Value-based pricing for gene therapy? Maybe not ready for hemophilia

BioMarin Pharmaceuticals' hemophilia A gene therapy valoctocogene roxaparvovec (valrox) could be on the market in mid-2020, making the question of how to structure reimbursement an immediate concern for payers. But given the alternative of paying a half-million dollars a year on factor replacement therapies, they may be willing to agree to a simple one-time price with biotech companies, said Matt Kapusta, CEO of UniQure, which is developing a hemophilia B drug called etranacogene dezaparvovec (etranadez).