RealiseD Webinar: Enhancing Patient-Centricity in Rare Disease Clinical Trials
Автор: RealiseD
Загружено: 2026-02-13
Просмотров: 28
Описание:
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.
Agenda
00:00:00 Introduction - by Geert Molenberghs, Professor at Hasselt University and KU Leuven
00:04:23 Presentation - by Rudradev Sengupta, Senior Trial Design Lead at One2Treat
00:28:19 Martin Posch - Professor of Medical Statistics at Medical University of Vienna
00:50:40 Marc Buyse - Founder of IDDI, CluePoints and One2Treat
00:54:11 Monica Ferrie - Director of Advocacy Beyond Borders
01:18:17 Audience Q&A
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