EU’s Innovative Medical Technology and EMA’s Measures · national healthcare systems: in order to promote public health in the current ... Yesterday Today Tomorrow 10. EMA Guido

An agency of the European Union

EU’s Innovative Medical Technology and EMA’s Measures

27 October 2017

Presented by Guido Rasi

Executive Director, European Medicines Agency (EMA)

Summit symposium 25-27 October 2017, Kyoto, Japan

Innovation landscape: what we see

New development paradigms are

progressing with unprecedented speed

Complex and challenging products to

develop, manufacture, evaluate and make

available to patients

EMA Guido Rasi - ICMRA Symposium 27 October 20171

A reflection at international level is needed to prioritise andrecommend cooperation in innovative areas

CRISPR-Cas9 genome editing

Opportunistic Innovation Landscape

Medical research and technological innovation

are both advancing at an exponential pace,

rapidly integrating each other

Research and technology meet when there is a

market opportunity, sometimes at unexpected

moments and places

This often brings radical systemic

changes


Emerging business models

Innovative industry is

increasingly transforming

itself into a service

provider

Industry needs also to

train specialised medical

staff for delivering

innovative therapies to

patients


Autologous CAR T-Cells are a personalized therapeutic procedure

One Leukopack can yield 100s of doses

Allogeneic CAR T-Cells are a universal product candidate with multiple

doses

Product

vs.

Service

Why we need a new approach to innovation

New competences for the regulatory and public

health systems, both for the evaluation and for the

delivery to patients

To build expertise to evaluate increasingly

complex products, Regulators need to reach out to

many stakeholders and interact with new

players outside the health arena

Shift from treatment to potentially curative

medicines require new approaches to value

assessment, payment and financing

Constraints driving strategic allocation of resources


“Regulators can be an enabler between science and healthcare systems by

scanning the horizon, identifying the main gaps and connecting the various

stakeholders together in order to bridge those gaps.”


“Regulators need to take a new role at the crossroad between science and

national healthcare systems: in order to promote public health in the current

environment, they can no longer be just a gateway between those two worlds;

they need to become a catalyst/enabler for science to be translated into patient-

centred healthcare and fits in the reality of healthcare systems.”

Regulators — an enabler between science and healthcare systems

EMA Regulatory Science Observatory: a collaborative approach



Horizon Scanning

Detection

trends in science,

technology, and

regulatory science

tools

Informing the Regulatory Science Strategy

Health trends

Scientific

literature

Media reports

Investors

International

liaison

Committees

Expert groups

EU bodies

collaboration

Products:

datasets mining

Draft legislation

Guidelines

Outreach

activities

Meetings

Conferences

Engaging with partners and stakeholders

Leveraging collaboration at EU and

International level with partners

Stakeholder engagement to avoid self-

referential outcomes

Identification of hotspots in the current

regulatory science discussions


EMA RSO

matrix

Patientsrepresentatives

Academia

Reg science

Industry

General public

HTAs

European

Commission

Payers

Healthcare

professionals

EU-IN

International

regulators

Investors EU

National

Competent

Authorities

EU bodies

and

agencies

Research

Infrastructures

Research

funding

EMA

Scientific

Committees

WPsAcademia

R&D

Other

(legal, ethical,

policy)

Identifying the main gaps: EMA baseline report

EMA Guido Rasi - Summit of ICMRA 25-27 October 20179

Trends in science and technology Trends in Regulatory Science tools

Connecting stakeholders: crucial role of Regulators


Parallel EMA-HTA

scientific advice

inclusion of additional

stakeholders e.g.

Patients, payers

EUnetHTA

Adaptive pathway

Scientific concept for

medicine development

and data generation

Integrated public

health players

advice

patient centred

value centred

access centred

Yesterday Today Tomorrow

10



Are we ready to embrace innovation in regulatory science?

Real World

Evidence

Integrating data e.g.

from Registry in our

assessment

Big data Change

in regulatory

paradigm

Use of Artificial

Intelligence in Regulatory

Decision Making?

• Big Data landscape from

a regulatory perspective

• Acceptability for

Regulatory Decision

Making (criteria)

11



PK-PD dose

finding/extrapolati

on

Clinical trials

Modelling

Disease modelling

(in-silico)

How are we impacting on science

12



Stratification

Biomarkers

Integrated Omics

Validation of

surrogate

endpoints

Development of

biomarker-driven

Personalised

Medicines

How we can impact on science

13


Leveraging collaborative research

EU-NTC14

Bridging the gaps and setting priorities

A model to set up priorities in the Regulatory Science Strategy

EMA Guido Rasi - Summit of ICMRA 25-27 October 201715

Big data

HTAs

Extrapolation

Personalised medicine

Health threats

AMR

Gene therapy

Oncology

Investment

Acti

vit

y

Disease Modelling

Pregnancy

Disruptive

innovation


Innovation in the global regulatory science ecosystem

16

European Medicines Agency

30 Churchill Place • Canary Wharf • London E14 5EU • United Kingdom

Telephone +44 (0)20 3660 6000 Facsimile +44 (0)20 3660 5555

Send a question via our website www.ema.europa.eu/contact

Follow us on @EMA_News

Thank you for your attention

Documents

EU’s Innovative Medical Technology and EMA’s Measures · national healthcare systems: in order to promote public health in the current ... Yesterday Today Tomorrow 10. EMA Guido