Enhancing Patient-Centricity in Rare Disease Clinical Trials
The webinar, held on February 10th, 2026, took a deep dive into the unique challenges of rare disease clinical trials—where small and heterogeneous patient populations make it difficult to measure outcomes that genuinely reflect patient priorities. During the session, the speakers showcased how hierarchical (multi‑component) endpoints—ranked by clinical relevance and paired with net treatment benefit—offered a powerful, patient‑centric framework for both trial design and analysis.
They demonstrated how this approach:
- Wove the patient voice directly into statistical design and key decisions.
- Balanced benefits and risks in ways that aligned with what patients value most.
- Enabled clearer, more transparent conversations among all stakeholders.
- Captured the full breadth of evidence to define success by outcomes that truly mattered to patients.
“Don’t miss this opportunity to share your expertise and help shape the future of clinical research in ultra-rare conditions!”
Watch the webinar!
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 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.
