World’s Most Expensive Drug Revives Push for New Payment Models

A gene therapy considered to be the world’s most expensive drug raises questions about how to pay for it, and the federal agency overseeing Medicare and Medicaid could play a central role in ensuring patient access to potentially lifesaving medicines.

The Food and Drug Administration approved late last year CSL Behring’s hemophilia B gene therapy Hemgenix, a one-off infusion that costs $3.5 million a dose, making it the world’s most expensive drug. The field of gene therapies, which modify a person’s cells to treat or potentially cure a particular disease, is growing exponentially. Industry group Alliance for Regenerative Medicine has ...