEEDING TO GO INTO A FULL REVIEW IS THERE'S SEVERAL NEW MEMBERS OF THE COMMITTEE. ONE, BETH, APPARENTLY NOT HERE AT THIS MOMENT, BUT ACTUALLY KNOWS THE WHOLE HISTORY OF IT BECAUSE SHE'S BEEN LISTENING TO THE PRIOR DISCUSSION AS A LIAISON, AND TWO OTHER MEMBERS OF THE COMMITTEE WERE ACTUALLY THERE FOR THE DISCUSSION, SO ON THE COMMITTEE, REMEMBER THE CHARGE THAT YOU GAVE TO THIS SUBWORK GROUP, AND THAT BASED ON A PRESENTATION AT A PRIOR MEETING FROM CATHY CAMP, THE COMMITTEE WANTED TO SEE SOME OTHER INFORMATION ADDED AND SEE US DEVELOP A WHITE PAPER, A POLICY BRIEF, A REVIEW, A STATE OF THE ISSUE DOCUMENT AND ALSO TO PULL FROM THAT HISTORY OF THE PROBLEMS WITH ACCESS TO MEDICAL FOODS WHAT SOME HAS BEEN DONE HISTORICALLY AND TO LAY OUT WHAT ARE SOME OF THE OPTIONS AND IF WE CAN COME UP WITH ANY RECOMMENDATIONS TO BRING TO THIS COMMITTEE, THAT THIS COMMITTEE WOULD THEN WANT TO BRING TO THE SECOND. IT'S PART OF THE REASON THE DISCUSSION WAS SO LIVELY I IS AT THE SAME TIME, THE WORK GROUP'S PROCESS IS INFORMING SOME OF THE ACTIONS OF MANY OF THE ORGANIZATIONS THAT ARE SENDING PEOPLE TO THE WORK GROUP, SO THERE'S A LOT OF INTEREST GOING ON. THIS COMMITTEE HAD ASKED US TO INCLUDE SOME INFORMATION ABOUT THE IOM REPORT AND THERE IS A DRAFT OF THIS PAPER IN PROCESS. SUE VERRE HAS DONE A LOT OF WORK, WE'RE INCLUDING INFORMATION ON MATERNAL PKU, SO WE'RE ATTEMPTING TO PUT THIS INTO THE DRAFT AND HAVE A DRAFT TO THIS COMMITTEE BEFORE THE NOVEMBER MEETING IN THE HOPES THAT IT COULD BE COMPLETED IF WE CAN GET IT ALL TO YOU IN TIME AND THIS IS JUST TO REMIND YOU THAT THE CHAIR IS SUE VERRE, CO-CHAIRS A LOT OF MEMBERS, LIVELY DISCUSSION, AND IN ORDER TO WORK ON THIS, WE'VE HAD THREE PHONE CALL MEETINGS SINCE THE LAST MEETING OF THIS COMMITTEE. AND WE ARE IN THE MIDDLE OF DRAFTING INCORPORATING THE RECOMMENDATIONS -- OR INCORPORATING THE DISCUSSIONS ON THIS COMMITTEE AND WILL BRING IT BACK TO YOU. BACK AND FORTH AND QUESTIONS AT THE END, I THINK. >> THANK YOU. THE QUALITY MEASURES WORK GROUP HAS A HARD TASK IN DEFINING EXACTLY WHAT QUALITY MEASURES ARE AND HOW WE GOT HERE, BUT IT IS REALLY THE NEXT LOGICAL STEP IN THE PRIOR WORK OF THE LONG TERM FOLLOW-UP COMMITTEE AND THE CONNECTIONS BECAME VERY APPARENT YESTERDAY. OUR REFINED CHARGE IS GOING TO BRIQ TBRING TO THIS COMMITTEE A REPORT HIGHLIGHTING THE STATE OF THE ART OF QUALITY MEASUREMENT AND IDENTIFYING OPPORTUNITIES TO USE QUALITY MEASURES FOR THE LONG TERM FOLLOW-UP OF NEWBORN SCREENING. WE WILL BE ILLUSTRATING THAT BY DEVELOPING A SET OF CASE STUDIES