RealiseD Webinar: Enhancing patient-centricity in Rare Disease Clinical Trials

Описание: 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.

You can find all the information in the project's website: https://realised-ihi.eu/webinars/

Agenda

00:00:00 Introduction - by Alicia Granados, Global Head of Rare Disease Medical Scientific Advocacy & Insights at Sanofi
00:17:21 Andrew Thomson - Owner and lead consultant at Regnitio
00:29:17 Karen Facey - Scientific Advisor in HTA at University of Oxford
00:42:23 Francois Meyer, Health Technology Assessment consultant
00:59:02 Florian Inning - Bundesverband Kleinwüchsige Menschen und ihre Familien e.V.
01:06:40 Panel discussion