EUCOPE’s Life Science Lectures – Episode Five

3. Jun 2024 — Register here

The new EU HTA procedure: How can JCAs support access to OMPs for people living with rare diseases

Episode 5 – 3 June 2024 (16:00 – 17:00 CET)

The new Regulation (EU) 2021/2282 on Health Technology Assessment (HTA) will apply from 12 January 2025, to oncology products and ATMPs, including Orphan Medicinal Products (OMPs) with an oncology indication. From 13 January 2028, the new procedure will apply to all OMPs irrespective of the therapeutic indication.

EUnetHTA 21 has developed proposals for methodological guidance that could be applied to JCA assessments, however these guidance documents have raised many questions with stakeholder groups, around the recommended methodologies for evidence generation and assessment. Especially the use of single-arm trials, which for many OMPs are the only viable approach to generate evidence needed for national decision-making, has been called into question.

During episode 5 of Life Science Lectures webinar series, panelists will be discussing the pragmatic and ethical challenges in evidence generation for OMPs, and draw on their clinical experiences as well as knowledge of national HTA procedures, to explore possible methodological approaches that will ensure joint clinical assessments (JCAs) speed up access to treatments for people living with rare diseases.

Panelists will explore the issues that could prevent the use of randomised controlled trials, and discuss alternative evidence generation approaches, drawing on their practical experiences, in order to inform the methodology discussion that is currently taking place in the Coordination Group of Member States and the HTA Stakeholder Network.

Webinar agenda


  • Alexander Natz, Secretary General, EUCOPE

General remarks & Panel discussion

  • Maurizio Scarpa, Coordinator, ERN for Rare Hereditary Metabolic Diseases (MetabERN)
  • Silke Honsek, Senior Manager, International Market Access & New Assets, AOP Health
  • Mark Sheehan, Associate Professor, Oxford Biomedical Research Centre
  • Sheela Upadhyaya, Life Sciences Consultant

Q&A with audience


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