WHO Updates Essential Medicines List to Expand Access to Modern Hemophilia Treatments
The World Health Organization (WHO) has revised its Essential Medicines List (EML) and Essential Medicines List for Children (EMLc), a milestone driven by the World Federation of Hemophilia (WFH) and its global partners. These changes bring the EMLs in line with international clinical guidelines for hemophilia and von Willebrand Disease (VWD), creating new opportunities for access to safer, more effective therapies for people with bleeding disorders (PWBDs).
The WHO EML serves as a cornerstone for national governments when making decisions on financing and coverage of essential medicines. With the updated listings, payers and policymakers will now be guided by recommendations that reflect the current global standard of care—prophylaxis—rather than outdated treatments.
Key changes include:
- Addition of emicizumab (bi-specific monoclonal antibody) to the core list,
- Addition of recombinant FVIII and FIX clotting factor concentrates (CFCs) to the core list,
- Transfer of plasma-derived FVIII and FIX CFCs, as well as desmopressin, from complementary to core lists, and
- Removal of cryoprecipitate (both pathogen-reduced and non-pathogen-reduced) and Factor IX Complex (prothrombin complex concentrate [PCC]) as treatment options.
The elimination of cryoprecipitate is particularly significant, as it cannot be used for prophylaxis and poses risks of bloodborne infection transmission. These revisions help ensure health systems prioritize virally safe and effective therapies, supporting equitable access worldwide.
Cesar Garrido, president of the WFH, emphasized the broader impact: “The updates to the WHO EML mean that thousands of PWBDs now have greater hope for a better future. The WFH is proud to have led a united global effort to make this milestone possible. Together, we are shaping a future where our vision of Treatment for All can become a reality.”
For payers and managed care experts, the updated WHO EML strengthens the evidence base for coverage decisions, reinforcing the clinical and policy imperative to move away from cryoprecipitate and toward modern prophylactic therapies.
Reference
WFH mobilizes global community to achieve milestone revisions to WHO EML. PR Newswire. September 12, 2025. Accessed September 30, 2025. https://www.prnewswire.com/news-releases/wfh-mobilizes-global-community-to-achieve-milestone-revisions-to-who-eml-302555143.html
