The series convenes researchers, clinicians, regulators, health technology assessment (HTA) experts, industry and patient representatives to discuss evidence generation and trial design for rare and ultra‑rare diseases. Registration is free but mandatory. Full agendas, speaker line‑ups and joining details are available on the RealiseD webinars page.RealiseD (compRehensive mEthodological and operational Approach to cLinical trIalS in ultra rarE Diseases) is an Innovative Health Initiative (IHI) project focused on developing and supporting methodological and operational approaches for clinical trials in rare and ultra‑rare diseases, including the design, analysis and interpretation of data from very small patient populations. An overview of the project’s scope is available via the IHI project factsheet for RealiseD.
ERDERA (the European Rare Diseases Research Alliance) works to strengthen rare disease research across Europe, including through its Clinical Research Network and a strong focus on meaningful patient engagement (Patient and Public Involvement and Engagement (PPIE)). RealiseD’s work aligns with these themes and, according to the IHI project factsheet, RealiseD’s tools and systems are intended to be tested and initiated within ERDERA.
Why these webinars may be useful to the rare disease community
Clinical research in rare and ultra‑rare diseases often requires careful choices about study design, feasibility and the interpretation of evidence. Across four sessions, the series addresses topics such as evidence assessment (including regulator and HTA perspectives), design options spanning single‑arm and randomised trials, and approaches intended to strengthen patient‑centredness in trial outcomes.
Webinar 1 — Realising clinical trials in ultra‑rare diseases
This opening session introduces RealiseD’s end‑to‑end approach to trials in ultra‑rare conditions, including shared principles for interpreting data from very small populations and practical approaches to operational barriers.
Register for Webinar 1
Webinar 2 — Evidence assessment framework: a mindset shift for developers, regulators and HTAs
This session examines why current regulatory and HTA frameworks can lead to uncertainty in rare diseases and discusses approaches for greater flexibility in how evidence is generated and assessed.
Register for Webinar 2
Webinar 3 — Single‑arm, RCT or something in between: enriching clinical trial design and analysis
Focusing on single‑arm trials (SATs), this webinar explores when SATs may be fit‑for‑purpose, how regulators and HTA bodies view them, and how statistical methods and real‑world evidence strategies can support decision‑making.
Register for Webinar 3
Webinar 4 — Enhancing patient‑centricity in rare‑disease clinical trials
The final session focuses on methods intended to embed patient priorities into trial design and analysis, supporting clearer multi‑stakeholder discussions about outcomes that matter to people living with rare conditions.
Register for Webinar 4
Practical details
Dates: 13, 20, 27 January and 10 February 2026
Format: Online
Cost: Free (registration required)
Series information: RealiseD webinars
