| Date & Time | 20 January 2026 |
|---|---|
| Location | Webinar |
Join us for an insightful webinar where we tackle one of the biggest hurdles in rare disease research: generating evidence that truly informs decisions.
Current evidence assessment frameworks used by regulators and HTA bodies often struggle to accommodate the specific challenges of rare and ultra-rare disease development. This misalignment can lead to unpredictable decision-making, delays, and inequalities in patient access.
This webinar will explore the key challenges in evidence generation for ultra-rare diseases, highlighting how traditional approaches may fall short. We will discuss the ongoing initiatives in Europe and internationally aimed at improving alignment, and the role of the RealiseD consortium in advancing scientific and operational solutions. The session will also emphasise the need for a fundamental shift in how stakeholders generate, interpret, and evaluate evidence to better serve patients with ultra-rare conditions.
“Explore why current evidence frameworks fall short in rare diseases—and discover how the RealiseD consortium is driving scientific and operational solutions to accelerate patient access and reshape evidence generation.”
Agenda
- Introduction – by Alicia Granados, Global Head of Rare Disease Medical Scientific Advocacy & Insights at Sanofi
- Presentation – by Francois Meyer, Health Technology Assessment consultant
- Discussion Pannel
- Andrew Thomson – Owner and lead consultant at Regnitio
- Karen Facey – Scientific Advisor in HTA at University of Oxford
- Florian Inning – Bundesverband Kleinwüchsige Menschen und ihre Familien e.V.
- Audience Q&A
- Closure
About the project: The RealiseD project, funded by the Innovative Health Initiative (IHI), aims to develop a collaborative and comprehensive methodological and operational framework to improve how clinical trials are designed and conducted in ultra-rare diseases.During this webinar, we will present the project’s objectives, structure, and innovative approaches, offering participants an opportunity to learn about current challenges and emerging solutions for evidence generation in ultra-rare disease research.
