Challenges, Regulatory Considerations, and Innovative Solutions for Designing Trials for Rare Diseases
May 19, 2025
Join the IDDI experts for an insightful webinar on the challenges, regulatory considerations and innovation solutions for designing clinical trials for rare diseases on:
Rare diseases are characterized by their low prevalence (e.g., 1 in 2000 people in Europe). According to Orphanet, a consortium of 40 countries that improves knowledge on rare diseases, there are an estimated 4,000 to 5,000 diseases worldwide for which no treatment is currently available. In Europe, 25 to 30 million people are estimated to be affected by these diseases. As Orphanet’s website states: “Rare diseases are rare, but rare disease patients are numerous”.
The development of drugs for rare diseases faces numerous challenges, with the limited availability of patients for clinical trials being a crucial obstacle. Consequently, regulatory agencies are more receptive to innovative (e.g., Bayesian) methods for designing rare-disease trials as compared to trials for more prevalent diseases.
KEY TAKE-AWAYS:
This presentation addresses the challenges in designing rare-disease trials, reviews relevant regulatory guidelines, and presents examples of non-standard designs used in developing treatments for rare diseases.
SPEAKERS:
Elisabeth (Els) Coart, Ph.D.
Director, Statistical Consulting & Research, IDDI
Els graduated in Bio-engineering at KU Leuven in 1995, got her PhD in Molecular Genetics in 2003 at Ghent University, and a Master’s degree in Biostatistics also at Ghent University, in 2007. After some years of experience in research and clinical biostatistics in diagnostic companies, Els joined IDDI in 2011, where she conducted research related to biomarkers in Alzheimer’s disease and validation of surrogate endpoints in advanced colorectal cancer. In 2015, Els joined the team of consultants at IDDI, led by the company founder, Marc Buyse. She served as Director, Consulting Services, from 2017 to 2019 and now focusses on consultancy activities. As a consultant, Els has helped a large number of pharmaceutical and biotechnology companies to design, implement and analyze clinical trials for drug development, with a focus on oncology and biomarkers. She is particularly interested in early-phase trials using frequentist and Bayesian methods, as well as phase trials with biomarker-driven designs. Moreover, given her strong background in assay development she is IDDI’s expert in analytical and clinical validation of in-vitro diagnostics. Els has published more extensively in Alzheimer’s disease, biomarkers, and oncology.
Tomasz Burzykowski, Ph.D.
VP, Research, IDDI
Tomasz is Professor of Biostatistics/Bioinformatics at the Data Science Institute of Hasselt University since 2002. In 2023-2024, he served as the President of the International Society for Clinical Biostatistics.
