In September 2025, the World Health Organization (WHO) made historic updates to its Essential Medicines List (EML) for adults and children, marking a major step forward for people with hemophilia and von Willebrand disease (VWD). The revisions align the list with modern treatment standards and prioritize safe, effective, and preventive therapies over outdated and risky ones.

Why the Update Matters

Before the change, the WHO’s list reflected 20th-century treatment approaches, relying heavily on plasma-derived products. These older therapies carried serious risks—especially in lower-income countries where blood screening may not prevent transmission of HIV and hepatitis. Modern best practices emphasize prophylactic (preventive) treatment rather than treating bleeds after they occur, a shift that the new EML finally recognizes.

Being on the EML helps governments justify funding for these therapies and often prompts pharmaceutical companies to provide tiered pricing or access programs, improving affordability and access worldwide. For rare diseases like hemophilia, EML inclusion is a crucial advocacy and equity tool. While these changes do not directly impact those living in developing countries where access to medication is very limited, it could help to pave the way in advocacy efforts to improve access and affordability. 

The WHO’s 2025 EML update is a landmark victory for the bleeding disorders community. It replaces outdated treatments with modern, preventive, and safer therapies, paving the way for better access, improved safety, and global health equity for individuals with hemophilia and VWD. 

Source: https://www.bleeding.org/news/who-updates-essential-medicines-list-to-improve-bleeding-disorders-care