The Food and Drug Administration recently approved betibeglogene autotemcel (Zynteglo), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions. Following its US regulatory approval, Bluebird Bio announced a price for Zynteglo of $2.8 million per patient. Prior to the manufacturer’s price announcement, we—the Institute for Clinical and Economic Review (ICER)—determined a value-based price range for Zynteglo between $2.1 and $2.4 million per patient using conventional cost-effectiveness analysis methods from the health system perspective.
