Gary Puckrein sees a dilemma coming that has him excited, and at the same time, worried.
In the next year or so, U.S. regulators may approve gene therapies – from Vertex Pharmaceuticals and Bluebird Bio – to treat sickle cell disease, an inherited group of blood disorders that affect an estimated 100,000 Americans. The condition is particularly prevalent among Black people, with 1 of every 365 Black individuals in the U.S. born with the condition, which can cause significant pain, infections, and lead to an early death.
Any treatment would be welcome news, especially since a gene therapy holds the promise of eradicating the illness with potentially just one treatment. But gene therapies are widely expected to come with a high price tag — and it’s estimated that roughly 40% of sickle cell patients are covered by Medicaid. That is raising concern that state Medicaid officials may soon face a struggle for coverage.
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