EMA Regulatory Science to 2025: Stakeholders’ outreach

1. EMA Regulatory Science to 2025: Stakeholders’ outreach Info session HCP and Patients’ organisations Presented by Lucia D’Apote on 17 May 2018 Scientific Committees Regulatory Science Strategy

2. Innovation landscape 1 • New development paradigms are progressing with unprecedented speed • Complex and challenging products to develop, manufacture, evaluate and make available to patients • A reflection at international level (ICMRA) is happening, to prioritise and recommend cooperation in innovative areas

3. Opportunistic innovation • Medical research and technological innovation are both advancing at an exponential pace, rapidly integrating with each other. • Research and technology meet when there is a market opportunity, sometimes at unexpected moments and places. • This often brings radical systemic changes.

4. Emerging business models: HTAs and payers • Highly innovative, potentially curative medicines require re-definition of value • Emerging business models are driving need to improve cooperation between EU Member States on a key element of drug-pricing decisions

5. 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 General assembly, 26 January 2018

6. New role for regulators

7. “Regulators need to take a new role at the crossroads 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, an enabler for science to be translated into patient-centred healthcare and fit in the reality of healthcare systems.”

8. Regulators as enablers between science and healthcare systems Identifying the main gaps Connecting various stakeholders together, in order to bridge gaps Scanning the horizon

9. Our response: EMA Regulatory Science Strategy • Regulatory Science Observatory (RSO) • Horizon Scanning process • Regulatory Science Strategy • EMRN Strategy to 2025

10. What is the EMA Regulatory Science Strategy “Regulatory science is defined as a range of scientific disciplines that are applied to the quality, safety and efficacy assessment of medicinal products and that inform regulatory decision-making throughout the lifecycle of a medicine. It encompasses basic and applied medicinal science and social sciences, and contributes to the development of regulatory standards and tools.”

11. EMA Regulatory Science Observatory: a collaborative approach Investors Health trends EU bodies collaboration Scientific literature Trends in science, technology, and regulatory science tools. Committees Expert groups International liaison Horizon Scanning Detection Meetings Conferences Products: datasets mining Draft legislation Guidelines Media reports

12. Identifying the main gaps: EMA baseline report

13. Engaging with partners and stakeholders Patientsrepresentatives Healthcare professionals Academia Reg science HTAs EU-IN Investors EU National Competent Authorities EU bodies and agencies EMA RSO matrix Research funding Payers European Commission International regulators EMA Scientific Committees WPs Research Infrastructures General public Other (legal, ethical, policy) Academia R&D Industry • 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

14. Stakeholders’ outreach • Semi-structure interview 1 hour • High level questions: • What are the top three science, technology and regulatory challenges and opportunities in your field of work? How each of them will impact medicines development, and then translation to clinical care? • How can regulators help navigate these challenges and opportunities? • Which therapeutic areas will be most impacted upon in the next 5 years? • Are there any key initiatives or consortia impacting these trends? • More broadly, are there any other concerns or recommendations you have for the agency? Healthcare professionals HTAs Investors Payers General public Industry

15. Building our regulatory science strategy - Timeline • Baseline report fiches • Baseline report • EMA Regulatory Science Strategy • Network Strategy to 2025 • 4Q2017 • 1Q2018 • 1Q2019 1H2020 • Sep 2017 Horizon Scanning: Detection, Triage and Impact analysis of emerging and future trends in science/ technology and regulatory science tools Baseline report: how EMA can meet emerging and future trends in science and technology, use resources and collaborations to advance strategic regulatory science • Stakeholders outreach • 4Q2018-1Q2019 • RS2025 public consultation • Oct • Stakeholders’ workshops • (1 Human & 1 Vet)

16. Questions and discussion