Uncertainty Surrounds Payment Models for World’s Priciest Therapeutic

January 24, 2023

With the US Food and Drug Administration’s (FDA’s) approval last year of Hemgenix, a hemophilia B gene therapy by CSL Behring and most expensive drug in history, insurers are looking to the Centers for Medicare and Medicaid Services (CMS) for new payment models. The agency has long been used as a model for pricing decision-making, especially for novel, expensive medications.

According to Celine Castronuovo, “For example, conversations on how health insurers can pay for expensive gene therapies came up in 2019, as the CMS was determining coverage for CAR T-cell therapy, a novel treatment for leukemia and lymphoma. The agency eventually said it would cover the therapy when administered at health-care facilities enrolled in the FDA’s safety program for the therapy and ‘used for a medically accepted indication.’ Policy analysts say there’s no clear one-size-fits all solution to pay for expensive gene therapies. But the CMS and other payers have explored some models already—such as subscription payments to manufacturers for therapy access, or coverage based on the value the treatment provides to patients.”

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