After decades of research during which gene therapy was considered a distant goal, FDA-approved gene therapy options for hemophilia are now available in the United States. As these therapies enter real-world clinical practice, patients and care teams are navigating new access and coverage considerations that differ from more traditional treatment models.
The current coverage environment for gene therapy is shaped by a combination of innovative payment structures, existing insurance and Medicaid frameworks, and evolving medical policies. In some cases, this has resulted in restrictive coverage criteria, and administrative requirements that extend beyond FDA-labeled indications.
These dynamics can create challenges for individuals seeking to explore gene therapy as a potential treatment option, as well as for clinicians and hemophilia treatment centers managing both clinical care and administrative demands. Addressing these challenges requires thoughtful collaboration among patients, providers, payers, manufacturers, and advocacy organizations to ensure that access pathways support informed, patient-centered decision-making.
The National Bleeding Disorders Foundation (NBDF) reaffirms its commitment to a thoughtful approach to gene therapy in hemophilia care.
NBDF’s stance is grounded in shared decision-making between the person with hemophilia, their hematologist, and the multidisciplinary care team, including the expertise of hemophilia treatment centers (HTCs). We believe treatment decisions should be individualized, clinically appropriate, and informed by both the available evidence and patient preferences.
Gene therapy represents an important advancement in the hemophilia treatment landscape and may be an appropriate option for some individuals. At the same time, it is one of several therapeutic options, and its role in care should be determined through thoughtful, patient-centered discussion within the care team rather than through one-size-fits-all assumptions.
NBDF does not promote or discourage any single treatment. Our role is to support informed decision-making and evidence-based care, including the principle that coverage criteria for FDA-approved therapies should cite and align with FDA-labeled indications and current standards of care. When a therapy is prescribed consistent with its labeled indication and clinical judgment, coverage frameworks should allow appropriate consideration of that therapy without relying on criteria that fall outside the label or accepted clinical practice.
We recognize that coverage decisions and treatment selection are inherently individualized and complex. NBDF remains focused on ensuring that patients and care teams can engage in shared decision-making supported by clear, evidence-based policies that reflect approved indications and real-world clinical care.
We value our close collaboration with our Medical and Scientific Advisory Council (MASAC), HTCs, clinicians, hospitals, industry partners, and other stakeholders across the hemophilia community. NBDF remains committed to working collaboratively to support patient-centered care and the responsible integration of new therapies as the treatment landscape continues to evolve.
