U.S. body says gene therapy may be more cost effective for spinal muscular atrophy

The Institute for Clinical and Economic Review (ICER) has issued a preliminary report evaluating two treatments for spinal muscular atrophy (SMA): nusinersen (Spinraza—Biogen), the only U.S.-approved treatment for the condition and one of the most expensive drugs on the market, and Novartis's experimental gene therapy, which is expected to re

The Institute for Clinical and Economic Review (ICER) has issued a preliminary report evaluating two treatments for spinal muscular atrophy (SMA): nusinersen (Spinraza—Biogen), the only U.S.-approved treatment for the condition and one of the most expensive drugs on the market, and Novartis's experimental gene therapy, which is expected to receive U.S. approval in early 2019. The Boston-based nonprofit says the gene therapy could be more cost effective once more is known about its U.S. price and long-term success rates. ICER's preliminary estimate is that Spinraza represents a cost of $728,000 per quality-adjusted life–year for individuals who are diagnosed before they show symptoms. Meanwhile, Novartis says the price of its gene therapy will ultimately be determined in negotiations with health plans, but it believes it would be cost-effective at $4 million–$5 million as a 1-time treatment. ICER said it instead used $2 million as an estimated price for the gene therapy. On that basis, when used in patients with symptomatic Type I SMA, it said the Novartis therapy represents a cost of $240,000 per quality-adjusted life–year.