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An Ethical and Financial Obligation for Sickle Cell Disease Gene Therapy in the United States
New and life-saving therapy for patients with sickle cell disease (SCD) will soon be within reach. Vertex Pharmaceuticals (collaborating with CRISPR Therapeutics) and bluebird bio have each submitted biologic license applications to the U.S. Food and Drug Administration (FDA) for SCD gene therapy, and a rapid decision is expected. These therapies will join stem cell transplantation as the only curative options for adults with SCD.
A report from the Institute for Clinical and Economic Review predicts that the price of SCD gene therapy will be $2 million per patient (1). Given that more than 50 000 patients with SCD may be …
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