The webinar held on January 20th, 2026 offered a compelling exploration of one of the most persistent challenges in rare disease research: generating evidence that truly supports informed decision‑making.
During the session, the speakers examined why existing evidence assessment frameworks—used by regulators and HTA bodies—had often lacked alignment and predictability, resulting in delays and inequities in patient access. They then delved into the current landscape, highlighting major initiatives across Europe and beyond that were working to address these systemic issues.
Finally, the webinar showcased how the RealiseD consortium advanced both scientific and operational solutions, while advocating for a fundamental transformation in how evidence for rare diseases is generated, interpreted, and assessed.
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 RealiseD
RealiseD is an Innovative Health Initiative lead y Sigmund Freud Private University and AstraZeneca that unites nearly 40 partners from academia, regulatory bodies, clinical research institutes and hospitals, patient organisations, pharmaceutical companies or European Research Infrastructures to establish new gold standards for clinical trials in rare and ultra-rare diseases.
