The five-week course will explore the therapeutic development process from the unique perspective of rare diseases. The journey will take the participants from discovery to approved treatment, show how therapeutic targets and drug candidates are identified and validated, and examine the special regulatory frameworks. It will offer an overview of the issues, challenges, and opportunities in translating research findings into treatments for rare diseases.
The course is designed for researchers and students in medicine and health-related research fields, as well as healthcare professionals wanting to further their knowledge of translational research in rare diseases. The course is also suitable for biotech and start-up developers and Patients Advocacy Organisation representatives who want to increase their understanding of current practices in therapeutic developments for rare diseases.
From bench to bedside
What is unique about this course is that it will take students through all of the different phases of drug development, giving further insights into aspects that are not covered by basic research education. The course covers the whole process from drug discovery to development, preclinical models and clinical studies, regulatory aspects and how to go about getting market authorisation. This is the A-Z of a drug development process until it reaches the patient and beyond.
Experts in the field of rare diseases will lead the participants in examining current research projects, learning about the definitions and contexts, as well as hearing from patients directly involved in clinical research. The course will also introduce some of the specific challenges of translational research for rare diseases, i.e that the target is often hard to identify, as well as some of the opportunities of drug repurposing which can speed up the drug development and is especially relevant for rare diseases.
European collaboration
The MOOC was created in collaboration between EATRIS, European Reference Network, EURORDIS, Rare Diseases Europe, French Fondation Maladies Rares and University of Leiden. The MOOC launched on October 17th and is part of five MOOCs in total. Part one is already out: Diagnosing Rare Diseases: from the Clinic to Research and back
The topics of the three next courses will be:
- Innovative therapies and personalized medicine for RDs
- RDs clinical trial methodologies
- Data and rare diseases: ethical and regulatory issue