To achieve Universal Health Coverage and “Leave no one behind”, Persons Living with a Rare Disease (PLWRD) across all countries 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 is 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 Access Task Force
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.
obejectives – IRDiRC/RDI Global Access Working Group :
- Identifying gaps and systemic barriers to access, 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 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.
- Essential List of Medicinal Products for Rare Diseases – Recommendations from the IRDiRC Rare Disease Treatment Access Working Group. William A. Gahl, Durhane Wong-Rieger, Virginie Hivert, Rachel Yang, Galliano Zanello, Stephen Groft. Orphanet Journal of Rare Diseases, 2021.
For more information contact, Mary Wang