Session (CTI)

Issue: Orphan Medicinal Products will become subject to mandatory Joint Clinical Assessments, from 13 January 2028 and we only have a few months to prepare the system to accommodate OMPs, that often have a different trial design. The Regulation (EU) 2021/2282 on health technology assessment recognises the ethical and practical challenges inherent to study design and evidence generation in the area of rare diseases – however, the current methodological guidance documents do not explicitly account for the specificities of OMPs. Overview: The panel will explore the rare disease companies’ requirement for clarity ahead of entering the JCA in 2028, and the readiness of the EU HTA as it relates to OMPs, covering inter alia the methodological guidance, interactions and advice opportunities and expert involvement in rare and ultra-rare diseases. The panel will also reflect on the challenge for smaller companies that often specialise in rare diseases in navigating the JCA. During the panel speakers representing rare disease companies, the EU HTA Coordination Group and patients will discuss the readiness of the EU HTA system for OMPs, and the implications for national access decisions, that will be based in part on the JCA report.