The IRDiRC’s Therapies Scientific Committee (TSC) is a multi-disciplinary, multi-stakeholder group of experts in medical research and drug development specialized in rare diseases, comprising representatives of academia, non-profit organizations, industry, regulators, patient experts, clinicians and research funders across the globe.
The TSC is devoted to pursue the therapeutic development agenda of IRDiRC, supporting the rare disease research community in developing “1000 new rare disease treatments by 2027”. The TSC publishes papers, reports and recommendations in the field, provides expert support to all IRDiRC initiatives, and runs multi-stakeholder Task Forces with internal and external experts to address defined challenges and roadblocks in rare disease drug development.
To pursue the 2027 IRDiRC goal, the key targets of the TSC in the next years will be:
- to support the re-definition of the development model for rare disease therapeutics enabling the best integration of the available tools and initiatives specific for rare disease, servicing both profit and non-profit developers, and
- to support the re-definition of the international agenda for rare disease research prioritizing areas of unsolved issues constituting roadblocks for the development of new treatment approaches.
Name | Title and Organization | |
---|---|---|
![]() | Daniel O'Connor (Chair) | Director, Regulatory Policy and Early Access, The Association of the British Pharmaceutical Industry (ABPI), UK |
![]() | Anneliene Jonker (Vice-Chair) | Program Lead Personalised Medicine, University of Twente, The Netherlands |
![]() | Alaa Hamed | Global Head of Medical Affairs Rare Diseases, USA |
![]() | Diana Kwast | Boardmember Dutch Pituitary Foundation, ePAG Endo-ERN, The Netherlands |
![]() | Emilio J.A. Roldan | Scientific Director of Qualix DoT, Argentina |
![]() | Janet Maynard | Director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine at U.S. Food and Drug Administration, USA |
![]() | Ken Sakushima | Office of Advanced Evaluation with Electronic Data Pharmaceuticals and Medical Devices Agency, Japan |
![]() | Maria Cavaller Bellaubi | Patient Engagement Manager, EURORDIS - Rare Diseases Europe, Spain |
![]() | Marjon Pasmooij | Science Programme Manager at Medicines Evaluation Board (CBG/MEB), University Medical Center Groningen, The Netherlands |
Martina Kawome | Ophtalmologist, Department of Ophtalmology, College of Health Sciences, Harare, Zimbabwe |
|
![]() | Michelle Farrar | Professor and Paediatric Neurologist at School of Clinical Medicine/Sydney Children's Hospital, Australia |
![]() | Rajesh Krishna | Distinguished Scientist and Global Lead, Rare Diseases Center of Excellence, Certara, USA |
![]() | Thomas Morel | Research Fellow at KU Leuven/Director, Global Patient-Centred Outcomes Research; Policy at UCB Biopharma, Belgium |
![]() | Trudy Nyakambangwe | Founder at Child And Youth Care Zimbabwe, Zimbabwe |
Related link :
- Therapies Scientific Committee (TSC) Meeting Reports
- Recommendations of the IRDiRC Therapies Scientific Committee to implement IRDiRC Policy 2013 and aim of new 200 therapies by 2020
- Data Mining and Repurposing Task Force
- Patient-Centered Outcome Measures Task Force
- Small Population Clinical Trials Task Force