THAT DEMONSTRATE THE VALUE OF WORK THAT'S ALREADY BEEN DONE, AND HIGHLIGHTING DIFFERENT APPROACHES WHICH DIFFERENT GROUPS ARE USING. AND TO HELP DEAL WITH THE PROBLEM OF EFFICIENT USE OF EXISTING RESOURCES AND GET MORE PEOPLE ENGAGED IN LONG TERM FOLLOW HAD BEEN-UP AND QUALITY MEASUREMENT WILL BE INCLUDING A HOW TO GUIDE ILLUSTRATING THE PROCESS DEVELOPING, IMPLEMENTING QUALITY MEASURES AND PARTICULARLY IDENTIFYING RESOURCES FOR ASSISTANCE SUCH AS STAFF TO GET A MEASURE APPROVED AT THE NATIONAL QUALITY FORUM. I CHAIR THIS GROUP AS A PRIMARY CARE PEDIATRICIAN WORKING WITH CHILDREN WITH SPECIAL NEEDS, AND AS A BOARD CERTIFIED CLINICAL INFORMATICIAN. MY CO-CHAIRS, AMY BROWER, JANA MONOCO, AND KATHRYN HASSELL, WORKING WITH THE GENETICS COLLABORATIVE. SO OUR DISCUSSION NOW, LOOK AT THE DIFFERENT APPROACHES TO QUALITY MEASUREMENT IN THE PUBLIC HEALTH SPHERE, AMONG SPECIALTY AND PRIMARY CARE PROVIDERS AND THE NEED FOR CONSUMER TESTIFY ANYTHING'S OF WHAT IS QUALIT -- WE HAVE LARGEMEMBERSHIP BRINGING TOGETHER DIFFERENT COMPONENTS OF THE QUALITY MEASUREMENT PROCESS PEOPLE HAD EXPERIENCED IN A RANGE OF PROCESS, AND WE'RE ALSO LOOKING FORWARD TO INPUT FROM CAMILLA MYSTERY, COMMITTEE MEMBER HERE WHO IS AT AHRQ WORKING VERY MUCH IN THIS AREA. THE LONG TERM FOLLOW-UP WORK GROUP HAS SORT OF SPLIT ITS ACTIVITIES SO THAT WE ARE HOLDING SEPARATE MONTHLY CALLS FOR EACH OF THE TWO PROJECTS AND THEN COMING TOGETHER QUARTERLY FOR DISCUSSIONS ACROSS THE FULL WORK GROUP. AT THE MEETING YESTERDAY, WE HAD SOME VERY ANIMATED DISCUSSION ABOUT THE EMERGING KEY FINDINGS FOR THE EXECUTIVE SUMMARY. THE CHANGING ENVIRONMENT, AVAILABLE RESOURCES AND OPPORTUNITIES. THERE IS A GREAT DEAL OF INTEREST AND INCENTIVE TO ENGAGE IN QUALITY MEASUREMENT, AND THAT, I THINK, WAS VERY APPARENT HERE AT THIS MEETING, WHERE IT CAME UP REPEATEDLY. BUT CONNECTING TO PRIOR WORK, IT IMPORTANT TO REMEMBER BACK IN 2008, THE LONG TERM FOLLOW-UP SUBCOMMITTEE PUBLISHED A PAPER EMPHASIZING THE NEED TO ENGAGE IN THE SAME KIND OF QUESTIONS WE'RE TALKING ABOUT NOW, ONE FROM A LEARNING HEALTHCARE SYSTEM PERSPECTIVE, OF ACQUIRING AND DISCOVERING NEW KNOWLEDGE, AND THEN BRINGING EVIDENCE-BASED TREATMENT INTO PRACTICE. MODERN EHRs NO LONGER JUST RECORD CARE, THEIR PURPOSE IS TO GENERATE NEW GUIDELINES AND UNDERSTANDING OF CARE AND THEN TO BRING THOSE GUIDELINES INTO THE PROCESS AND CHANGE WHAT HAPPENS DURING AN ENCOUNTER. WE ALSO BACK IN 2008 STRESSED THE IMPORTANCE OF COORDINATED CARE IN A MEDICAL HOME AND CONTINUOUS QUALITY IMPROVEMENT SO WE'RE