Zynteglo Is Cost Effective At $2.1M, ICER Proposes; Validation For Bluebird’s Pivot To US?
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.
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The firm is laying off a big piece of its workforce and deprioritizing some investments to free up capital for potential upcoming launches, but that could add commercial risks.
Bluebird bio’s decision to focus on the US market is being seen as a broader signal of the need for changes in market access policies to enable gene therapies to be made available for European patients. Only collaborative work will create solutions acceptable to payers and companies, and finding an acceptable price that both can live with will be crucial.
Results from the US Food and Drug Administration’s RCT DUPLICATE demonstration are in. The findings both legitimize certain real-world evidence studies and also highlight the challenges involved in using RWE to replicate randomized clinical trials.