ArticleOrphanet journal of rare diseases2019
Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best-worst scaling experiment in caregivers and adult patients.
Article in Orphanet journal of rare diseases, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed, 36 citations in OpenAlex.
- The Impact of Clinical Trial Participation on Quality of Life in Duchenne Muscular Dystrophy.Pharmaceutical medicine · 2026Article
- Gene Therapy: Knowledge, Attitudes, and Preferences Among Individuals with Alpha-1 Antitrypsin Deficiency.Chronic obstructive pulmonary diseases (Miami, Fla.) · 2026Article
- The Paradox of Life Extension with Traditional Cost-Effectiveness Analysis: A Case Study with Duchenne Muscular Dystrophy.Journal of health economics and outcomes research · 2026Article
- Preferences for gene therapy in Duchenne muscular dystrophy: insights from patient and caregiver interviews and attribute development.Frontiers in pharmacology · 2025Article
- Preferences for genetic interventions for SCA and Huntington's disease: results of a discrete choice experiment among patients.Orphanet journal of rare diseases · 2024Article
- Perspectives of the Friedreich ataxia community on gene therapy clinical trials.Molecular therapy. Methods & clinical development · 2024Article
- Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of Disease.Journal of neuromuscular diseases · 2024Article
- Genetic Interventions for Spinocerebellar Ataxia and Huntington's Disease: A Qualitative Study of the Patient Perspective.Journal of Huntington's disease · 2024Article
- Eliciting Exploratory Patient Preference Data: A Case Study in a Rare Disease.Pharmaceutical medicine · 2024Article
- Patient Preferences Regarding Surgical Treatment Methods for Symptomatic Uterine Fibroids.Therapeutic innovation & regulatory science · 2023Review
- Best-Worst Scaling Survey of Inpatients' Preferences in Medical Decision-Making Participation in China.Healthcare (Basel, Switzerland) · 2023Article
- Impact of Potential Symptoms and Risks Associated with Acid Sphingomyelinase Deficiency on Patients and Caregivers: A Best-Worst Scaling Study.Patient preference and adherence · 2023Article
- A Comparison of Caregiver and Patient Preferences for Treating Duchenne Muscular Dystrophy.The patient · 2022Article
- The Role of Patient Involvement When Developing Therapies.Nucleic acid therapeutics · 2022Article
- Ethical challenges for a new generation of early-phase pediatric gene therapy trials.Genetics in medicine : official journal of the American College of Medical Genetics · 2021Review
- Patients' and caregivers' maximum acceptable risk of death for non-curative gene therapy to treat Duchenne muscular dystrophy.Molecular genetics & genomic medicine · 2021Article
- The Organic Food Choice Pattern: Are Organic Consumers Becoming More Alike?Foods (Basel, Switzerland) · 2021Article
Corrections and comments
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Authors and funding
14 authors at 7 institutions in 1 country.
Funding
Abstract
purposeSeveral gene therapy trials for Duchenne muscular dystrophy initiated in 2018. Trial decision making is complicated by non-curative, time-limited benefits; the progressive, fatal course; and high unmet needs. Here, caregivers and patients prioritize factors influencing decision making regarding participation in early-phase gene therapy trials.
methodsWe conducted a best-worst scaling experiment among U.S. caregivers and adults with Duchenne (N = 274). Participants completed 11 choice sets, choosing features they cared about most and least when deciding whether to participate in a hypothetical gene therapy trial. We analyzed the data using sequential conditional logistic regression.
resultsParticipants prioritized improved muscle function in trial decision making. Concerns about participation limiting later use of gene transfer and editing were also important, as were improved lung and heart function. Low risk of death fell near the middle. Participants cared least about muscle biopsies and potential for randomization to placebo. Adults with Duchenne and caregivers of non-ambulatory children significantly prioritized improved lung function compared to caregivers of ambulatory children.
conclusionOur data demonstrate prioritization of anticipated benefits and opportunity costs relative to potential harms and procedures in gene therapy trial decision making. Such data inform protocol development, education and advocacy efforts, and informed consent.
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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.