| Date & Time | 10 February 2026 |
|---|---|
| Location | Webinar |
Join Our Upcoming Webinar: Putting Patients at the Center of Rare Disease Clinical Trials
Rare disease clinical trials face unique challenges—small, diverse populations make it difficult to capture outcomes that truly matter to patients. In this webinar, we’ll explore how hierarchical (multi-component) endpoints, prioritised by clinical relevance and combined with net treatment benefit, create a patient-centric framework for trial design and analysis. You’ll learn how this approach:
- Embeds the patient voice into statistical design and decision-making.
- Balances benefits and risks in alignment with patient preferences.
- Enables transparent discussions among stakeholders.
- Captures the totality of evidence to define success by what matters most to patients.
“Don’t miss this opportunity to share your expertise and help shape the future of clinical research in ultra-rare conditions!”
Agenda
- Introduction – by Geert Molenberghs, Professor at Hasselt University and KU Leuven
- Presentation – by Rudradev Sengupta, Senior Trial Design Lead at One2Treat
- Discussion Pannel
- Martin Posch – Professor of Medical Statistics at Medical University of Vienna
- Marc Buyse - Founder of IDDI, CluePoints and One2Treat
- Monica Ferrie – Director of Advocacy Beyond Borders
- Simona Giorgi – Scientific Director at Dravet Syndrome Foundation Spain
- 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.
