FDA Approves Sickle Cell Gene Therapies; Federal Program to Aid Low-Income Access

March 19, 2024
FDA Approves Sickle Cell Gene Therapies; Federal Program to Aid Low-Income Access
  • The U.S. FDA has approved new gene therapies for sickle cell disease.

  • A federal program is set to make these therapies accessible to low-income patients through Medicaid.

  • The program will negotiate drug discounts and is slated to begin next year.

  • Gene therapies have a high upfront cost, up to $3 million, but may save on long-term health care expenses.

  • Initial focus is on sickle cell treatments, with potential to expand to other conditions.

  • Sickle cell disease predominantly affects Black Americans, among others, with an estimated 100,000 affected in the U.S.

  • Illinois has committed to the program, while other states are in discussions, but some may struggle to participate without Medicaid expansion.

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