BASICALLY RIGHT ON TARGET THERE. THERE ARE RESOURCES COMING FORWARD FROM NEW STEPS, THE REGIONAL COLLABORATIVES, AS WELL AS FROM CMS, WE THINK CAN MAKE THIS PROCESS EASIER AND MORE COST-EFFECTIVE IN THE FUTURE, AND RECEIPT MAINING OPPORTUNITIES FOR THINGS THAT ARE NEEDED BUT NO ONE HAS ENGAGED IN, PARTICULARLY THE CUSTODIANSHIP AND ADVOCACY OF FOREIGN MEASURES. ONE OF THE VERY INTERESTING CASE STUDIES WE DISCUSSED A LITTLE BIT YESTERDAY WAS A PROJECT AT MOUNTAINS STATES USING A CHECKLIST INTEGRATED INTO AN EHR, ALLATIVES TO PAYING PEOPLE FOR DATA, A PROCESS FOR COLLECTING DATA DURING AN ENCOUNTER, TO GET PEOPLE TO COVER KEY ITEMS WITH PATIENTS, AND THIS HAS NOW BEEN TRANSFERRED TO SOME OF THEIR OTHER CONDITIONS. OTHER ITEMS WE HOPE WE WILL BRING TO YOU IN MAY WILL BE A DESCRIPTION OF THE CONNECTION BETWEEN QUALITY ASSESSMENT, QUALITY IMPROVEMENT AND CLINICAL DECISION SUPPORT. WE ALSO WANT TO GET MORE PEOPLE FAMILIAR WITH THE EFFORTS AT ONS, AHRQ AND CMS TO DEVELOP NEW STANDARDS FOR INTEGRATING QUALITY MEASUREMENT INTO CARE, INCLUDING THE ELECTRONIC QUALITY MEASURES TO DEFINE WHAT IS DONE, THE QRDA QUALITY REPORTING DOCUMENT ARCHITECTURE TO GET PHYSICIANS TO COMEU COMMUNICATE WITH PUBLIC HEALTH AND PAYORS, AND THE QUALITY DATA MODEL TO FACILITATE EXTRACTION OF DATA DIRECTLY FROM EHRs WITHOUT DUPLICATE DATA ENTRY. >> SO THE OTHER THINGS GOING ON OUTSIDE THIS COMMITTEE IN THE WORK GROUP THAT WE JUST WANTED TO BE SURE, THEY WERE DISCUSSED DURING OUR SUBWORK GROUP MEETING AND WE WANT TO BE SURE THAT THE COMMITTEE IS AWARE OF THEM, HAVE TO DO WITH MEDICAL FOODS. IT IS IMPORTANT TO KNOW AND WE DISCUSSED THIS AND I REALLY WANT TO SAY IT AGAIN IN FRONT OF THE FULL COMMITTEE, THAT ACCESS TO MEDICAL FOODS IS NOT THE ONLY ISSUE IN LONG TERM FOLLOW-UP. THERE ARE MAJOR ISSUES WITH ACCESS TO CARE, A GREAT EXAMPLE WAS GIVEN DURING OUR MEETING AND I JUST WANT TO PUT IT ON THE RECORD FOR THE FULL COMMITTEE THAT CHILDREN WITH CONGENITAL HEART DEFECT DON'T ALWAYS HAVE ACCESS TO THE MEDICALLY RECOMMENDED CARE AND MONITORING FOR FOLLOW-UP FOR THOSE CHILDREN. MEDICAL FOODS HAS BEEN AN IMPORTANT ISSUE FOR DECADES NOW AND THIS COMMITTEE HAS DECIDED TO PUT SOME ATTENTION TO IT, AND AT THE SAME TIME THAT THE SUBWORK GROUP IS WORKING ON THIS POLICY PAPER, THERE SEEMS TO BE A PARTICULARLY GOOD TIME TO HAVE SUCH A PAPER, THE AMERICAN MEDICAL ASSOCIATION HAS JUST PASSED A RESOLUTION BROUGHT BY THE AMERICAN COLLEGE OF MEDICAL GENETICS THAT SAYS THE AMA IS SOLIDLY BEHIND COVERAGE FOR NUTRITIONAL -- FOR MEDICAL FOODS FOR