On 14 November, from 09:00 to 13:00 CET, RWE4Decisions held its annual hybrid Symposium in Brussels: “Developing Real-World Evidence to Deliver Innovation in HTA“, moderated by François Meyer (RWE4Decisions Facilitator) and Ashley Jaksa (Aetion). We explored how Real-World Evidence can drive innovation in Health Technology Assessment (HTA), focusing on the implications of the implementation of the European Health Data Space and HTA Regulation on HTA/Payer decisions across the European Union.
Member of the European Parliament’s Committee on the Environment, Public Health and Food Safety (ENVI) and former rapporteur of the European Health Data Space Regulation
RWE4Decisions Facilitator
Coordinating Advisor on Health, Permanent Representation of Spain to the European Union
Head of Real-World Evidence, European Medicines Agency (EMA)
Head of Department for Health Policy and Health Economics, AOK Rheinland/Hamburg
Member, Acute Leukemia Patients Network (ALAN)
Secretary-General, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)
VP Scientific Strategy and Partnerships, Aetion
RWE4Decisions Facilitator
Chief Specialist, Finnish Medicines Agency (FIMEA)
Associate Director for Data Access and Analysis, National Institute for Health and Care Excellence (NICE)
Board Chairman, RaDiOrg – Rare Disease Belgium
Vice-President and Head of Patient Value & Access – Europe & Canada, Takeda
Advisory Member, Directorate General for Common Portfolio of SNS and Pharmacy Services, Spanish Ministry of Health
Senior Adviser and former CEO, Belgian Institute for Health and Disability Insurance (INAMI-RIZIV)
The Policy plenary session was kickstarted by two insightful presentation: firstly, Enrique Terol (Permanent Representation of Spain to the EU) reminded that the EHDS, pharmaceutical package and HTAR aim to create an ecosystem where RWD/E is usable and accessible, with success dependent on Member States’ data interoperability infrastructures. Then, Patrice Verpillat (EMA) provided an update on DARWIN EU and the EMANS strategy, underscoring the shared goal of regulators, HTA bodies and Payers to facilitate ‘the path to accessibility’ for patients, and the role of the HTAR to revive their collaboration.
Moving into the discussion section, Anne-Pierre Pickaert (Acute Leukemia Advocates Network) represented the patient voice, describing the delicate line between unethical Randomised Control Trials and Single Arm Trials to enable better and faster patient access to innovative therapies. Then, Christoph Rupprecht (AOK Rheinland/Hamburg) provided a Payer perspective from Germany, and shared about the routine data collection AbD in Germany connecting RWE and RCTs. Finally, Alexander Natz (EUCOPE) discussed the crucial role of JSCs, and provided examples where Real-World Evidence was crucial to closing data gaps and enable patient access.
Karen Facey (RWE4Decisions Facilitator) presented the RWE4Decisions 2024 activities to support the EU health agenda, and particularly our work on PLEG. Although PLEG is not currently in the HTAR, HTA/Payers are hugely interested in making PLEG more efficient.
Ashley Jaksa (Aetion) gave an introductory presentation into the new Stakeholder Actions to Generate better Real-World Evidence, discussing the timeline and methodology of their revision, their dissemination, and their division into the four pillars to support development of robust RWE for HTA/Payer decision-making (as outlined in our 2022 publication).
This was followed by a panel discussion with four representatives of the RWE4Decisions Learning Network, who were involved in the development of the actions: Piia Rannanheimo (FIMEA), Shaun Rowark (NICE), Stefan Joris (RaDiOrg – Rare Diseases Belgium), and Andre Vidal Pinheiro (Takeda), bringing perspectives from National HTAs, Collaborative HTAs, Patient Groups, and Pharmaceutical Industry. As part of their interactive discussion, focused on the importance of collaboration and transparency, they presented their favorite actions for their Stakeholder Group, and the day-to-day steps they are taking towards their implementation.
The dissemination of the Stakeholder Actions will be at the centre of our work in 2025 — stay tuned!
Carlos Martin Saborido (ISCIII) discussed the next steps for the HTAR implementation, stressing the importance of early RWD collection to facilitate the HTA pathway and inform reimbursement. “We need to be proactive, not reactive” and work with the Regulator to help reduce uncertainties for Rare Diseases.
Jo de Cock (RIZIV-INAMI) closed with remarks about the unique role of the RWE4Decisions Learning Network to evolve the thinking around the positioning of RWE over the past years. RWE4Decisions will continue to engage with Payers and other stakeholders as European and national policies evolve.
Public Webinar: From Policy to Practice: EHDS Implementation to Support Better Real-World Evidence for HTA/Payers
Public Webinar: HTA/Payer Collaboration in the Nordics: The Role of Real-World Evidence
Public Webinar: Can Endpoints from Digital Technology Provide Meaningful Outcomes for HTA?
