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>>Anabela Marcal:
Hello, everyone.

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My name is Anabela Marcal

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and I'm the European
Medicines Agency

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liaison official to the U.S.
Food and Drug Administration.

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I've been at EMA for 24 years,
and I had different roles

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including in clinical
trials, pharmacovigilance,

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and inspections.

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In this lecture, I'll
give you a broad overview

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of the E.U. regulatory
framework for medicines,

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in particular, support,
research, and development.

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I'll start this lecture
by giving an introduction

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to the European Medicines Agency
and the E.U. Regulatory Network.

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And then we'll focus
on the role of the EMA

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to support medicine's
development

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and the tools that are
available for that,

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in particular,
Innovation Task Force,

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scientific advice, pediatric
investigation plans,

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orphan designation,
the PRIME scheme,

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and support to small
and medium enterprises.

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Setting the scene. About
European Medicines Agency.

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The European Medicines Agency

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serves a market of
447 million people

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living in the European Union.

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EMA protects public
and animal health

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in 27-member states plus Norway,
Iceland, Liechtenstein,

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by ensuring that all medicines
in the E.U. market

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are safe, effective,
and of high quality.

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The medicines EMA recommends for
marketing authorization account

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for 21.5 percent of
global pharmaceutical sales.

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The European Medicines Agency

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is a decentralized agency
of the European Union,

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established in 1995,

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initially located in London
and currently in Amsterdam.

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EMA is responsible

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for the scientific
evaluation, supervision,

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and monitoring of human
and veterinary medicines

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for use in the European Union.

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EMA brings together the
best scientific expertise

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on medicines from
across all of the E.U.

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And in fact, even if we are
only 800 staff members,

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we have, at our disposal,

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a network of over
4,000 scientific experts

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who are made
available to the EMA

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by the E.U. member states,
National Competent Authorities.

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The agency works with
seven scientific committees

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who evaluate medicines
along their life cycle

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from early stage of development,
through marketing authorization,

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to the safety monitoring
once they are on the market.

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These seven scientific
committees include CHMP,

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or the Committee for
Human Medicinal Products.

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This is the main
committee responsible

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to provide scientific
recommendations on the approval,

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changes, suspension, withdrawals
of medicines for human use.

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Then we have the Committee for
Veterinary Medicinal Products

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that is the equivalent of CHMP,

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but for veterinary
medicines. We have COMP --

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that is the Committee on
Orphan Medicinal Products --

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whose main responsibility

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is to give the orphan
designation status.

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We have the HMPC, the
Herbal Medicines Committee,

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the Pediatric
Committee, or PDCO,

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that looks at the Pediatric
Investigation Plans.

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We have CAT, or the Committee
on Advanced Therapies,

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looking at ATMPs, and we
have the Pharmacovigilance

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Risk Assessment Committee.

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The committees in general
are composed by one member

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plus one alternate
by each member state.

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In addition, in some committees,

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we have additional members
with scientific expertise.

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We have patients and
healthcare representatives

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as well in some
of the committees.

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Decisions on these committees,
like scientific decisions,

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are taken by majority vote.

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The committees are supported by
the so-called working parties,

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which are smaller groups with
expertise on a specific area.

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For instance, the Blood
Products Working Party,

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Biosimilars Working Party,
Pharmacokinetics Working Party.

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The European Medicines
Agency is part of the E.U.

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Medicines Regulatory Network,

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which is made up of the National
Medicines Regulatory

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Authorities from the 27
E.U. member states

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and the European Commission.

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As I mentioned before,
the Network is supported

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by a pool of 4,000 experts
from across Europe.

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And we all use
the same standards

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and they have the same
regulations in place.

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I'm going to give you
some understanding of,

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what is the role of the EMA
in supporting research

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and development of medicines,

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i.e., before a Marketing
Authorization Application

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is submitted?

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EMA stimulates research
and innovation

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in pharmaceutical sector as part
of its public health mandate.

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How are we doing it?

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We have EMA's
Innovation Task Force

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that provides a forum
for early dialogue

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with applicants,
in particular,

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small and medium enterprises
and academic sponsors,

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to proactively identify
scientific, legal,

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and regulatory issues

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related to emerging
therapies and technologies.

