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March 22, 2021

Gene Therapy for Sickle Cell Disease—A Debt to Be Paid

Author Affiliations
  • 1Albert Einstein College of Medicine and Montefiore Medicine, Bronx, New York
JAMA Pediatr. 2021;175(6):565-566. doi:10.1001/jamapediatrics.2020.7147

Eleven months into the global coronavirus disease 2019 (COVID-19) pandemic that has disproportionately sickened and killed people of color and the poor in the US, in this issue of JAMA Pediatrics DeMartino et al1 have delivered what amounts to an early warning about what may well be the next manifestation of the systemic disparities that continue to plague health care in the US. In their assessment of the budget impact and affordability of a gene therapy for severe sickle cell disease on the 10 state Medicaid programs with the highest prevalence of the heritable, life-shortening condition, the authors describe that rarest of all therapeutic interventions—a potential cure. That’s the good news. The bad news, or should I say, the old news, is the cost of the treatment, which is likely to be more than $1 million per patient, and could “force Medicaid plans to implement strategies to limit access.” Considering the fact that 6 of the 10 states in the analysis chose not to expand Medicaid coverage under the Patient Protection and Affordable Care Act,2 limiting access to a treatment with such a large price seems more likely than the “unclear” possibility the article postulates. With more than half of the 100 000 people in the US with SCD enrolled in Medicaid,3 that system’s policies are central to any effort to ameliorate the impact of the disease.

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