ICER Report At a Glance: Beti-Cel for Beta Thalassemia

July 19, 2022

Beta Thalassemia is a blood disorder that results in dangerously low levels of functional hemoglobin and requires expensive lifelong medical care, including regular blood transfusions. Beti-cel is a gene therapy by Bluebird Bio that can potentially cure the disorder by genetically modifying a patient’s own hematopoietic stem cells (HSCs). The Institute for Clinical and Economic Review (ICER) has just released its assessment of Beti-cel, finding that it is effective and could save money on treatment, but more comprehensive long-term data would be necessary to determine the degree of the net health benefit over the current standard of care.

According to ICER, “In summary, despite remaining uncertainties, the evidence suggests that beti-cel provides net health benefits to patients with TDT. Given the high annual costs of standard care, cost-effectiveness modeling finds that this new treatment meets commonly accepted value thresholds at an anticipated price of $2.1 million with an 80% payback option for patients who do not achieve and maintain transfusion independence over a five-year period. However, if half of the lifetime cost savings from the therapy are returned to society, then discounts between 15% and 38% off the anticipated price are required to meet commonly accepted thresholds”

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(Source: ICER, July 19th, 2022)

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