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EMA provides support
to early stage

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of medicine development
through a number of tools,

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including pediatric
procedures, scientific advice,

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qualification of
novel methodologies,

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support to small
and medium enterprises,

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and orphan designation.
Companies can also consult EMA

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to determine whether a
medicine they are developing

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is classified as an advanced
therapy medicinal product.

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EMA provides
regulatory, financial,

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and administrative assistance

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to small and medium-sized
enterprises as well.

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Let's explore a little bit
more each of these tools

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available to applicants and
sponsors during early stage

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of development of
medicinal products.

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Innovation Task Force.

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Innovation Task Force
has a double remit.

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In one side, it provides advice
related to specific products,

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technologies, or methodologies.

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This is a
multidisciplinary group,

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including scientific,
regulatory,

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and legal competencies.

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And it was set up to ensure
coordination across the agency

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and to provide a forum
for early dialogue

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with applicants on
innovative aspects

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on medicines development.

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The companies can come for the
so-called ITF briefing meetings.

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That is a forum for early
dialogue with the applicants

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on their specific product
or methodology or technology.

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These are very
informal meetings,

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and the companies
get clarifications

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on some of their issues

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or, for instance,
lack of guideline

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for a specific technology
or emerging therapy.

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But in the other
side, the regulator,

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EMA, also becomes aware

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of what is being developed
in terms of innovation.

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This group is also responsible
a [sic] cross development,

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meaning addressing really the
impact of emerging therapies

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and technologies on
the regulatory system.

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So, assists knowledge
exchange on innovative

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strategies involving
the Regulatory Network.

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And it also collaborates with
the National Innovation Office

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through the E.U.
Innovation Network,

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which aims at facilitating

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the development of innovative
medicines by addressing gaps

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in early regulatory
support to innovation.

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Another tool available
to companies,

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it's the so-called
business pipeline meetings.

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The business pipeline meetings
are unique opportunity

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[sic] to establish and
--confidential

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and mutually
beneficial discussion

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on a regulatory pipeline
from a company.

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For example, what are
the marketing authorization

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applications coming
in the future?

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Any pediatric
investigation plans,

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scientific advice,
orphan designation,

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extensions of existing
authorizations.

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The primary goal of these
meetings is to identify,

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at an early stage,
any issues impacting

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the progress of the
product portfolio

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and to effectively
anticipate expertise

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that will be needed,
any guideline discussions,

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changes in technology,
and drug development.

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The focus is pretty much

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on general aspects
of product portfolio

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while specific scientific
or regulatory issues

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can be discussed at
scientific advice stage,

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pre-submitting meetings,
pediatric investigation plans,

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or orphan designation.

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Scientific advice.

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Scientific advice is
about advice to a sponsor

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on any scientific questions
or technical questions

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on the development
of a medicine.

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And these can be questions
about the manufacturing,

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the nonclinical studies,
the clinical trials,

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how to study the product
in specific populations,

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for instance, for rare
diseases or pediatric use.

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A sponsor can come at any point
in time of the development,

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irrespective of eligibility
to the centralized procedure.

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I.e., not all
medicines are eligible

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to come to the
centralized procedure

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or the procedure for
authorization of medicines

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that is coordinated by the
European Medicines Agency,

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but scientific advice,
any company for any product

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can come and ask
scientific advice.

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Scientific advice
is perspective,

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meaning it doesn't focus
on development strategies.

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It's not the pre-assessment
of an application.

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It is possible to have
parallel scientific advice

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with health technology
assessment bodies.

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And it's also possible to have
parallel scientific advice

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with the Food and
Drug Administration.

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Scientific advice is voluntary.

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It's not a mandatory procedure
and it's not legally bidding.

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Also, it's not a guarantee
for marketing authorization.

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But we know that complying
with scientific advice

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increases the chance of
receiving a positive outcome

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at the time of marketing
authorization application.

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Some analysis that we
did some years ago

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showed that 15 percent
of all products

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that came to scientific advice

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and then requested the Marketing
Authorization Application

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through the centralized
procedure

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had a negative opinion compared
to 25 percent for products

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that didn't come for
scientific advice.

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Of course, there are
a lot of variabilities

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because the fact that a company
comes for scientific advice,

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it doesn't mean that the company

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will follow the
advice being given.

