ArticlePharmacoEconomics2017
Economic Evaluation in Duchenne Muscular Dystrophy: Model Frameworks for Cost-Effectiveness Analysis.
Article in PharmacoEconomics, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers, 3 of them syntheses that pooled it.
What it found
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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.
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Who cites it
21 citing papers in PubMed, 3 syntheses or guidelines pooled it.
- The Economic Burden of Duchenne Muscular Dystrophy: A Systematic Review.PharmacoEconomics · 2026Pooled it
- Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments.Orphanet journal of rare diseases · 2021Pooled it
- Characterizing health state utilities associated with Duchenne muscular dystrophy: a systematic review.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2020Pooled it
- Disease progression and economic burden of duchenne muscular dystrophy: A retrospective study using Swedish register data.Journal of neuromuscular diseases · 2026Article
- Systematic Literature Reviews of Health-State Utility Values, Costs, and Healthcare Resource Use in Duchenne Muscular Dystrophy.Journal of health economics and outcomes research · 2026Article
- Development of a cost-effectiveness analysis model for Duchenne muscular dystrophy utilizing the national registry in Japan.Scientific reports · 2025Article
- Economic Evaluations of Treatments for Duchenne Muscular Dystrophy: The Caregiver QALY Trap.PharmacoEconomics · 2024Article
- Developing a Natural History Model for Duchenne Muscular Dystrophy.PharmacoEconomics - open · 2024Article
- Estimating health state utilities in Duchenne muscular dystrophy using the health utilities index and EQ-5D-5L.Journal of patient-reported outcomes · 2023Article
- Are We Capturing the Socioeconomic Burden of Rare Genetic Disease? A Scoping Review of Economic Evaluations and Cost-of-Illness Studies.PharmacoEconomics · 2023Article
- Reliability and Validity of the Korean Version of the Duchenne Muscular Dystrophy Functional Ability Self-Assessment Tool.Annals of rehabilitation medicine · 2023Article
- Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States.Journal of market access & health policy · 2023Article
- Direct costs of adhering to selected Duchenne muscular dystrophy Care Considerations: Estimates from a midwestern state.Muscle & nerve · 2022Article
- Labor market participation and productivity costs for female caregivers of minor male children with Duchenne and Becker muscular dystrophies.Muscle & nerve · 2021Article
- Diseases Costs and Impact of the Caring Role on Informal Carers of Children with Neuromuscular Disease.International journal of environmental research and public health · 2021Article
- Cost Effectiveness of Nusinersen in the Treatment of Patients with Infantile-Onset and Later-Onset Spinal Muscular Atrophy in Sweden.PharmacoEconomics · 2019Article
- Incomplete description of the current body of evidence of the health economics of Duchenne muscular dystrophy.Orphanet journal of rare diseases · 2019Review
- Producing a preference-based quality of life measure for people with Duchenne muscular dystrophy: a mixed-methods study protocol.BMJ open · 2019Article
- Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients.Journal of market access & health policy · 2019Article
- Another Rush to Judgment: The Imaginary Worlds of ICER and Recommendations in Duchenne Muscular Dystrophy.Innovations in pharmacy · 2019Article
Corrections and comments
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Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundSeveral treatments are on the horizon for Duchenne muscular dystrophy (DMD), a terminal orphan disease. In many jurisdictions, decisions regarding pricing and reimbursement of these health technologies comprise evidence of value for money.
objectiveThe objective of this study was to develop a cost-effectiveness model based on the Duchenne muscular dystrophy Functional Ability Self-Assessment Tool (DMDSAT), a new rating scale created specifically to measure disease progression in clinical practice and trials and model DMD in economic evaluations, and compare it with two alternative model structures.
methodsWe constructed three Markov cohort state-transition models to evaluate the cost-effectiveness of a hypothetical intervention for DMD versus standard of care in a UK setting. Model I was based on the DMDSAT, model II on stages of disease as defined in the DMD clinical care guidelines and model III on patients' ventilation status. The conceptual model structures were formulated in collaboration with three DMD experts.
resultsAll three models were judged to have good validity with regards to the appropriateness of the choice of modelling technique, conceptual representation of the disease, model input data and model outcomes. Across frameworks, lifetime direct medical costs with standard of care ranged between £217,510 and £284,640, total costs between £624,240 and £713,840, and total number of quality-adjusted life-years between 5.96 and 7.17.
conclusionsWe present a first version of a model for the economic evaluation of treatments for DMD based on the DMDSAT, as well as two alternative frameworks encompassing conventional staging of disease progression. Our findings should be helpful to inform health technology assessments and health economic programmes of future treatments for DMD.
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