TREATMENT OF INBORN ERRORS OF METABOLISM. I WON'T GET INTO ALL THE DETAILS OF PART 2 OF THAT RESOLUTION, BUT IT DOES MEAN THAT THEY HAVE MADE A POWERFUL -- THAT A POWERFUL ORGANIZATION HAS MADE A STATEMENT THAT COVERAGE IS NEEDED, YORK IS WORKING ON SUCH A RESOLUTION, THEY INTEND TO BRING IT TO THE ACADEMY OF FAMILY PHYSICIANS NATIONALLY, AAP, I UNDERSTAND, IS POSSIBLY WORKING WITH ASP, AND THE VERY IMPORTANT MILITARY HAS MADE SOME PROGRESS IN THIS. SO THIS IS AN AREA RECEIVING SOME NATIONAL ATTENTION. HOPEFULLY PEOPLE ARE TALKING ABOUT APPROACHING LEGISLATORS AND SO THIS IS ONE OF THE REASONS WE'RE IN SUCH A HURRY TO GET ALL THIS BACKGROUND OUT THERE AND GET A GOOD EXECUTIVE SUMMARY THAT PEOPLE CAN TAKE AROUND AS TALKING POINTS SO THAT PEOPLE WHO ARE TRYING TO MAKE PROGRESS WILL BE INFORMED. >> FINALLY, DR. MCDONOUGH REGRETS HE CAN'T BE HERE HIMSELF TO SHARE WITH YOU SOME OF THE IMPORTANT IDEAS HE KEEPS BRINGING BACK TO OUR WORK GROUP. IF YOU CAN MEASURE IT AND YOU DON'T MEASURE IT, IT'S NOT IMPORTANT. WE REALLY DON'T KNOW HOW MANY STATES ARE NOT DOING LONG TERM FOLLOW UP AND WHAT THEY'RE MISSING, AND AN INTERESTING INTERSECTION BETWEEN MEDICAL FOODS AND QUALITY IS THE UNKNOWN PERCENTAGE OF PREGNANT WOMEN WITH PKU WHO HAVE GOOD CONTROL DURING THEIR PREGNANCY AND WHETHER WE'RE SEEING A RETURN OF MATERNAL PKU SIB DROME THAT'S ALMOST THE STEP BACKWARDS FROM WHERE WE BEGAN NEWBORN SCREENING 50 YEARS AGO. MANY OTHER AREAS OF MEDICINE REGIONAL VARIATION IN OUTCOMES UTILIZATION HEALTH SERVICES HAVE BEEN IMPORTANT GUIDES AND PERHAPS WE NEED TO KNOW MORE ABOUT HOW OUTCOMES VARY IN DIFFERENT PARTS OF THE COUNTRY AND WHY, AND WHAT ARE BEST PRACTICE FOR DEALING WITH THE CONDITIONS DETECTED BY NEWBORN SCREENING. AND AGAIN, TO THANK THE COMMITTEE FOR YOUR ATTENTION TO LONG TERM FOLLOW-UP, DR. MCDONOUGH WANTED TO SHARE A PICTURE OF MATERNAL CHILD INTERACTION TAKEN ON HIS RECENT TRIP TO ALASKA. >> THANK YOU BOTH VERY MUCH. NICE PRESENTATION. QUESTIONS, COMMENTS? THERE'S ALSO A LOT OF ACTIVITY IN THIS WORK GROUP AS WELL, SO THANK YOU BOTH VERY MUCH. LABORATORY PROCEDURES AND STANDARDS WORK GROUP. KELLIE KELM WILL PRESENT THIS UPDATE. OH, SUSAN THANKFULLY AS WELL. OKAY. >> WE'RE ACTUALLY BOTH STILL HERE! >> SO WE HAD A VERY ATYPICAL WORK GROUP MEETING BUT IT WAS ACTUALLY EXTREMELY INTERESTING. SO HERE IS OUR CURRENT WORK GROUP ROSTER. WE ACTUALLY REALIZED AS WE WERE DISCUSSING YESTERDAY, WE'VE LOST A FEW TO RETIREMENT IN THE LAST YEAR OR TWO, SO WE'RE DEFINITELY LOOKING FORWARD TO WORKING WITH DEBBIE