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So, this can be also a factor
that then influences

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at the time of marketing
authorization application.

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When is scientific advice
procedure most useful?

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Scientific advice is most useful
for innovative medicines,

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mainly when it looks like there
is insufficient or lack of

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E.U. guidelines
or guidance documents,

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or if the developer chooses
to deviate from the existing

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scientific guidelines
in its development plan,

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or if the medicine developer
has limited knowledge

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about the E.U. medicines
regulation,

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such as some academic
groups or micro,

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small, and medium
sized enterprises.

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One variability
of scientific advice

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is the so-called
protocol assistance.

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Protocol assistance is
scientific advice available

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for developers of designated
orphan medicines.

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And these scientific advice or
these specific scientific advice

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for developers of
orphan medicines

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can also address questions
related to the criteria

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for authorization
of an orphan medicine,

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such as the demonstration
of significant benefit

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or similarity

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or clinical superiority
over other medicines.

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How long does it take
for a scientific advice?

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Which procedural steps
does a company need to follow

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to have its scientific advice?

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We always recommend what we
call the planning phase.

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And the planning phase,
it's like 20 to 45 days

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before submitting the request
for scientific advice.

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So, the sponsor notifies the
EMA that intends to submit

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a request for scientific advice.

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At that point in
time, we will appoint

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the Scientific Advice
Working Party coordinators.

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So, these are two members
from the Scientific Advice

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Working Party that
will take the lead

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in the assessment of
the scientific advice.

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And then there is the formal
request that will be validated

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by the EMA.

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Once validated, then we
start evaluation phase.

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That is a 40 or
70 day procedure.

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And during this
evaluation phase,

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00:15:40,739 --> 00:15:44,143
the coordinators form assessment
teams and prepare the report.

253
00:15:45,177 --> 00:15:47,579
After the report is prepared,
there will be a discussion

254
00:15:47,646 --> 00:15:49,682
at the Scientific Advice
Working Party.

255
00:15:50,516 --> 00:15:53,319
If requested, and if
considered needed,

256
00:15:53,385 --> 00:15:56,488
there is possibility for
a meeting with the developer.

257
00:15:56,555 --> 00:15:59,158
The Scientific Advice
Working Party can also

258
00:15:59,224 --> 00:16:02,628
consult experts or
patients' representatives.

259
00:16:03,262 --> 00:16:06,265
And finally, the Scientific
Advice Working Party

260
00:16:06,332 --> 00:16:09,802
will provide the
recommendation to the CHMP

261
00:16:09,868 --> 00:16:13,605
who will issue a final
response letter to the sponsor

262
00:16:13,672 --> 00:16:16,675
on the specific request
for scientific advice.

263
00:16:20,813 --> 00:16:22,648
As I mentioned in
the previous slide,

264
00:16:22,715 --> 00:16:25,517
there is a possibility
for a sponsor

265
00:16:25,584 --> 00:16:29,521
to ask for parallel
EMA-FDA scientific advice.

266
00:16:30,322 --> 00:16:33,525
Again, this is voluntary
at the request of the sponsor.

267
00:16:34,326 --> 00:16:38,931
It's usually accepted for
priority products or a product

268
00:16:38,997 --> 00:16:42,067
with significant
development challenges,

269
00:16:42,134 --> 00:16:45,838
and especially if limited
clinical trial opportunities,

270
00:16:45,904 --> 00:16:47,639
for instance, such
as rare disease.

271
00:16:48,607 --> 00:16:53,045
And what happens here is that
the questions are put --

272
00:16:53,112 --> 00:16:57,716
on product development
are put to both EMA and FDA.

273
00:16:58,283 --> 00:17:00,185
So, it's a parallel procedure

274
00:17:00,252 --> 00:17:02,554
and they are submitted
at the same time.

275
00:17:02,621 --> 00:17:05,891
And these allows discussions
between EMA and FDA

276
00:17:05,958 --> 00:17:07,826
on the questions
put by the sponsor,

277
00:17:07,893 --> 00:17:11,029
and then a joint discussion
with the sponsor,

278
00:17:11,096 --> 00:17:12,765
the so-called
trilateral meeting.

