New way for states to cover pricey gene therapies will start with sickle cell disease
By Nada Hassanein,
New Jersey Monitor
| 03. 14. 2024
The U.S. Food and Drug Administration late last year approved two breakthrough gene therapies for sickle cell disease patients. Now a new federal program seeks to make these life-changing treatments available to patients with low incomes — and it could be a model to help states pay for other expensive therapies.
The new sickle cell treatments have brought hope to those with the debilitating blood disorder, which is hereditary and disproportionately affects Black people. But the therapies come with a price tag of as much as $3 million for a course of treatment, which can take up to a year. Despite those high upfront costs, cell and gene therapies have the potential to reduce health care spending over time by addressing the underlying cause of the disease.
Under the so-called Cell and Gene Therapy Access Model, the federal government will negotiate discounts with sickle cell drug manufacturers Vertex Pharmaceuticals, CRISPR Therapeutics and Bluebird Bio on behalf of state Medicaid agencies, which provide health care coverage to low-income patients. To participate, state Medicaid agencies must agree to prices based on those negotiations...
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Image by Ed Uthman from Flickr
Last year, the U.S. Food and Drug Administration (FDA) approved two gene therapy procedures that can treat and, in some cases essentially cure sickle cell disease, a genetic blood disorder that causes pain and...