Panel Value & Access track
S1001 - EU HTA for Orphan Medicinal Products: Where do the trade-offs start? How will JCA be applied in the context of rare diseases and what impact will it have on national-level policies?
Wed, Mar 19, 2025 · 3:00–4:10 PM CET Montreal Room, Congress Center Basel
About this session
As of 2025, a still unquantified number of treatments for rare diseases that will seek approval in Europe will already be undergoing the new Joint Clinical Assessment, whenever labeled as oncology drugs or ATMPs. However, as of 2028, the progressively expanding scope of the EU HTA Regulation will include all orphan designated products. The challenges coming with new developments in rare diseases will then have to be fully onboarded by the EU assessors. With that, the policy questions about the access and reimbursement of those treatments, as well as the place of rare diseases into our healthcare systems, will have to integrate this double dimension: European – with the JCA – and national – with access decisions that give JCA due consideration although keeping full discretion when considering the balance between evidence acceptance and value appraisal.
The panel is meant to focus on how the JCA is expected to strengthen decision-making when difficult trade-offs are necessary already at the level of evidence generation, focusing precisely on the rare diseases space. The specific rare disease context often raises methodological challenges that have to be addressed throughout the arc of the whole assessment process, both at the early stages of scientific assessment of the evidence package and, consequently, in the decision itself.
The panel will include EUCOPE/EFPIA member representatives, bringing direct experience from their preparatory efforts and forecasting. Patient representatives in the rare disease space (Eurordis) to report about their expectations in JCA and - when possible - past experience in national HTA settings, and HTA/policy makers involved at both levels of the decision process.
Programme:
- Panel Discussion — Anja Schiel, François Houyez, Mihaela Veringa
Speakers
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Treatment Information and Access Director, Health Policy Advisor · European Organisation for Rare Diseases (EURORDIS)
Panelist
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Special Advisor, Lead Methodologist; Leader international HTA (iHTA) NoMA · Norwegian Medicines Agency (NoMA)
Panelist