279
00:17:14,433 --> 00:17:17,469
This is a good way of supporting
global development plans

280
00:17:17,536 --> 00:17:20,639
because EMA and FDA will
discuss on the questions

281
00:17:20,706 --> 00:17:22,408
from the sponsor,

282
00:17:22,474 --> 00:17:26,145
and the idea is to achieve
some convergence.

283
00:17:26,745 --> 00:17:29,548
But even if convergence
will not be achieved

284
00:17:29,615 --> 00:17:34,620
because of the regulatory or
legal framework from each side,

285
00:17:35,220 --> 00:17:38,323
at least there is clear
dialogue between the EMA,

286
00:17:38,390 --> 00:17:39,725
the FDA, and the sponsor.

287
00:17:39,792 --> 00:17:43,162
And the sponsor will
better understand in case

288
00:17:43,228 --> 00:17:45,564
there will be
different requirements

289
00:17:45,631 --> 00:17:49,301
from the FDA and EMA for
the specific development.

290
00:17:50,235 --> 00:17:53,439
At the end of this parallel
scientific advice procedure,

291
00:17:53,505 --> 00:17:56,408
the agencies issue
their own responses

292
00:17:56,475 --> 00:17:58,410
to sponsor questions in line

293
00:17:58,477 --> 00:18:02,181
with the usual procedure
of each agency.

294
00:18:06,885 --> 00:18:10,122
Another consideration
during the pre --

295
00:18:10,189 --> 00:18:13,091
or during the development
phase of a medicine,

296
00:18:13,826 --> 00:18:17,229
it's regarding the Pediatric
Investigation Plan

297
00:18:18,130 --> 00:18:20,032
or the so-called PIP.

298
00:18:20,098 --> 00:18:23,402
The Pediatric Investigation
Plan is a development plan

299
00:18:23,936 --> 00:18:27,506
to ensure that necessary data
are obtained in the conditions

300
00:18:27,573 --> 00:18:31,176
in which a medicinal product may
be authorized to treat children.

301
00:18:33,312 --> 00:18:36,548
Companies can come for early
pediatric interaction meetings.

302
00:18:37,416 --> 00:18:41,186
This PIP should be submitted
around end of Phase I studies,

303
00:18:41,253 --> 00:18:43,956
start of Phase
2 proof of concept.

304
00:18:45,324 --> 00:18:48,594
It should outline the
timing and measures,

305
00:18:48,660 --> 00:18:51,430
like all the studies,
formulations being developed,

306
00:18:51,997 --> 00:18:55,734
to demonstrate quality, safety,
and efficacy in children.

307
00:18:57,102 --> 00:19:00,806
It is possible to ask
for a deferral or waiver

308
00:19:01,573 --> 00:19:03,542
of a pediatric
investigation plan.

309
00:19:04,710 --> 00:19:07,246
And modifications to
the plan are possible

310
00:19:07,312 --> 00:19:09,848
through the development
of the product.

311
00:19:10,749 --> 00:19:13,485
This PIP is binding
on the company.

312
00:19:14,119 --> 00:19:15,387
And in the E.U.,

313
00:19:15,454 --> 00:19:18,690
when you come for a marketing
authorization application,

314
00:19:18,757 --> 00:19:24,229
you need to have an adopted
pediatric investigation plan.

315
00:19:24,763 --> 00:19:27,499
Or if you don't have an adopted
pediatric investigation plan,

316
00:19:27,566 --> 00:19:32,938
you should have agreed,
adopted, deferral, or waiver

317
00:19:33,005 --> 00:19:34,573
by the Pediatric Committee.

318
00:19:35,374 --> 00:19:38,443
And at the time of the marketing
authorization application,

319
00:19:38,510 --> 00:19:40,479
there will be a
compliance check.

320
00:19:40,546 --> 00:19:44,716
So, even if you have agreed
pediatric investigation plan,

321
00:19:44,783 --> 00:19:47,553
there will be a
compliance check to check

322
00:19:47,619 --> 00:19:50,689
where you are with the studies

323
00:19:50,756 --> 00:19:52,925
and with the measures
that were agreed

324
00:19:54,026 --> 00:19:56,361
in that pediatric
investigation plan.

325
00:20:02,534 --> 00:20:04,136
Orphan designation.

