RealiseD Webinars: Driving Innovation in Rare Disease Clinical Trials
Start 2026 with fresh insights! Join #RealiseD’s webinar series (13 Jan–10 Feb) on innovative clinical trials in rare diseases.
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Read the following introduction to the webinars by RealiseD:
The RealiseD project will host a series of four-part webinars that will focus on key topics aimed at reshaping the landscape of clinical research for rare and ultra-rare diseases. Beyond informative, the sessions have been designed as a call to action for researchers, regulators, health technology assessment bodies, pharmaceutical companies, and patient organisations to come together and define new standards for evidence generation and trial design.
The series will tackle some of the most critical challenges in the field, including the complexity of trial methodologies and the urgent need for patient-centered approaches. Each webinar will feature leading experts offering fresh perspectives on innovative solutions and collaborative strategies designed to accelerate progress and expand patient access to life-changing therapies.
Webinar 1: Realising Clinical trials in Ultra-Rare diseases
The opening webinar sets the stage for the RealiseD initiative, introducing its vision and methodology for tackling the unique challenges of ultra-rare disease trials. Professor Dieter Hilgers of Sigmund Freud University will guide participants through the project’s objectives and structure, highlighting how RealiseD aims to foster collaboration from the earliest stages of research. The discussion will address the obstacles that have long hindered progress in this area and propose practical solutions, while also establishing shared principles for interpreting data in ultra-rare conditions. This is an opportunity to be part of a transformative movement that seeks to redefine what is possible in rare disease research.
Webinar 2: Evidence Assessment Framework – The need for a mindset shift for developers, regulators and HTAs
The second webinar turns the spotlight on one of the most critical issues in rare disease research: the generation and assessment of evidence. François Meyer, a leading consultant in health technology assessment, will examine why current frameworks often fail to deliver predictable and aligned outcomes, resulting in delays and inequalities in patient access. The session will explore the global and European initiatives aimed at addressing these shortcomings and present the RealiseD consortium’s efforts to advance scientific and operational solutions. More importantly, it will advocate for a fundamental shift in mindset—one that embraces flexibility and innovation in the way evidence is generated, interpreted, and assessed.
Webinar 3: Single arm, RCT or something in between – how to enrich clinical trial design and analysis in rare diseases
Single-arm trials remain a cornerstone of rare disease research, but their limitations and complexities cannot be ignored. In the third webinar, experts Yulia Dyachkova (Merck Healthcare), Jenny Devenport (Roche), Cornelia Dunger-Baldauf (Novartis) abd Guillaume Canaud will lead a deep dive into the evolving role of SATs in drug development. The discussion will cover regulatory and HTA perspectives, review case studies of success and failure, and explore advanced statistical methods designed to optimise trial design. This session is not just about understanding the current landscape—it is about shaping the future of evidence generation for rare and ultra-rare diseases.
Webinar 4: Enhancing patient-centricity in Rare Disease Clinical Trials
The final webinar focuses on what truly matters: the patient. Rare disease trials often struggle to capture outcomes that reflect patient priorities but RealiseD is working to change that. Rudradev Sengupta, Senior Trial Design Lead at One2Treat, will present an approach that embeds patient voices into every stage of trial design and analysis. By using hierarchical endpoints and net treatment benefit, this framework balances benefits and risks in alignment with patient preferences, enabling transparent discussions among stakeholders and ensuring that success is defined by what matters most to patients. This is a vital conversation for anyone committed to making clinical research more humane and impactful.
About RealiseD
RealiseD is an ambitious project funded by the Innovative Health Initiative and led by Sigmund Freud Private University and AstraZeneca. Bringing together nearly 40 partners from academia, regulatory bodies, clinical research institutes, hospitals, patient organisations, pharmaceutical companies, and European Research Infrastructures, this initiative aims to establish new gold standards for clinical trials in rare and ultra-rare diseases. Its mission is clear: to accelerate research, reduce inequalities, and ensure that patients receive timely access to treatments that can transform lives.
Professor Amy Jayne McKnight
Media
Please do contact our rare disease team by email for further information.