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As the U.S. health care system prepares for expensive gene therapies, a preliminary analysis suggests that a pair of forthcoming treatments for sickle cell disease would be cost-effective if priced as high as $1.9 million, depending upon certain variables.

The medications — one is being developed by Bluebird Bio and the other by a partnership between Vertex Pharmaceuticals and CRISPR Therapeutics — have not yet been approved by the Food and Drug Administration. As a result, pricing has not been disclosed, but analyst estimates have suggested the treatments may be priced at roughly $2 million.


Based on that information, the Institute for Clinical and Economic Review determined the therapies would be fairly priced when compared with the standard of care, which involves both managing a chronic disease and acute bouts of illness. In 2016, there were 134,000 hospital stays in the U.S. that cost $811.4 million, according to data from the Agency for Healthcare Research and Quality.

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