326
00:20:04,836 --> 00:20:07,639
The EMA is responsible
for reviewing applications

327
00:20:07,706 --> 00:20:09,975
from sponsors
for orphan designation.

328
00:20:11,276 --> 00:20:13,445
And orphan designation
is in fact a --

329
00:20:13,512 --> 00:20:16,848
as a basis for the access
to related incentives

330
00:20:16,915 --> 00:20:18,584
such as development support,

331
00:20:18,650 --> 00:20:21,153
fee reduction for a
number of activities,

332
00:20:21,219 --> 00:20:23,789
and 10 years of
market exclusivity

333
00:20:23,855 --> 00:20:25,824
at the time of marketing
authorization.

334
00:20:27,793 --> 00:20:29,661
To qualify for
orphan designation,

335
00:20:29,728 --> 00:20:32,130
a medicine must meet
a number of criteria.

336
00:20:33,966 --> 00:20:37,836
It should be for a condition
for which the prevalence

337
00:20:37,903 --> 00:20:40,572
should not be more
than five in 10,000.

338
00:20:41,740 --> 00:20:45,677
Or, it must be unlikely
that marketing of the medicine

339
00:20:45,744 --> 00:20:47,546
would generate
sufficient returns

340
00:20:47,613 --> 00:20:51,049
to justify the investment
needed for its development.

341
00:20:52,484 --> 00:20:55,454
It must be intended for
the treatment, prevention of --

342
00:20:55,520 --> 00:20:58,857
or diagnosis of a disease
that is life-threatening

343
00:20:58,924 --> 00:21:01,026
or chronically debilitating.

344
00:21:02,728 --> 00:21:05,797
And no satisfactory
method of diagnosis,

345
00:21:05,864 --> 00:21:09,434
prevention, or treatment of
the condition can be authorized,

346
00:21:09,501 --> 00:21:12,004
or if such a method exists,

347
00:21:12,070 --> 00:21:15,874
the medicine must be
of significant benefit

348
00:21:15,941 --> 00:21:18,110
for those affected
by the condition.

349
00:21:20,245 --> 00:21:22,581
Scientific advice
for orphan medicines

350
00:21:22,648 --> 00:21:26,284
is called protocol assistance,
and as I mentioned before,

351
00:21:26,985 --> 00:21:29,388
also covers questions
on the type of studies

352
00:21:29,454 --> 00:21:30,656
needed to demonstrate

353
00:21:30,722 --> 00:21:33,091
the significant benefit
of the medicine.

354
00:21:35,027 --> 00:21:38,363
Assessment by the Committee
for Orphan Medicinal Products

355
00:21:39,064 --> 00:21:41,733
is followed by
the European Commission decision

356
00:21:41,800 --> 00:21:45,237
on designating a
product as orphan --

357
00:21:46,805 --> 00:21:50,776
with orphan designation.
It can be granted at any stage

358
00:21:50,842 --> 00:21:53,545
before marketing authorization
application submission.

359
00:21:54,513 --> 00:21:56,581
Maintenance of the
orphan designation

360
00:21:56,648 --> 00:21:58,050
needs to be confirmed

361
00:21:58,116 --> 00:22:00,986
before the granting of the
marketing authorization.

362
00:22:01,053 --> 00:22:03,855
And all designations
are included in the Community

363
00:22:03,922 --> 00:22:07,359
register of orphan medicinal
products for human use.

364
00:22:13,231 --> 00:22:16,668
PRIME, or the so-called
Priority Medicines.

365
00:22:16,735 --> 00:22:18,270
It's a scheme
that was launched

366
00:22:18,336 --> 00:22:21,873
by the European
Medicines Agency in 2016

367
00:22:22,674 --> 00:22:25,644
and it aims to foster
development of medicines

368
00:22:25,711 --> 00:22:27,612
with major public
health interests

369
00:22:28,280 --> 00:22:29,881
and unmet medical need.

370
00:22:31,383 --> 00:22:35,754
What are the advantage [sic]
of a company coming for PRIME?

371
00:22:37,856 --> 00:22:40,959
If PRIME status is granted,

372
00:22:41,026 --> 00:22:43,228
there will be an
increased support

373
00:22:43,295 --> 00:22:45,197
during development
to the company.

