ArticleAnnals of internal medicine2023
Distributional Cost-Effectiveness of Equity-Enhancing Gene Therapy in Sickle Cell Disease in the United States.
Article in Annals of internal medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
20 citing papers in PubMed, 2 syntheses or guidelines pooled it, 35 citations in OpenAlex.
- Methodological heterogeneity and equity challenges of distributional cost-effectiveness analysis in healthcare: a systematic review from 2017 to 2025.Archives of public health = Archives belges de sante publique · 2026Pooled it
- Balancing benefits and burdens: a systematic review on ethical and social dimensions of gene and cell therapies for hereditary blood diseases.BMC medical ethics · 2025Pooled it
- Article
- A Threshold Inequality Aversion Parameter Approach to Interpret Distributional Cost-Effectiveness Analysis Results.Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research · 2026Article
- Cost-effectiveness of gene therapy for sickle cell disease in Uganda: tailoring high-income evidence to Uganda's context.Gene therapy · 2026Article
- Article
- How to Fix a Broken Health Care System: Pathways to Maximize Health and Well-being for All.Diabetes care · 2026Review
- Article
- The Impact of Alteplase Coverage on Health Equity for the Treatment of Ischemic Stroke in the USA: A Distributional Cost-Effectiveness Analysis.Applied health economics and health policy · 2025Article
- Distributional Cost-Effectiveness Analysis in Genomic Medicine: Considerations for Addressing Health Equity.Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research · 2025Article
- Prophylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in People Living With Severe Hemophilia A : A Cost-Effectiveness Analysis.Annals of internal medicine · 2025Article
- Medicaid Expansion Among Nonelderly Adults and Cardiovascular Disease: Efficiency Vs. Equity.The Milbank quarterly · 2025Article
- Genome editing strategies for targeted correction of β-globin mutation in sickle cell disease: From bench to bedside.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Using Distributional Cost-Effectiveness Analysis to Improve Equity and Efficiency.Journal of the American College of Cardiology · 2025Article
- The Impact of Tocilizumab Coverage on Health Equity for Inpatients with COVID-19 in the USA: A Distributional Cost-Effectiveness Analysis.PharmacoEconomics · 2025Article
- Modeling the Population Equity of Alzheimer Disease Treatments in the US.JAMA network open · 2024Article
- Cost-Effectiveness of Lovotibeglogene Autotemcel (Lovo-Cel) Gene Therapy for Patients with Sickle Cell Disease and Recurrent Vaso-Occlusive Events in the United States.PharmacoEconomics · 2024Article
- Gene Therapy Versus Common Care for Eligible Individuals With Sickle Cell Disease in the United States : A Cost-Effectiveness Analysis.Annals of internal medicine · 2024Article
- Health Equity Considerations in Cost-Effectiveness Analysis: Insights from an Umbrella Review.ClinicoEconomics and outcomes research : CEOR · 2024Review
- Framework for determining the optimal course of action when efficiency and affordability measures differ by perspective in cost-effectiveness analysis-with an illustrative case of HIV treatment in Mozambique.Cost effectiveness and resource allocation : C/E · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 2 institutions in 1 country.
Funding
Abstract
backgroundGene 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.
objectiveTo compare gene therapy versus standard of care (SOC) in patients with SCD by using conventional CEA and DCEA.
designMarkov 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.
interventionGene 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.
conclusionGene 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.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.