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Distributional Cost-Effectiveness of Equity-Enhancing Gene Therapy in Sickle Cell Disease in the United States.

Authors :
Goshua G
Calhoun C
Ito S
James LP
Luviano A
Krishnamurti L
Pandya A
Source :
Annals of internal medicine [Ann Intern Med] 2023 Jun; Vol. 176 (6), pp. 779-787. Date of Electronic Publication: 2023 May 30.
Publication Year :
2023

Abstract

Background: Gene therapy is a potential cure for sickle cell disease (SCD). Conventional cost-effectiveness analysis (CEA) does not capture the effects of treatments on disparities in SCD, but distributional CEA (DCEA) uses equity weights to incorporate these considerations.<br />Objective: To compare gene therapy versus standard of care (SOC) in patients with SCD by using conventional CEA and DCEA.<br />Design: Markov model.<br />Data Sources: Claims data and other published sources.<br />Target Population: Birth cohort of patients with SCD.<br />Time Horizon: Lifetime.<br />Perspective: U.S. health system.<br />Intervention: Gene therapy at age 12 years versus SOC.<br />Outcome Measures: Incremental cost-effectiveness ratio (ICER) (in dollars per quality-adjusted life-years [QALYs] gained) and threshold inequality aversion parameter (equity weight).<br />Results of Base-Case Analysis: Gene therapy versus SOC for females yielded 25.5 versus 15.7 (males: 24.4 vs. 15.5) discounted lifetime QALYs at costs of $2.8 million and $1.0 million (males: $2.8 million and $1.2 million), respectively, with an ICER of $176 000 per QALY (full SCD population). The inequality aversion parameter would need to be 0.90 for the full SCD population for gene therapy to be preferred per DCEA standards.<br />Results of Sensitivity Analysis: SOC was favored in 100.0% (females) and 87.1% (males) of 10 000 probabilistic iterations at a willingness-to-pay threshold of $100 000 per QALY. Gene therapy would need to cost less than $1.79 million to meet conventional CEA standards.<br />Limitation: Benchmark equity weights (as opposed to SCD-specific weights) were used to interpret DCEA results.<br />Conclusion: Gene therapy is cost-ineffective per conventional CEA standards but can be an equitable therapeutic strategy for persons living with SCD in the United States per DCEA standards.<br />Primary Funding Source: Yale Bernard G. Forget Scholars Program and Bunker Endowment.<br />Competing Interests: Disclosures: Disclosures can be viewed at www.acponline.org/authors/icmje/ConflictOfInterestForms.do?msNum=M22-3272.

Details

Language :
English
ISSN :
1539-3704
Volume :
176
Issue :
6
Database :
MEDLINE
Journal :
Annals of internal medicine
Publication Type :
Academic Journal
Accession number :
37247420
Full Text :
https://doi.org/10.7326/M22-3272