374
00:22:46,031 --> 00:22:49,067
There will be an early
appointment of rapporteurs.

375
00:22:49,134 --> 00:22:53,105
Rapporteurs are the members
of the committees

376
00:22:53,171 --> 00:22:54,840
that take the lead
on the assessment

377
00:22:54,906 --> 00:22:58,977
of a certain medicinal product.
In general, for CHMP --

378
00:22:59,044 --> 00:23:01,780
for the Committee of
Human Medicinal Products,

379
00:23:01,847 --> 00:23:03,014
there is a rapporteur

380
00:23:03,081 --> 00:23:05,917
and a co-rapporteur
appointed for each product.

381
00:23:07,152 --> 00:23:10,055
But in addition to the CHMP
rapporteurs,

382
00:23:10,122 --> 00:23:12,691
there could be also appointment
of a CAT,

383
00:23:12,758 --> 00:23:14,159
Committee for Advanced Therapies

384
00:23:14,226 --> 00:23:17,295
rapporteur if the product
is an ATMP.

385
00:23:22,067 --> 00:23:27,038
Once the PRIME status
is given -- is granted,

386
00:23:27,706 --> 00:23:30,108
a kickoff meeting
will be organized

387
00:23:30,175 --> 00:23:32,644
with the EMA coordinator

388
00:23:32,711 --> 00:23:34,980
and the rapporteurs
that have been appointed.

389
00:23:35,680 --> 00:23:38,049
In general, that kickoff meeting
happens three,

390
00:23:38,116 --> 00:23:42,087
four months after the granting
of the PRIME status.

391
00:23:44,122 --> 00:23:46,758
There will be also --
later on in the development,

392
00:23:46,825 --> 00:23:50,295
there will be a submission
readiness meeting.

393
00:23:50,362 --> 00:23:54,766
And this happens, in general,
around one year

394
00:23:54,833 --> 00:23:56,535
before the submission

395
00:23:56,601 --> 00:23:58,737
of the marketing
authorization application.

396
00:23:59,371 --> 00:24:02,774
Other [sic] advantage
of the PRIME designation

397
00:24:02,841 --> 00:24:04,810
is that there
will be possibility

398
00:24:04,876 --> 00:24:08,380
for scientific advice
at key development milestones,

399
00:24:09,114 --> 00:24:12,217
and under certain criteria,
there will be also possibility

400
00:24:12,284 --> 00:24:15,053
for expedited follow-up
scientific advice,

401
00:24:15,120 --> 00:24:17,055
which shorten timelines.

402
00:24:18,857 --> 00:24:20,725
This PRIME scheme tries
to make better

403
00:24:20,792 --> 00:24:23,929
use of existing regulatory
and procedural tools.

404
00:24:25,063 --> 00:24:28,166
It enables, also, accelerated
assessment of medicines.

405
00:24:28,733 --> 00:24:32,537
It doesn't mean that a medicine
that is under PRIME scheme

406
00:24:32,604 --> 00:24:35,106
will be granted
accelerated assessment,

407
00:24:35,173 --> 00:24:40,545
but there is an increased
potential to get it.

408
00:24:43,014 --> 00:24:46,184
Recently, we have reported
on five years' experience

409
00:24:46,251 --> 00:24:47,719
of PRIME scheme.

410
00:24:47,786 --> 00:24:50,655
And we could conclude that PRIME
helped patients benefit

411
00:24:50,722 --> 00:24:53,625
from new treatment options
since its launch.

412
00:24:54,259 --> 00:24:56,361
In fact, there was
a reduced time

413
00:24:56,428 --> 00:24:59,164
to marketing authorization
for PRIME products.

414
00:24:59,998 --> 00:25:02,634
This consistent reduction
was linked

415
00:25:02,701 --> 00:25:06,872
to a reduction
of the clock stop duration.

416
00:25:07,739 --> 00:25:10,008
The clock stops, it's the time
that companies take,

417
00:25:10,075 --> 00:25:11,343
during the marketing

418
00:25:11,409 --> 00:25:14,713
authorization application
assessment,

419
00:25:14,779 --> 00:25:17,349
to respond to the questions
from the Committee

420
00:25:17,415 --> 00:25:19,084
for Human Medicinal Products.

