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) | Expert Medical Assessor, Medicines and Healthcare products Regulatory Agency, The United Kingdom |
 | Anneliene Jonker (Vice-Chair) | Funding Officer, TechMed Centre, University of Twente Enschede, The Netherlands |
 | Annemieke Aartsma-Rus | Leader of the “DMD exon skip group”, Professor, Department of Human Genetics, Leiden University Medical Center, The Netherlands |
 | Janet Maynard | Director , Office of Orphan Product Development, Office of the Commissioner, Food and Drug Administration, USA |
 | Ken Sakushima | Office of Advanced Evaluation with Electronic Data Pharmaceuticals and Medical Devices Agency |
 | Maurizio Scarpa | Director, Regional Coordinating Center for Rare Diseases, Udine University Hospital, Italy Coordinator, European Reference Network For Hereditary Metabolic Diseases (MetabERN) |
 | Michela Gabaldo | Head Alliance Management & Regulatory Affairs Manager at SR-TIGET San Rafaele Telethon Institute for gene therapy, Milan, Italy |
 | Robin Conwit | Medical Officer/ Program Director Division of Clinical Research, National Institute of Neurological Disorders and Stroke (NINDS), NIH and Associate Professor Georgetown University, USA |
 | Sangeeta Jetwha | Head, Patient Partnership, Rare Diseases, Roche, Switzerland |
 | Thomas Morel | Research Fellow at KU Leuven / Director, Global Patient-Centred Outcomes Research & Policy at UCB Biopharma, Belgium |
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