2.1 ema regulatory science to 2025 · 1 regulatory science engagement plan to 2025 embrace science,...

An agency of the European Union

EMA Regulatory Science to 2025 A work-in-progress

PCWP/HCPWP – 25 September 2018

Presented by Tony Humphreys Head of Scientific Committees Regulatory Science Strategy

Regulatory Science Engagement Plan to 2025 1

Embrace science, technology and communication tools which are changing society at fast pace

Continue to advance the value and impact of what we do

Strategically recruit and manage resources on key activities

Remain competitive as reference system in Europe and beyond

Align EMA’s Regulatory Science Strategy with the EMRN future strategy and cascade activities in relevant programming documents

What we must do to get there

1

2

3

4

5

1

• Baseline report to identify science and technology trends and regulatory tools in consultation with Committee Chairs

2

• Stakeholders outreach - interviews to verify the baseline report’s findings and identify any missed trends

3

• Version 1 of the EMA Regulatory Science to 2025 compiled on the basis of inputs from step 1 and 2

What we have done so far



1 Horizon scanning fiches to a baseline report

1 2

3 4

What is the baseline report

A survey of the key trends in science and technology and regulatory tools impacting the operations of the EMA.

An attempt to map the “as is” situation across a very diverse series of topics and to be used as a reference source to support the development of a Regulatory Science Strategy through 2018.

It documents succinctly current understanding of these trends likely impact on operations current levels of network engagement preliminary recommendations for future engagement


Status overview of the fiches (Horizon Scanning)


Trends in science and technology

Major therapeutic areas

Oncology

CNS Neurodegenerative diseases

CNS psychiatry

Diabetes

Obesity

HIV

Vaccines

Immunotherapies

Gene therapy and Regenerative Medicine

Gene therapy

Cells and Tissues based products

New materials

Trends in science and technology

Personalised medicine

Personalised medicine

Biomarkers

Methods, technologies and other trends

Nanotechnology

New Omics (e.g. microbiomics)

Taxonomy of disease

Digital health and wearable technology

Novel manufacturing and 3D printing

Status overview of the fiches (Horizon Scanning)


Trends in the use of regulatory science tools Access pathways PRIME Adaptive Pathways Biosimilars Synergies with HTAs activities Synergies with payers activities Non clinical methodology Novel non-clinical models Application of 3Rs in development of medicines Environmental risk assessment (veterinary elements) Clinical methodology Modelling and Simulation Extrapolation Patient reported Outcomes (PROs) New endpoints Bayesian methods Co-acting medicinal products

Trends in the use of regulatory science tools Special populations Pregnancy Paediatric Geriatric Risk-benefit evaluation Risk-benefit project Big data and e-Health Big data Real world evidence Open science Inform social and behavioural science Health threats AMR Emerging Health threats

From ‘fiches’ to Baseline report

7

Introduction

Short summary of topic

Challenges &

opportunities

Maturity and impact

on regulatory

system

Key emerging

trends

Recommendations to meet challenges and opportunities

Recommended actions

Annex

Resource documents

EMA and network

competencies

Regulatory science

initiatives

Regulatory Science Engagement Plan to 2025


2 Stakeholders outreach interviews

1 2

3 4

Engaging with partners and stakeholders


Stakeholders outreach – February to September 2018

EMA RSO matrix

EU-IN EU NCA

International

regulators

Other (legal, ethical, policy)

All EMA scientific

committees & WPs

EC

EU bodies & agencies

Patient reps (3)

Healthcare professiona

ls (17) HTAs (2)

Payers (3)

Industry (16)

Academia R&D (12)

Research infrastructu

res

Research funding (2)

Investors

Academia Regulatory

Science

To be engaged

Engaged

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


3 EMA Regulatory Science to 2025

1 2

3 4

Structure of Regulatory Science to 2025

11

Vision

Strategic Goal

Core Recommendations

Underlying Actions

Impact Estimation

Where we want to be

What we must achieve to get there

Specific objectives

Planned actions to achieve objectives

Measurable outcomes


Human Vision

To underpin its mission of protecting human health, EMA must

catalyse and enable regulatory science and

innovation to be translated into patient-

centred access in evolving healthcare

systems.

