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 | |
|---|---|---|
 | Shekhar Natarajan (Chair) | Vice President, Head of EU and International Regulatory Affairs, Dyne Therapeutics, USA |
 | Diana Kwast (Vice Chair) | Executive Director, (Un)limited Forward! (patient-led, non-profit foundation for rare diseases & MLTC), EUPATI and EUCAPA Fellow, and PhD Candidate, University Medical Center Groningen (UMCG), The Netherlands |
 | Dorota Zgodka | Pharmaceutical and Healthcare Executive, ERDERA - The European Rare Disease Research Alliance, Multi-stakeholder Advisory Board member, Switzerland |
 | Emilio J.A. Roldan | Scientific Director of Qualix DoT, Argentina |
 | Jill M. Weimer | Senior Director of Therapeutic Development Scientist in the Pediatrics and Rare Diseases Group Sanford Research, USA |
| Joao Rocha | Associate Professor of Pharmacology and Pharmacotherapy, University of Lisbon, Portugal | |
 | Maria Cavaller Bellaubi | Director Global Regulatory Policy, AstraZeneca, Spain |
 | Marjon Pasmooij | Head of the Science Department at the Dutch Medicines Evaluation Board, 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 | Senior Distinguished Scientist and Global Head, Certara Centers of Excellence, USA |
 | 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
- Towards responsible and equitable implementation of Digital Twins in Rare Disease Research and Care: a landscape and gap analysis
- Improving the adoption of digital biomarkers and endpoints in rare diseases therapeutic development
- Facilitating the development of Preventive Medicines at scale for rare diseases
- Engagement of Young People Living with Rare Diseases in Therapy Development
