Radar on Drug Benefits

The FDA on Sept. 29 approved Relyvrio (sodium phenylbutyrate/taurusodiol), making it only the third medication to treat patients with amyotrophic lateral sclerosis (ALS). While ALS advocates and some clinicians applauded the agency’s decision, other providers have questioned the drug’s effectiveness, and some experts believe the drug is not cost-effective with its approximately $158,000 annual wholesale acquisition cost (WAC). 

For instance, the nonprofit Institute for Clinical and Economic Review (ICER) found that Relyvrio, which was formerly known as AMX0035, would be cost-effective if it was priced at between $9,100 and $30,600 per year. And Bruce Booth, a former scientist who’s now a partner at the Atlas Venture biotechnology venture capital firm, wrote on Twitter that the $158,000 price for Relyvrio was “even more egregious than I thought!” 

The FDA’s controversial accelerated approval of Biogen Inc.’s Aduhelm (aducanumab) has yielded new fallout, as members of the U.S. House of Representatives have introduced a bill seeking to unwind CMS’s national coverage determination that limited access to the Alzheimer’s treatment — and prevent CMS from issuing restrictive NCD decisions on future drugs.  

D.C. insiders tell AIS Health, a division of MMIT, that the bipartisan bill’s fate will likely depend on the outcome of the upcoming midterm elections. They add that the NCD bill represents a renewed effort by the pharmaceutical industry to flex its lobbying muscle after it faced an unprecedented defeat with the passage of Medicare drug price negotiation as part of the Inflation Reduction Act.