Development Advisory

Processes

New regulatory

science tools

Omics

Wearable

AI

Risk-benefit evaluation

Clinical and non-clinical

methodologies

Engagement and

communication Access

HTAs

Payers

Patients and HCP

International Academia

Lifecycle data assessment

and generation

Priority areas - Driving the evidence generation

Human strategic goals


Catalysing the integration of science

and technology in drug development

Driving collaborative evidence generation

Improving the scientific quality of evaluations

Advancing patient-centred access to

medicines in partnership with

healthcare systems

Enabling and leveraging research and innovation

in regulatory science

Addressing emerging health threats and supply challenges

Human strategic goals and core recommendations

15

CATALYSING THE INTEGRATION OF SCIENCE AND TECHNOLOGY IN DRUG DEVELOPMENT

• Diversify and integrate the provision of regulatory advice along the development continuum

• Promote and invest in PRIME scheme

• Develop framework for integrated evaluation of Medical Devices, IVDs and borderline

products

• Develop a collaborative framework to support developments in personalised medicine,

biomarkers and ‘omics’

• Support translation of cell, genes and tissue based products into patient treatments

• Facilitate the implementation of novel manufacturing technologies

• Develop understanding of and regulatory response to nanotechnology and new materials’

utilisation in pharmaceuticals Regulatory Science Engagement Plan to 2025


16

DRIVING COLLABORATIVE EVIDENCE GENERATION – IMPROVING THE SCIENTIFIC QUALITY OF EVALUATIONS

• Leverage novel non-clinical models and 3Rs

• Optimise capabilities in modelling and simulation and extrapolation

• Foster innovation in clinical trials

• Develop the regulatory framework for emerging clinical data generation

• Expand benefit-risk assessment and communication

• Invest in special populations initiatives

• Develop adaptive pathways via scientific advice in conjunction with key stakeholders

• Exploit digital technology and artificial intelligence in decision-making



17

ADVANCING PATIENT-CENTRED ACCESS TO MEDICINES IN PARTNERSHIP WITH HEALTHCARE SYSTEMS

• Promote use of high quality real world evidence in decision-making

• Develop network competences and specialist collaborations to engage with big data

• Enable HTAs’ preparedness and downstream decision-making for innovative medicines

• Share key therapeutic innovation impact assessment with Payers

• Reinforce patients involvement in medicines development

• Deliver real-time electronic product information (EPI)

• Promote the uptake of biosimilars in the healthcare system

• Implement the strategy for external communications to further promote trust and confidence in the EU

regulatory system



18

ADDRESSING EMERGING HEALTH THREATS AND AVAILABILITY /THERAPEUTIC CHALLENGES

• Assign Network Resources to Implement Health Threats Plan

• Continue to promote the responsible use of antimicrobials and their alternatives

• Promote global cooperation to anticipate and address supply challenges

• Support the Development and Implementation of a Repurposing Framework



19

ENABLING AND LEVERAGING RESEARCH AND INNOVATION IN REGULATORY SCIENCE

• Develop and exploit a synergistic, network-led partnership with academic researchers to

facilitate and apply innovation

• Leverage the establishment of regulatory science research hubs to accelerate innovative drug

development

• Collaborate with the new research hubs to disseminate knowledge, expertise and innovation

across the network and to its stakeholders

• Optimise the leverage of knowledge and expertise in the EU and globally



4 Next steps

1 2

3 4

October/November 2018

December 2018

2Q-3Q 2019

4Q 2019

Next steps

June 2019 End of Public consultation

December 2018

RSS Human and

Veterinary released for 6-months

public consultation


24 October 2018

Human stakeholders

workshop

8 November 2018

Veterinary stakeholders

workshop

July to September

2019 Finalisation of

EMA’s Regulatory Science to 2025 with

SciCoBo input

4Q 2019 Publication of

EMA Regulatory Science to

2025

Key input into Network Strategy

2025

Any questions?

[email protected] 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

Further information

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2.1 ema regulatory science to 2025 · 1 regulatory science engagement plan to 2025 embrace science,...

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