The nation’s Medicare and Medicaid agency is mulling how to pay for a costly gene therapy procedure that could potentially cure sickle cell disease for 100,000 Americans.
A clinical trial underway at the National Institutes of Health already shows promise to repair the gene that causes sickle cell disease, a painful, inherited blood disorder. It’s frequently mentioned by NIH Director Francis S. Collins when highlighting the work of the agency, especially before congressional spending panels. CRISPR Therapeutics and Vertex Announce had their application to treat sickle cell fast tracked through the Food and Drug Administration’s review process in January.
But ...