Upcoming event
05
Nov
06
Nov
VASCERN Days 2026
The RealiseD project will host a four-part webinar series focused on reshaping clinical trials in rare and ultra-rare diseases. The series brings together researchers, regulators, health technology assessment bodies, pharmaceutical companies, and patient organisations to address key challenges in clinical trial design and evidence generation.
These webinars aim to define new standards for rare disease research by promoting innovative trial methodologies and stronger patient-centred approaches. Each session features experts from academia, industry, and regulatory backgrounds, offering practical perspectives on how to improve research quality and patient access to therapies.
Webinar 1 (13 January 2026): Realising Clinical trials in Ultra-Rare diseases
The opening webinar introduces the RealiseD initiative and its approach to tackling the unique methodological barriers that slow progress in ultra-rare disease trials. Professor Dieter Hilgers (Sigmund Freud University) outlines objectives, collaborative frameworks, and shared principles for interpreting data in very small populations.
Register for Webinar 1: Click here
Webinar 2 (20 January 2026): Evidence Assessment Framework – The need for a mindset shift for developers, regulators and HTAs
The second webinar focuses on the challenges of generating and assessing evidence in rare disease trials. François Meyer, an expert in health technology assessment, discusses why current frameworks can delay access and explores global and European efforts to promote flexibility, innovation, and alignment in evidence evaluation.
Register for Webinar 2: Click here
Webinar 3 (3 February 2026): Single arm, RCT or something in between – how to enrich clinical trial design and analysis in rare diseases
Session three explores single-arm trials (SATs), randomised controlled trials (RCTs), and hybrid designs. Industry and methodological experts examine regulatory and HTA perspectives, review case studies, and offer advanced statistical strategies to optimize trial design for rare and ultra-rare diseases.
Register for Webinar 3: Click here
Webinar 4 (10 February 2026): Enhancing patient-centricity in Rare Disease Clinical Trials
The final webinar focuses on embedding patient priorities into trial design. Rudradev Sengupta (One2Treat) presents frameworks using hierarchical endpoints and net treatment benefit to balance risk and benefit in a way that reflects patient preferences in clinical outcomes.
Register for Webinar 4: Click here