Distributional Cost-Effectiveness of Equity-Enhancing Gene Therapy in Sickle Cell Disease in the United States and Distributional Cost-Effectiveness: Putting Money Where Your Mouth Is on Health Equity

Gene therapy is a potential cure for sickle cell disease (SCD). Conventional cost-effectiveness analysis (CEA) does not capture the effects of treatment on disparities in SCD. This study compared gene therapy versus standard of care in patients with SCD by using conventional CEA and distributional CEA, the latter of which gave extra credit to interventions with measured effects on reducing health disparities. The accompanying editorial discusses the findings and the need to raise the profile of this methodological approach.

Use this article and editorial to:

  • Review currently available therapy for SCD. This In the Clinic article may be helpful.
  • How does distributional CEA differ from traditional CEA? What are the advantages and disadvantages of each method?
  • A commonly used threshold for cost-effectiveness is less than $100,000 per quality-adjusted life-year gained. Do you think this is a reasonable threshold in the United States? How about in less affluent parts of the world?

Annals of Internal Medicine is the premier internal medicine academic journal published by the American College of Physicians (ACP). It is one of the most widely cited and influential specialty medical journals in the world.

Back to the July 2023 issue of ACP IMpact