How much can a company charge for a one-time therapy that may cure a debilitating genetic disease? The answer for drugmaker Bluebird Bio: $2.8 million.
of whom live in low- and middle-income countries. Some of these patients struggle to access basic blood transfusions, suggesting gene therapy will be impossible for these patients, Hadjidemetriou said.
ICER also said beti-cel met value thresholds for cost-effectiveness at Bluebird's initial suggested price tag of $2.1 million, if this scenario included a payback option for patients who did not maintain transfusion independence. In addition, among the 1500 or so patients in the US with beta-thalassemia who depend on frequent infusions, not all will seek the gene therapy.
And in September, one month after the approval of beti-cel, the FDA granted an accelerated approval to a second gene therapy from Bluebird — elivaldogene autotemcel , also known as eli-cel, which slows the progression of neurologic dysfunction in boys ages 4-17 with early, active cerebral adrenoleukodystrophy, also known as CALD.And there's more.
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