We told you last week about a new gene therapy from Novartis, just approved by the Food and Drug Administration, that will treat a rare and fatal childhood disease called spinal muscular atrophy — and will cost $2.1 million.
The one-dose treatment’s eye-popping price has sparked a national debate, but STAT’s Damian Garde writes that, for parents of children affected by the disease, the cost is well worth it — and could actually save them money as a one-time treatment compared with expensive hospitalizations or an alternative drug on the market that costs $375,000 a year.
“Novartis has argued that its therapy, approved last month as Zolgensma, is cost-effective even at $2.1 million,” Garde writes. “SMA is a progressive disease that gradually erodes muscular function. Patients often need wheelchairs and at-home care, and many suffer from lung infections that require hospitalization, all of which can add up to far more than the cost of Zolgensma.”
On the other hand, some experts worry that “if drug companies continue to price each new therapy at a premium to the last, the system might buckle beneath the cost,” Garde says. If gene therapies for more common diseases are priced like Novartis’s drug, “the aggregate cost, passed down through insurers and across the health care system, could become untenable.”