Introduction
Treatments are often unavailable for rare disease patients, especially in low-and-middle-income Countries. Reasons for this include lack of financial support for therapies and onerous regulatory requirements for approval of drugs. Other barriers include lack of reimbursement, administrative infrastructure, and knowledge about diagnosis and drug treatment options.
The goal of this Working Group, leaving no one behind, requires that access to treatments be available for rare disease patients.
Objectives
To identify the barriers to accessing rare disease drugs, particularly in low-and-middle income populations.
Plans and Timeline
Formation of the Working Group and kick-off of discussions via teleconferences (Q4 2021)
Members
- Alba Ancochea – Alianza Iberoamericana de Enfermedades Raras (ALIBER)
- Gareth Baynam – Western Australian Department of Health, Australia
- Marc Dooms – University Hospitals Leuven, Belgium
- Kelly du Plessis – Rare Diseases South Africa , South Africa
- Michelle Erwee – Takeda, Singapore
- Domenica Taruscio – Istituto Superiore Sanità, Italy
- Durhane Wong-Rieger – Canadian Organization for Rare Disorders, Rare Diseases International
- Rachel Yang – China Alliance For Rare Diseases
- Galliano Zanello – IRDiRC
- Ayda Ramazzina – Rare Diseases International
- Mary Wang – Rare Diseases International
- Safiyya Gassman – Pfizer, USA
- Manuel Posada – Instituto de Salud Carlos III, Spain
- William A. Gahl – National Human Genome Research Institute at NIH, USA
- Virgine Hivert – EURORDIS, France
- Flaminia Macchia – Rare Diseases International
- Ramaiah Muthyala – Indian Organization for Rare Diseases , India
- Daniel O’Connor – Medicines and Healthcare products Regulatory Agency, UK
- Samuel Agyei Wiafe – Rare Disease Ghana Initiative, Ghana
- Scott Williams – Sanofi Genzyme, USA