| Date & Time | 3 February 2026 |
|---|---|
| Location | Webinar |
Help Shape the Future of Rare Disease Evidence Generation!
Join this insightful webinar that will tackle one of the most complex challenges in clinical research: designing and analysing trials for rare and ultra-rare diseases. The session will explore innovative trial designs and advanced analytical approaches that can transform evidence generation for these conditions. The discussion will focus on raising awareness of emerging methodologies, fostering collaboration from the very beginning, identifying current challenges in rare disease trials, and working toward shared principles for interpreting ultra-rare disease data.
A key topic of the session will be single-arm trials (SATs). SATs remain widely used in rare disease research and may be accepted by regulators in specific contexts, such as when treatment effects are dramatic or placebo use would be unethical. During the webinar, we will review regulatory and HTA positions on SATs, examine case studies where SATs succeeded and where they fell short, and discuss the challenges they pose. Another key topic will be starting from a single patient and ultimately leading to a US FDA approval, how it is initiated a real-world evidence clinical trial. We will also look at evolving statistical methods and strategies to optimize study design and meet evidence needs.
“Don’t miss this opportunity to share your perspective and help drive innovation in rare disease trials. Together, we can accelerate patient access and improve outcomes worldwide.”
Agenda
- Introduction – by Ralf-Dieter Hilgers, Professor at SFU and RealiseD coordinator
- Presentation – by Yulia Dyachkova, Associate Director Biostatistician at Merck Healthcare
- Presentation – Real-World Evidence as a Trial Model for Rare Disorders by Guillaume Canaud
- Starting from a single patient and ultimately leading to a US FDA approval, he will describe how they initiated a real-world evidence clinical trial. Through this approach, they were able to demonstrate both efficacy and safety.
- Discussion Pannel
- Juan Jose Abellan – Statistics and real-world evidence senior specialist at the European Medicines Agency
- François Houyez – Treatment Information and Access Director at Eurordis
- Angelika Geroldinger – Austrian Medicines and Medical Devices Agency (MEA)
- Yulia Dyachkova – Associate Director Biostatistician at Merck Healthcare
- Jenny Devenport – Global Head of Methods, Collaboration, & Outreach (MCO) at Roche
- Cornelia Dunger-Baldauf – Prime Force consultant at Novartis
- Audience Q&A
- Closure
About the project: RealiseDis 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.