ON SOME -- FINDING SOME NEW MEMBERS. SO PITCH FOR ANYBODY OUT THERE. SO THE TWO PROJECTS THAT OUR WORK GROUP WERE RECENTLY TASKED WITH FROM THE COMMITTEE WAS, NUMBER ONE, TO EXPLORE THE ROLE OF NEXT GENERATION SEQUENCING IN NEWBORN SCREENING, AND NUMBER TWO, TO REVIEW DATA RELATED TO THE TIMELINESS GOALS AND TO LOOK AT THINGS SUCH AS IMPLICATIONS OF EARLIER TESTING WINDOWS IN 24 TO 48 HOURS AND UNFORESEEN CONSEQUENCES IN OTHER ITEMS AS WELL. WHEN WE WERE TALKING ABOUT THIS MEETING AND REALIZED THAT ALL THESE PRESENTATIONS WERE ACTUALLY GOING ON WITH THE LARGE COMMITTEE, WE THOUGHT WHAT WOULD BE INTERESTING FOR US IS -- MOST OF US ARE LAB PEOPLE, GETTING TOGETHER AND TALKING ABOUT THE PRESENTATIONS AND THAT WE DEPARTMENT NEED ANY ADDITIONAL ONES BECAUSE THE COMMITTEE MEETING WAS REALLY COVERING ALL THESE TOPICS. SO THAT'S WHAT WE DID. WE ACTUALLY HAD TWO HOURS OF JUST DISCUSSION, SO WE'RE GOING TO SUMMARIZE SOME INTERESTING POINTS THAT CAME UP THAT WE GOT TO TALK ABOUT. SO WE STARTED IN TERMS OF NEXT GEN SEQUENCING, BOTH THAT AS WELL AS THE INSIGHT PRESENTATIONS TO SOME OF THE DISCUSSION THAT WAS INSPIRED BY THESE PRESENTATIONS WAS -- WHAT CAME UP IN PART WAS A LITTLE BIT OF DISCUSSION OF NON-NEWBORN, BUT CHILDHOOD PERIOD TESTING AND WHETHER WE COULD PLAY A ROLE THERE. I THINK WE'VE TALKED ABOUT THAT BEFORE IN THE COMMITTEE A FEW YEARS AGO BUT THAT CAME UP AGAIN AS WE TALKED ABOUT SOME OF THE THINGS THAT PEOPLE ARE INTERESTED IN THINKING ABOUT AND TESTING FOR AND WHETHER OR NOT, IF NOT IN THE NEWBORN PHASE, IS THERE ANOTHER TIME WE COULD TEST, BUT THEN IS THERE ANOTHER TIME WHERE WE CAN HAVE ALL CHILDREN TESTED IF WE CONSIDER IT A PUBLIC HEALTH ACTIVITY, WHICH IS ALWAYS A CONCERN. SO THAT WAS REALLY INTERESTING ALSO LEADING UP INTO A DISCUSSION OF WHETHER OR NOT THERE WAS A ROLE FOR DRAFTING GUIDELINES FOR LABORATORIES IN TERMS OF USING OLDER DATA, AND HERE TOUCHING ON A FEW THINGS, THE REQUEST FOR SICKLE CELL DATA THAT'S COMING TO A LOT OF LABS, WHEN THEY'RE ASKED ABOUT REALLY OLD DATA, 20 YEARS OLD, HOW LONG WE KEEP THE DATA, TECHNOLOGY, KNOWLEDGE CHANGES, SOME STATES ARE REQUIRED TO KEEP THIS INFORMATION FOR A PERIOD OF TIME TIME, IT CAN CREATE A LIABILITY, RAW DATA 20 YEARS FROM NOW, IS ACTIVITY GOING TO CHANGE, IS OUR KNOWLEDGE GOING TO CHANGE, SO IS THERE SOME -- I KNOW NUN ONE LIKENO ONELIKES TO DISCARD DATA BUT IN SOME WAYS IS IT A LIABILITY TO HAVE IT, IS IT A LIABILITY TO RE-TEST, WOULD THAT BE THE MOST APPROPRIATE THING. AND LASTLY, CARLA BROUGHT UP, 20 YEARS FROM NOW, WE'RE