421
00:25:20,118 --> 00:25:23,922
This reduction in clock stop
was more pronounced

422
00:25:23,989 --> 00:25:28,326
for small and medium enterprises
and for more complex products

423
00:25:28,393 --> 00:25:30,829
like advanced therapy
medicinal products.

424
00:25:31,663 --> 00:25:34,399
Accelerated assessment
was also facilitated.

425
00:25:35,066 --> 00:25:37,335
Accelerated assessment
is a tool

426
00:25:37,402 --> 00:25:40,138
that companies can request
at the time of submission

427
00:25:40,205 --> 00:25:42,374
and marketing
authorization application,

428
00:25:42,440 --> 00:25:45,043
requesting a shorter timeline

429
00:25:45,110 --> 00:25:47,312
for the evaluation
of the medicinal product.

430
00:25:49,080 --> 00:25:53,385
The fact that the product
was under the PRIME scheme

431
00:25:53,451 --> 00:25:57,022
facilitated the granted
of the accelerated assessment,

432
00:25:57,088 --> 00:26:01,159
as well as keeping it throughout
the assessment procedure.

433
00:26:03,228 --> 00:26:05,397
Companies who were
within the PRIME scheme

434
00:26:05,463 --> 00:26:08,333
also benefited from
enhanced regulatory support

435
00:26:09,000 --> 00:26:14,539
and from scientific advice
at several points in time.

436
00:26:15,340 --> 00:26:17,108
And it's concluded
that the compliance

437
00:26:17,175 --> 00:26:20,679
with scientific advice
led to wider success rate

438
00:26:20,745 --> 00:26:23,915
of marketing authorization
applications being granted.

439
00:26:25,083 --> 00:26:30,021
In general, PRIME was given
for a broad range

440
00:26:30,088 --> 00:26:31,957
of unmet medical needs,

441
00:26:32,023 --> 00:26:35,393
in particular, oncology
and neurology products.

442
00:26:39,197 --> 00:26:42,701
And finally, I would like
to refer to the support

443
00:26:42,767 --> 00:26:46,237
that EMA gives to small
and medium-sized enterprises.

444
00:26:47,305 --> 00:26:50,408
EMA supports SMEs
to promote innovation

445
00:26:50,475 --> 00:26:52,944
and the development
of an authorization

446
00:26:53,011 --> 00:26:55,547
of new human
and veterinary medicines.

447
00:26:55,613 --> 00:26:59,217
There are specific criteria
to qualify for an SME.

448
00:26:59,884 --> 00:27:02,687
And for that, the company
must be legally established

449
00:27:02,754 --> 00:27:08,326
in the E.U. or EEA area,
with less than 250 employees

450
00:27:08,393 --> 00:27:11,463
and a turnover of not
more than 50 million

451
00:27:11,529 --> 00:27:14,866
or a balance sheet total
of not more than 43 million.

452
00:27:16,234 --> 00:27:19,938
EMA supports these SMEs
through its SME Office.

453
00:27:20,005 --> 00:27:24,609
So, we have a dedicated office
just to support these companies.

454
00:27:25,310 --> 00:27:27,746
And this office
provides regulatory,

455
00:27:27,812 --> 00:27:29,981
administrative,
and procedural assistance.

456
00:27:31,116 --> 00:27:33,885
There are also financial
incentives for EMA procedures,

457
00:27:33,952 --> 00:27:37,389
for instance, such as scientific
advice with reduced fee.

458
00:27:38,156 --> 00:27:41,659
There is support for translation
of product information

459
00:27:41,726 --> 00:27:44,596
from initial marketing
authorization applications

460
00:27:44,662 --> 00:27:46,431
because once
the marketing authorization

461
00:27:46,498 --> 00:27:48,299
is granted in the E.U.,

462
00:27:48,366 --> 00:27:50,435
the product information
needs to be translated

463
00:27:50,502 --> 00:27:53,271
in the 24 language
of the EU.

464
00:27:54,406 --> 00:27:57,275
And there is also
a lot of communication

465
00:27:57,342 --> 00:28:00,945
and engagement activities,
such as info days, newsletters,

466
00:28:01,012 --> 00:28:03,515
and target mailings
for these companies.

