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 out-of-pocket costs, 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.

RDI Essential Medicines Working group

Why Access matters

Currently, only a small fraction of rare diseases have approved therapies. While some treatments focus on disease management and supportive care, many go further—slowing, stopping, or preventing serious symptoms. Some therapies even transform lives by addressing the underlying causes of the disease.

The WHO Model List Model List of Essential Medicines contains medicines considered to be most effective and safe to meet the needs of a health system. The model list is a fundamental tool in facilitating access to medicines globally and national public health planning.
The Essential Medicines List (EML) serves as a critical advocacy tool to enhance the accessibility and affordability of these essential medicines. The EML also serves as foundational tool to improve access to essential diagnostics, specialist care, comprehensive treatment, and complementary support services in national settings. For PLWRD, access to these resources can mean the difference between effective care and ongoing challenges in managing their conditions.

Reflecting this committment, on April 24, 2023, RDI delivered a statement to the 24th WHO Expert Committee on the Selection and Use of Essential Medicines. Representing the global rare disease community, RDI called upon Member States to accelerate efforts toward Universal Health Coverage (UHC) and ensure greater inclusivity for persons living with rare diseases, particularly in accessing quality, affordable essential medicines and diagnostics.

Building on these advocacy efforts , in June 2024, RDI launched the Essential Medicines Working Group (EM-WG), composed of 12 RDI Member organizations representatives from 8 countries worldwide. The EM-WG provides a strategic opportunity for RDI members to leverage the EML’s potential and collaborate toward common objectives that benefit people living with rare diseases worldwide. The EM-WG aims to facilitate understanding of the WHO Model List of Essential Medicines (EML) as a tool to improve access to medicines.

Objectives of the Essential medicines wg

• Map experiences and interests within the rare disease community (RDI members and non-members) regarding the WHO Model Lists
• Analyze relevant applications (both successful and unsuccessful) to the EML, identifying pitfalls and success factors
• Collaborate with the EML Secretariat, gathering key questions and concepts to facilitate engagement with the WHO Expert Committee

Essential Medicines working group members

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IRDiRC/RDI Global working group

Between 2021-2023 RDI coordinated the International Rare Diseases Research Consortium (IRDiRC) RDI/Global Access Working Group to improve access to rare disease treatments, with a specific focus on low and middle-income countries (LMICs). The IRDiRC/RDI Global Access Working Group conducted a study to identify the barriers to accessing rare disease medicines. The study gathered real-world experiences and case studies on accessing medicines. Two case studies highlighted specific challenges: the first, focusing on cystinosis, involved interviews conducted in 11 countries, including Australia, Canada, Colombia, Ghana, Malaysia, Mexico, New Zealand, South Africa, Spain, Thailand, and Vietnam. The second, centered on cystic fibrosis, gathered insights from 7 countries—Brazil, Ireland, Israel, Italy, Latvia, South Africa, and Turkey. Across these diverse settings, a wide range of access challenges were reported, emphasizing the critical importance of early diagnosis and the availability of diagnostic tools to ensure timely treatment and effective care planning.

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Key resources

• Fact Sheet: WHO Essential Medicine List: Opportunities for Rare Diseases
  • English: Fact Sheet: WHO Essential Medicine List: Opportunities for Rare Diseases
  • Español/Spanish: Hoja Informativa: Lista de Medicamentos Esenciales OMS: Oportunidades para las Enfermedades Raras
  • Português/Portugese: Ficha de informação: Lista de Medicamentos Essenciais: Oportunidades para as Doenças Raras

• WHO Model List of Essential Medicines

• RDI Statement at the 24th WHO Expert Committee on Selection and Use of Essential Medecines-Open Session 24 April 2023, Geneva.

• Essential List of Medicinal Products for Rare Diseases – Recommendations from the IRDiRC Rare Disease Treatment Access Working Group. Orphanet Journal of Rare Diseases, 2021.