Decades of advocacy spearheaded by the World Federation of Hemophilia (WFH) have resulted in a victory for the global bleeding disorders community. The World Health Organization (WHO) Model List of Essential Medicines (EML) has been updated to include safer and more effective medicines for the treatment of bleeding disorders.
Emicizumab was added for patients with hemophilia A, both with and without inhibitors. Recombinant clotting factor concentrates were added to the core list, and plasma-derived clotting factor concentrates were moved from the complementary list to the core list. Factor IX complex (also known as prothrombin complex concentrate (PCC), a product containing factor IX along with other clotting factors, was removed as a therapeutic alternative to pure factor IX due to safety concerns and the preference for more precise, modern treatments. Pathogen-reduced and standard cryoprecipitate remain on the list, but they are no longer indicated for routine treatment of hemophilia or von Willebrand disease (VWD). Desmopressin, used to treat mild hemophilia and types 1 and 2 VWD, was moved from the complementary list to the core list.
Inclusion in the EML’s core list has far-reaching effects. Many countries base their national formularies on the WHO EML, and once listed, the medicines are more likely to be integrated into national treatment guidelines. Pharmaceutical companies often engage in tiered pricing, access agreements, or donations for medication that is on the EML. In countries with public insurance or universal coverage, being on the EML strengthens the case for reimbursement.
“We have helped align global policy with the best clinical practice standards, making the EML a strong advocacy tool to countless patients and families in countries that don’t have full access to these therapies,” said Glenn Pierce, MD, PhD, vice president, medical, WFH said, in a statement issued by WFH.
WFH played a central role in advocating for these treatments to be recognized as essential by the WHO. A letter of support to the WHO director general was co-signed by 115 national member organizations of WFH. Other patient and professional organizations strengthened the advocacy efforts, including the National Bleeding Disorders Foundation (NBDF). NBDF and Hemophilia Federation of America (HFA) issued a joint statement raising concerns about WHO’s previous listing of cryoprecipitate, calling for the prioritization of safer alternatives. Rare Diseases International (RDI) formed an Essential Medicines Working Group to mobilize patient organizations around the world to engage with the EML process.
“This is a huge step forward for bleeding disorders care,” said Dawn Rotellini, chief operations officer, NBDF. “NBDF has consistently supported WFH’s effort in getting these changes made to the EML. It’s a victory for all people living with bleeding disorders throughout the world.”
