TrialSite News: “UK’s NICE Rejects bluebird bio: Questions Value Price Tag & Nominal Clinical Evidence While Sponsor Shocked”

A gene therapy for patients with transfusion-dependent beta thalassemia (TDT) didn’t fare well after a thorough review by the United Kingdom’s National Institute for Health and Care Excellence (NICE) turned down biotech firm bluebird bio’s Zynteglo (betibeglogene autotemcel; beti-cel). As it turns out, the UK research agency declared that the clinical trials involved with the Zynteglo investigation were considerably “small” while trial participants were not monitored for sufficient periods of time. Although the British research agency did acknowledge that perhaps at some point those patients treated with the gene therapy may not require blood transfusions, or at least need them not as frequently.

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