Posted by Michael Wonder on 12 Dec 2019
Value-based pricing for gene therapy? Maybe not ready for haemophilia.
12 December 2019 - Insurers grappling with the million-dollar-plus costs of gene therapy have touted "value-based" arrangements that allow payment over time or on the basis of patient outcomes.
But developers of hemophilia gene therapies believe they won't be widely subjected to them because the one-time treatments will be offset by long-term savings that could amount to tens of millions of dollars.
BioMarin Pharmaceuticals' haemophilia A gene therapy 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 haemophilia B drug called etranacogene dezaparvovec.