SO DEPENDENT ON INTERPRETING ALL THESE THINGS WITH SOFTWARE, WE MAY HAVE SOFTWARE THAT WON'T TALK TO THE OLD SOFTWARE SO IT MIGHT BE A MOOT POINT ANYWAY. DID YOU HAVE ANY OTHER THOUGHTS? SO SCOTT WAS WITH US AND WE ALSO TALKED BRIEFLY ON HIS PRESENTATION ON HIS PRESENTATION AS WELL AS THE COMMITTEE'S VOTE AS WE CONSIDER CONDITIONS FOR EVIDENCE REVIEW. WE JUST CAME UP WITH THE COST DATA, THAT WAS ANOTHER DISCUSSION ABOUT WHETHER WE NEEDED A MATRIX FOR THE NOMINATION PROCESS. I THINK WE HAVE A LOT OF MATRICES HERE. HERE WAS ANOTHER INTERESTING CONVERSATION, THERE WAS DISCUSSION ON THE FREQUENCY OF CONDITION WE CAN SCREEN FOR, I'M SURE A LOT OF PROGRAMS MUST THINK ABOUT, AND OF COURSE WE ALWAYS HAVE THE DISCUSSION OF HOW IT'S GOING TO BE HARDER TO DO PILOTS WHEN CONDITIONS ARE MORE RARE TO FIND THE ONE CASE OR MORE, BUT THAT SOME OTHERS IN THE GROUP ARGUED THAT WE CAN'T LOWER THE BAR WHEN THAT HAPPENS. SO SOME INTERESTING THOUGHTS, BUT -- AND IT WAS BROUGHT UP THAT LABORATORIES ARE USED TO A PROCESS, YOU KNOW, A CHECKLIST IF YOU WILL, AND BRINGING UP THAT IT'S IMPORTANT TO REMOVE SUBJECTIVITY FROM THE PROCESS. ONCE AGAIN WHAT WAS BROUGHT UP IS NOT TO BYPASS THE FOLLOW-UP TESTING PIECE OF THE WHOLE NEWBORN SCREENING PROCESS. >> SO MULTIPLE TIMES YESTERDAY, ALTHOUGH THE TIMELINESS DISCUSSIONS WERE TODAY, TIMELINESS WAS BROUGHT UP IN SOME OF THE DISCUSSIONS YESTERDAY, SO IT WAS BROUGHT UP DURING MICHELLE'S TALK AND A COMMENT OR ONE OF MICHELLE'S SLIDES SHOWS THAT WHEN -- WHAT THEY'VE HYPOTHESIZED THAT BY ADDING NEXT GEN SEQUENCING, IT WOULD ADD A MINIMUM OF TWO DAYS TO THE PROCESS. SO WE TALKED ABOUT TIMELINESS AND NOT JUST THE IMPACT OF MOLECULAR TESTING BUT PREVIOUSLY WHEN KELLIE AND I PRESENTED FOR THE TIMELINESS WORK GROUP 1.0, WE DIDN'T KNOW WE WERE 1.0 AT THE TIME, BUT DURING THE PRESENTATION OF THE RECOMMENDATIONS OF THE WORK GROUP, WE HAD SAID THAT YOU REALLY NEED TO USE CAUTION IN THESE RECOMMENDATIONS BECAUSE YOU DON'T WANT TO DO MORE HARM THAN GOOD BY FOCUSING ON MEETING THE GOALS, YOU MAY ACTUALLY SAY THEN WE DON'T NEED TO DO THE SECOND TIER TESTING, THEN YOU HAVE INCREASE IN FALSE POSITIVES. WE TALKED A LITTLE BIT ABOUT THAT ISSUE EARLIER TODAY. AND SO THAT'S REALLY WHERE OUR DISCUSSION LED, SO WE THOUGHT WE MAY NEED TO REVISIT THE RECOMMENDATIONS AS WE GET MORE DATA, AND I THINK THAT WAS MENTIONED EARLIER TODAY, THAT WE NEED TO BE ABLE TO CAPTURE THE IMPACT OF THAT SECOND TIER OR ADDITIONAL TESTING THAT'S PERFORMED AND HOW THAT MAY IMPACT THE ACTUAL TIME IT TAKES TO GET TO A