Bluebird Could Find US Tailwind For Zynteglo After ICER Report
The cost-effectiveness watchdog now assumes an upfront $2.1m payment instead of five annual payments. The company switched gears to the US after the TDT gene therapy stumbled in Europe.
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According to a study presented at the American Society of Hematology, based on existing gene therapy prices, equitable use of sickle cell disease gene therapy requires expanding into patients who are younger and have less severe disease.
Although there were no cases of hematological malignancy reported and no evidence of insertional oncogenesis in patients treated with beti-cel, FDA raises a theoretical risk due to delayed platelet engraftment, as well as malignancies seen in other lentiviral vector programs.
Draft evidence report from the Institute for Clinical and Economic Review estimates the potentially curative rare disease treatment could offset about $1.4m in health system costs over a lifetime.