To achieve Universal Health Coverage and “Leave no one behind”, Persons Living with a Rare Disease (PLWRD) across the globe and all rare diseases must have equitable access to medicines and therapies.
access to medicines
Treatments considered “standard-of-care” for many rare diseases are often unavailable to PLWRD, especially in low-and-middle-income countries (LMICs).
Among the many barriers to access are a lack of transparency in pricing, inefficient regulatory processes, as well as barriers along the patient access pathway including lack of reimbursement, diagnostic challenges, limited knowledge of treatment options, and the absence of infrastructure to manage administration and monitor use.
IRDiRC/RDI Global working group
In 2021, RDI and the International Rare Diseases Research Consortium (IRDiRC) launched the IRDiRC/RDI Global Access Working Group to improve access to rare disease treatments, with a specific focus on LMICs.
The new Global Access Working Group builds on the outputs of the former IRDiRC Working Group on Access which aimed at improving standards of care for rare diseases by promoting access to approved therapies.
Under the auspices of IRDiRC, the Working Group published the “Essential List of Medicinal Products for Rare Diseases” in July 2021. The current list is comprised of 204 drugs with orphan designation and/or rare disease indication by the US Food and Drug Administration (FDA), European Medicines Agency (EMA), and/or China’s National Medical Products Administration (NMPA) databases.
working group members
| Gareth Baynam | Western Australian Department of Health, Australia |
| Marc Dooms | Orphan Drug Pharmacist at the University Hospitals Leuven |
| William A. Gahl | National Human Genome Research Institute, NIH, USA |
| Virgine Hivert | EURORDIS- Rare Diseases Europe, France |
| Flaminia Macchia | Rare Diseases International (RDI) |
| Ramaiah Muthyala | Indian Organization for Rare Diseases |
| Daniel O’Connor | Medicines and Healthcare products Regulatory Agency, UK |
| Manuel Posada | Instituto de Salud Carlos III, Spain |
| Ayda Ramazzina | Rare Diseases International (RDI) |
| Mary Wang | Rare Diseases International (RDI) |
| Samuel Wiafe | Rare Disease Ghana Initiative |
| Scott Williams | Sanofi Genzyme, USA |
| Durhane Wong-Rieger | Canadian Organization for Rare Disorders |
| Rachel Yang | China Alliance For Rare Diseases |
| Galliano Zanello | IRDiRC |
| Alba Ancochea | Alianza Iberoamericana de Enfermedades Raras (ALIBER) |
| Kelly du Plessis | Rare Diseases South Africa |
| Michelle Erwee | Takeda, USA |
| Safiyya Gassman | Pfizer, USA |
objectives – IRDiRC/RDI Global Access Working Group :
- Identifying gaps and systemic barriers to accessing, particularly in LMICs
- Build on and update the Essential List of Medicinal Products for Rare Diseases
- Build the capacity of RDI Members to advocate for the inclusion of rare disease medicines and diagnostic tools on the WHO Essential Medicines List and WHO Essential In Vitro Diagnostic List
- Convene multi-stakeholder dialogues on access to medicines in LMICs
The International Rare Diseases Research Consortium (IRDiRC) promotes international collaboration and advances rare disease research worldwide.
RDI is a member of IRDiRC’s Consortium Assembly and 13 RDI Members are part of the IRDiRC Patient Advocacy Constituent Committee composed of umbrella patient advocacy organisations.
Key resources
For more information contact, Mary Wang