RESULT. WE ALSO TALKED ABOUT -- I'LL CALL IT REGIONALIZATION. SO CURRENTLY THERE ARE SEVERAL REGIONAL PROGRAMS THAT DO NEWBORN SCREENING AND THAT'S BEEN CHOSEN BY THE STATES AND IT'S WORKING VERY WELL WHERE THERE'S ONE LAB DOING TESTING FOR MULTIPLE STATES. I THINK MAE MENTIONED YESTERDAY AS THEY LOOK AT NEXT GEN SEQUENCING, IN A STATE THE SIZE OF WISCONSIN, IT'S VERY EXPENSIVE, AND SO THEY MAY HAVE TO BATCH -- THEY MAY NOT BE ABLE TO DO NEXT DP. E GEN SEQUENCING EVERY DAY, THEY NEED TO FIND A MORE ECONOMICAL WAY OF DOING IT. SO TESTING FOR ADDITIONAL STATES AS WELL, IT ACTUALLY BECOMES MORE ECONOMICAL AND ACTUALLY IMPROVES THEIR TIMELINESS, AND SO THERE'S SOME CONDITIONS AS THERE'S A HIGHER AND HIGHER UP TAKE ON MORE MOLECULAR TECHNOLOGIES THAT THAT MIGHT BE BENEFICIAL FOR SOME STATES. AND THEN FINALLY, KIND OF GETTING TO THE POINT OF TIMELINESS, SO WHEN WE PUT TOGETHER THE RECOMMENDATIONS, THOSE RECOMMENDATIONS REALLY FOCUS AT THE END OF A LAB RESULT, SO GETTING A REPORT OUT, IT DOESN'T GO TO DIAGNOSIS. SO SOME OF THE SECOND TIER TESTING THAT'S DONE MAY ACTUALLY DECREASE THE TIME TO DIAGNOSIS, AND ULTIMATELY THAT'S WHAT MATTERS. SO WE SHOULDN'T BE BOUND BY OUR RECOMMENDATIONS AND MAYBE AT SOME POINT WE CAN FIGURE OUT WHAT IS A PERCENTAGE THAT WE SHOULD ACTUALLY BE MEETING SO THAT WE'RE NOT DOING MORE HARM THAN GOOD. WE ALSO HAD CONTINUED DISCUSSION ABOUT JUST SOME OF THE PREANALYTIC ISSUES THAT WE'RE STILL SEEING, SO THE TIMELINESS RECOMMENDATION CAME OUT FEBRUARY OF '15, AND THERE HAS BEEN A HUGE IMFA CYS EMPHASIS THROUGHOUT THE STATE, AND YOU'VE HEARD TALKS ABOUT THE COIN PROJECT AND 360 AND SOME OF THE PROGRESS THAT'S BEEN MADE, BUT YOU ALSO SAW DATA THAT SHOW THAT DESPITE ALL THOSE EFFORTS, IT'S STILL VERY HARD TO ACHIEVE THE RECOMMENDATIONS, AND ONE OF THE ISSUES, NEWBORN SCREENING PROGRAMS OFTEN TRY TO TAKE ON THE ENTIRE ROLE OF THE NEWBORN SCREENING SYSTEM, YET THEY ONLY REALLY HAVE IMPACT ON THE THINGS THAT THEY CAN TOUCH DAILY, AND SO WE NEED TO FIGURE OUT HOW TO ACHIEVE THOSE BETTER PARTNERSHIPS WITH THE HOSPITALS AND BIRTHING CENTERS AND MIDWIVES WHO ARE COLLECTING THOSE SPECIMENS, AND THEN THAT'S A VERY SHORT WINDOW IN A CHILD'S LIFE. THEN YOU HAVE THE ENTIRE SPECTRUM AFTER THAT. SO THE FOLLOW-UP AND DIAGNOSIS AND TREATMENT OF THOSE LONG TERM FOLLOW-UPS, SO THERE ARE ISSUES WITH TURNOVER AT HOSPITALS, SO A PROGRAM MAY BE ABLE TO GO IN AND IN A SMALL STATE MAY BE ABLE TO EDUCATE AT EVERY SINGLE FACILITY EVERY YEAR, BUT THERE'S STILL GOING TO BE NEW STAFF EVERY TIME THEY GO