Global Access

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 BaynamWestern Australian Department of Health, Australia
Marc DoomsOrphan Drug Pharmacist at the University Hospitals Leuven
William A. GahlNational Human Genome Research Institute, NIH, USA
Virgine HivertEURORDIS- Rare Diseases Europe, France
Flaminia MacchiaRare Diseases International (RDI)
Ramaiah MuthyalaIndian Organization for Rare Diseases
Daniel O’ConnorMedicines and Healthcare products Regulatory Agency, UK
Manuel PosadaInstituto de Salud Carlos III, Spain
Ayda RamazzinaRare Diseases International (RDI)
Mary WangRare Diseases International (RDI)
Samuel WiafeRare Disease Ghana Initiative
Scott WilliamsSanofi Genzyme, USA
Durhane Wong-RiegerCanadian Organization for Rare Disorders
Rachel YangChina Alliance For Rare Diseases
Galliano ZanelloIRDiRC
Alba AncocheaAlianza Iberoamericana de Enfermedades Raras (ALIBER)
Kelly du PlessisRare Diseases South Africa
Michelle ErweeTakeda, USA
Safiyya GassmanPfizer, 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