467
00:28:06,284 --> 00:28:10,088
So, this was a brief summary
of the tools

468
00:28:10,155 --> 00:28:14,025
that are available from the EMA
to support research

469
00:28:14,092 --> 00:28:16,961
and development
in advance of submission

470
00:28:17,028 --> 00:28:19,097
of a marketing
authorization application.

471
00:28:20,265 --> 00:28:22,267
Let's test your knowledge now.

472
00:28:23,101 --> 00:28:25,437
So, a few questions,
true or false.

473
00:28:26,271 --> 00:28:28,807
The E.U. Regulatory Network
for medicines

474
00:28:29,374 --> 00:28:31,309
includes
the European Commission,

475
00:28:31,376 --> 00:28:33,211
the European
Medicines Agency,

476
00:28:33,278 --> 00:28:35,780
and the E.U. member states
Regulatory Authorities.

477
00:28:37,715 --> 00:28:40,251
This is true and
we work together.

478
00:28:40,318 --> 00:28:43,855
It's because of that, we call it
the E.U. Regulatory Network.

479
00:28:43,922 --> 00:28:46,024
We make use --
as I mentioned,

480
00:28:46,091 --> 00:28:49,327
we make use of the resources
available to the Network

481
00:28:49,394 --> 00:28:50,995
and everyone contributes.

482
00:28:52,330 --> 00:28:54,499
An applicant can request
scientific advice

483
00:28:54,566 --> 00:28:56,201
for a post-marketing study.

484
00:28:57,702 --> 00:29:01,372
True. Scientific
advice can be given

485
00:29:01,439 --> 00:29:05,276
for any type of
question and any --

486
00:29:05,343 --> 00:29:08,546
at any time of the development,
including for products

487
00:29:08,613 --> 00:29:10,882
that are even already
authorized or products

488
00:29:10,949 --> 00:29:14,252
that are not yet authorized
but would like to propose,

489
00:29:14,319 --> 00:29:17,155
as part of their
initial dossier,

490
00:29:17,222 --> 00:29:20,225
some proposal for
post-marketing studies.

491
00:29:22,293 --> 00:29:23,962
Scientific advice is provided

492
00:29:24,028 --> 00:29:27,599
only regarding clinical
development. False.

493
00:29:27,665 --> 00:29:31,769
It can be clinical, nonclinical,
pharmacovigilance,

494
00:29:32,770 --> 00:29:34,372
and manufacturing.

495
00:29:36,141 --> 00:29:39,110
In a parallel scientific
advice EMA/FDA,

496
00:29:39,177 --> 00:29:42,413
there will be a single agreed
response sent to the sponsor.

497
00:29:44,616 --> 00:29:47,919
That's false. During
parallel scientific advice

498
00:29:47,986 --> 00:29:53,825
between EMA-FDA, both agencies
discuss, in a bilateral way,

499
00:29:53,892 --> 00:29:57,829
and also discuss with the
sponsor in a trilateral way.

500
00:29:57,896 --> 00:30:03,801
But at the end, each agency
will follow their own process

501
00:30:03,868 --> 00:30:08,072
and will provide their
feedback to the sponsor.

502
00:30:10,375 --> 00:30:13,411
Any product is eligible
for PRIME scheme.

503
00:30:14,746 --> 00:30:18,416
No. Not every product is
eligible for PRIME scheme.

504
00:30:19,050 --> 00:30:20,685
So, you need to
demonstrate that,

505
00:30:21,452 --> 00:30:25,190
you know, there is an advantage
for public health --

506
00:30:25,256 --> 00:30:27,058
that there is an
unmet medical need.

507
00:30:28,960 --> 00:30:31,329
An agreed Pediatric
Investigation Plan

508
00:30:31,396 --> 00:30:33,431
is a requirement at
the time of submission

509
00:30:33,498 --> 00:30:35,533
of a marketing
authorization application.

510
00:30:37,001 --> 00:30:40,538
That is true. A company
needs to submit a PIP

511
00:30:40,605 --> 00:30:43,241
with its marketing authorization
application dossier.

512
00:30:43,875 --> 00:30:45,977
Or, if doesn't have
an agreed PIP,

513
00:30:46,477 --> 00:30:50,315
it needs to submit a deferral
or a waiver from that.

514
00:30:55,920 --> 00:30:57,522
Thank you very much.