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As the U.S. health care system braces for expensive gene therapies, a new analysis suggests that a forthcoming treatment for a rare disease called beta thalassemia would be cost-effective — even if priced at $2.1 million.

The therapy, which is being developed by Bluebird Bio, is still being reviewed by the U.S. Food and Drug Administration and approval is not expected for several months. Although a formal price has not been disclosed, the company has previously indicated it was looking to charge $2.1 million for its treatment using a five-year installment plan.


Based on that information, the Institute for Clinical and Economic Review determined the therapy would be fairly priced when compared with the standard of care for the inherited blood disorder, which affects an estimated 1,500 to 2,000 Americans. Typically, patient care includes regular blood transfusions and chelation therapy, and the overall costs can add up to about $1.4 million over a patient’s lifetime.

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