America’s New Most Expensive Drug Reignites Debate Over How to Price Gene Therapies
The FDA on Nov. 22 approved Hemgenix (etranacogene dezaparvovec-drlb), the first gene therapy for adults with hemophilia B, a genetic bleeding disorder. CSL Behring, the medication’s commercial rights holder, set a list price of $3.5 million, making it the most expensive drug in the U.S.
That price exceeds the $2.96 million health benefit price threshold (HBPB) that the nonprofit Institute for Clinical and Economic Review (ICER) determined recently in an evidence report, meaning it is not cost-effective according to the nonprofit’s guidelines.
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