Therapies Scientific Committee (TSC)

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