Dataset Information

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

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.

Objective

To compare gene therapy versus standard of care (SOC) in patients with SCD by using conventional CEA and DCEA.

Design

Markov model.

Data sources

Claims data and other published sources.

Target population

Birth cohort of patients with SCD.

Time horizon

Lifetime.

Perspective

U.S. health system.

Intervention

Gene therapy at age 12 years versus SOC.

Outcome measures

Incremental cost-effectiveness ratio (ICER) (in dollars per quality-adjusted life-years [QALYs] gained) and threshold inequality aversion parameter (equity weight).

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.

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.

Limitation

Benchmark equity weights (as opposed to SCD-specific weights) were used to interpret DCEA results.

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.

Primary funding source

Yale Bernard G. Forget Scholars Program and Bunker Endowment.

SUBMITTER: Goshua G

PROVIDER: S-EPMC10370480 | biostudies-literature | 2023 Jun

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

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

Goshua George G Calhoun Cecelia C Ito Satoko S James Lyndon P LP Luviano Andrea A Krishnamurti Lakshmanan L Pandya Ankur A

Annals of internal medicine 20230530 6

<h4>Background</h4>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.<h4>Objective</h4>To compare gene therapy versus standard of care (SOC) in patients with SCD by using conventional CEA and DCEA.<h4>Design</h4>Markov model.<h4>Data sources</h4>Claims data and other published sources.< ...[more]

PMID: 37247420

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Project description:BackgroundSickle cell disease (SCD) and its complications contribute to high rates of morbidity and early mortality and high cost in the United States and African heritage community.ObjectiveTo evaluate the cost-effectiveness of gene therapy for SCD and its value-based prices (VBPs).DesignComparative modeling analysis across 2 independently developed simulation models (University of Washington Model for Economic Analysis of Sickle Cell Cure [UW-MEASURE] and Fred Hutchinson Institute Sickle Cell Disease Outcomes Research and Economics Model [FH-HISCORE]) using the same databases.Data sourcesCenters for Medicare & Medicaid Services claims data, 2008 to 2016; published literature.Target populationPersons eligible for gene therapy.Time horizonLifetime.PerspectiveU.S. health care sector and societal.InterventionGene therapy versus common care.Outcome measuresIncremental cost-effectiveness ratios (ICERs), equity-informed VBPs, and price acceptability curves.Results of base-case analysisAt an assumed $2 million price for gene therapy, UW-MEASURE and FH-HISCORE estimated ICERs of $193 000 per QALY and $427 000 per QALY, respectively, under the health care sector perspective. Corresponding estimates from the societal perspective were $126 000 per QALY and $281 000 per QALY. The difference in results between models stemmed primarily from considering a slightly different target population and incorporating the quality-of-life (QOL) effects of splenic sequestration, priapism, and acute chest syndrome in the UW model. From a societal perspective, acceptable (>90% confidence) VBPs ranged from $1 million to $2.5 million depending on the use of alternative effective metrics or equity-informed threshold values.Results of sensitivity analysisResults were sensitive to the costs of myeloablative conditioning before gene therapy, effect on caregiver QOL, and effect of gene therapy on long-term survival.LimitationThe short-term effects of gene therapy on vaso-occlusive events were extrapolated from 1 study.ConclusionGene therapy for SCD below a $2 million price tag is likely to be cost-effective when applying a societal perspective at an equity-informed threshold for cost-effectiveness analysis.Primary funding sourceNational Heart, Lung, and Blood Institute.

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Dataset Information

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

Background

Objective

Design

Data sources

Target population

Time horizon

Perspective

Intervention

Outcome measures

Results of base-case analysis

Results of sensitivity analysis

Limitation

Conclusion

Primary funding source

Publications

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

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