Roctavian withdrawal: exploring the gap between innovation and healthcare system readiness in hemophilia A gene therapy

The voluntary withdrawal of valoctocogene roxaparvovec (Roctavian) represents an inflection point in hemophilia A gene therapy. Notably, this decision was not driven by safety or efficacy concerns, but by challenges to economic sustainability, market access, and healthcare system readiness. This paper examines the clinical evidence supporting gene therapy, including durability and safety, and highlights how uncertainty in long-term transgene expression complicates both clinical management and economic evaluation. The Roctavian case is not isolated but reflects a broader pattern observed across gene therapies, where high costs, limited uptake, and system-level constraints have led to market withdrawal or restricted access. These dynamics reveal a gap between scientific innovation and healthcare delivery, suggesting that the barriers to gene therapy are not biological but structural. Addressing this gap will require not only technological advances but also sustainable reimbursement models, coordinated long-term follow-up infrastructure, and healthcare systems capable of supporting advanced therapies beyond their approval.