WHO updates hemophilia therapies on its essential medicines list
This move may improve treatment access for patients worldwide
With encouragement from the World Federation of Hemophilia (WFH) and its partners, the World Health Organization (WHO) updated its essential medicines lists to better align with international clinical guidelines for managing hemophilia and other bleeding disorders.
These lists — the Essential Medicines List (EML) and the EML for Children (EMLc) — serve as a comprehensive directory of medicines deemed necessary for public health systems worldwide. Governments use them to prioritize medication availability and guide pricing and reimbursement decisions.
The recent updates ensure that these decision-makers have access to the latest, expert-backed recommendations on hemophilia care. WFH hopes this will lead to more equitable access to hemophilia treatment.
“The updates to the WHO EML mean that thousands of [people with bleeding disorders] now have greater hope for a better future,” Cesar Garrido, WFH president, said in an organization press release. “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.”
The need for an update
Hemophilia is a rare disease where a lack of blood clotting factors leaves individuals susceptible to excessive, prolonged, or spontaneous bleeding.
Various prophylactic therapies are available that, when taken on a routine basis, prevent these bleeds from happening or reduce their frequency. Prophylaxis is now the internationally recognized standard of care for hemophilia.
That includes factor replacement therapies, which supply the body with a version of the missing clotting factor, and treatments like Hemlibra (emicizumab-kxwh), which work in different ways to promote blood clotting.
On the EML, core medicines are considered the most effective, safe, and cost-effective options for managing a condition. They are the minimum medicines that any basic healthcare system should have, at prices individuals and healthcare systems can afford.
Complementary medicines are also highly effective but may be less accessible due to higher costs or the need for specialized facilities, equipment, or training.
The first EML was published in 1977, and the first EMLc in 2007. An expert committee revises them every two years. After the last EML revisions in 2023, WFH felt that the lists didn’t adequately reflect hemophilia care standards. It outlined these concerns in a comment published in The Lancet Haematology.
Over the past two years, WFH has directly communicated with the WHO to advocate for changes in the 2025 revisions to better support the global hemophilia community.
The WHO expert committee convened earlier this year to discuss the changes that would be made. WFH made a statement in favor of updating the hemophilia medicines during the public session of that meeting.
2025 EML revisions
The 2025 update added lab-made (recombinant) factor replacement therapies and Hemlibra to the core list of essential medicines on the EML and EMLc. Human blood-derived factor replacement treatments and desmopressin were moved from the complementary list to the core list.
Cryoprecipitate, a human blood-derived product that has fallen out of favor because it is not considered safe, was removed as a hemophilia treatment. Prothrombin complex concentrates (factor IX complex), previously listed as a therapeutic alternative to factor replacement therapy, were also deleted.
“The WHO’s decision to update the Essential Medicines Lists marks a major step forward in ensuring access to modern, effective treatments for people with bleeding disorders worldwide,” said Glenn Pierce, MD, PhD, vice president, medical, at WFH.
Various clinicians, hemophilia treatment centers, scientific and patient advocacy organizations supported WFH’s efforts.
A total of 115 WFH national member organizations signed a joint letter of support to the WHO director-general, with another similar letter signed by more than 120 expert clinicians worldwide. Various other individual groups sent their own letters. Pierce credits those collaborators with making the updates possible.
“Together, we have helped align global policy with the best clinical standards, making the EML a strong advocacy tool to countless patients and families in countries that don’t have full access to these therapies,” Pierce said.
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