The EHC joins the International Society on Thrombosis and Haemostasis (ISTH), European Association for Haemophilia and Allied Disorders (EAHAD), World Federation of Hemophilia (WFH), and other leading international organisations in issuing a statement and urgent call-to-action for a continued commitment to innovation and access in haemophilia care.

Over the past decade, haemophilia treatment has advanced significantly from traditional intravenous factor replacement to subcutaneous substitution therapies, rebalancing agents, and gene therapies. These innovations offer significantly improved outcomes and quality of life for people with haemophilia, including those with mild or moderate disease, women, and individuals with related bleeding disorders.

In recent years, three gene therapies—ROCTAVIAN, HEMGENIX, and BEQVEZ—have received regulatory approval, marking a historic milestone. Concerningly, several companies have recently discontinued, halted or scaled back gene therapy programs, citing limited interest from the community and commercial uncertainty.

Claims of disinterest from the haemophilia community are misleading and jeopardise the progress of gene therapy advancements. Current slow adoption is significantly linked to commercial uncertainties, fragmented health-care systems, reimbursement challenges, long-term outcome uncertainties, and systemic barriers, not a lack of interest and/or need.

More than ever, scientific-medical societies and patient communities call for and support the development of safe, effective gene and other novel haemophilia therapies.

We urge all stakeholders—industry, regulators, payers, funders, researchers, clinicians, and patients—to:

• Sustain research, investment and clinical developments in innovative and advanced therapies, including gene therapies and non-replacement therapies;
• Optimise and expand access to existing treatments worldwide;
• Decrease unnecessary burdens in clinical development and drug approval processes
• Develop clear, fair reimbursement frameworks; and
• Strengthen global collaboration, research and education.

The future of haemophilia care depends on collective action. Innovation and access to treatments must remain a priority so that all people with haemophilia, wherever they live, can benefit from safe, effective, and life-changing therapies.

In addition to ISTH, EAHAD and WFH, this statement is endorsed by:

• American Society of Gene & Cell Therapy (ASGCT)
• Asian-Pacific Society on Thrombosis and Hemostasis (APSTH)
• Association for Haemophilia and Allied Disorders-Asia Pacific (AHADAP)
• European Haemophilia Consortium (EHC)
• European Society for Gene and Cell Therapy (ESGCT)
• Grupo Cooperativo Latinoamericano de Hemostasia y Trombosis (Grupo CLAHT)
• National Bleeding Disorders Foundation (NBDF)