IN. SO WE NEED TO FIGURE OUT A WAY TO MAINTAIN THE IMPROVEMENT THAT HAPPENS AT HOSPITALS AND BIRTHING CENTERS AND WHOEVER COLLECTS THE SPECIMENS. WE TALKED ABOUT MEETING TO FIND A CHAMPION. HOW DO YOU FIND THE PERSON THAT'S GOING TO BE ABLE TO ENGAGE AND CONTINUE THAT ENGAGEMENT AND BE ABLE TO NOT JUST TRAIN ONE GROUP BUT HAVE A TRAIN THE TRAINER WITHIN EACH FACILITY. WE TALKED ABOUT COURIER, IT'S EXPENSIVE, AND EVEN IF YOU HAVE A COURIER SYSTEM IN PLACE, YOU'RE ABLE TO PAY FOR THAT IN A PROGRAM, THERE'S STILL SOME ISSUES WITH COURIERS. ONE OF THE STATES TALKED ABOUT HOW THEY HAVE A PERSON DEDICATED TO BASICALLY WATCHING THE SHIPMENTS THAT ARE GOING TO BE COMING IN COMPARING THAT WITH WHAT'S ACTUALLY COME IN, TRYING TO PINPOINT AND FIGURE OUT WHERE THOSE SHIPMENTS ARE THAT ARE LOST SOMEWHERE IN A HUB. WE TRIED WITH TIMELINESS 2.0, WE HAD A CALL WITH JOINT COMMISSION BUT THERE'S STILL A NEED FOR A ROLE WITH THE JOINT COMMISSION SO WE NEED TO TRY TO FIGURE OUT HOW WE GET IN THERE FURTHER AND HAVE SOME FURTHER CONVERSATIONS. THEN WE ALSO TALKED ABOUT TRANSPARENCY IN THE TIMELINESS DATA AVAILABLE TO THE PUBLIC, SO IN SOME STATES, IT'S BEEN ABLE TO BE PUBLISHED ON A WEBSITE AND IT'S TRANSPARENT, AND IN OTHER AREAS, THAT'S STILL NOT AVAILABLE. IF ANYONE HAS ANY QUESTIONS, WE'LL ATTEMPT TO TALK YOU THROUGH OUR FREEFORM DISCUSSION WE HAD LISTED. >> THANK YOU BOTH VERY MUCH. ANY QUESTIONS OR COMMENTS RELATED TO THE PRESENTATION? CLEARLY A LOT OF WORK GOING ON IN THIS WORK GROUP AS WELL, SO THANK YOU VERY MUCH. APPRECIATE IT. SO WE ARE SCHEDULED TO ADJOURN AT 2:15, BUT THE LAST ITEM IS IF THERE'S ANY NEW BUSINESS I TO COME BEFORE THE COMMITTEE AND I'LL CERTAINLY ENTERTAIN ANY ITEMS THAT PEOPLE WANT TO BRING FORWARD AS POTENTIAL NEW BUSINESS FOR THE COMMITTEE. I THINK WE'VE HEARD A LOT OF THINGS THAT ARE ALREADY GOING TO BE INCORPORATED INTO NEW BUSINESS GOING FORWARD, BUT ARE THERE ANY QUESTIONS, COMMENTS COMING? OKAY. WELL, THEN BASED ON THAT, I THINK THIS HAS BEEN A VERY INFORMATIVE MEETING. I THINK IT'S VERY CLEAR THAT OUR NEW MEMBERS ARE ALL RIGHT INTEGRATED INTO THE COMMITTEE AND HAVE ALREADY PLAYED A ROLE IN MAKING THINGS HAPPEN, SO I APPRECIATE THE WORK OF THE ENTIRE COMMITTEE AS WELL AS HRSA WITH GETTING THINGS ORGANIZED AND DEBBIE FOR HER ROLE IN MAKING THIS ALL HAPPEN AND THE ORGANIZATIONAL REPRESENTATIVES AND EVERYBODY ELSE WHO'S CONTRIBUTED TO THIS MEETING. SO WITH THAT, I WANT TO THANK YOU ALL, AND WE LOOK FORWARD TO OUR TELECONFERENCE MEETING IN NOVEMBER. THANK YOU. I'LL CONCLUDE.