ArticleThe patient2015
Caregiver preferences for emerging duchenne muscular dystrophy treatments: a comparison of best-worst scaling and conjoint analysis.
Article in The patient, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers, 5 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
27 citing papers in PubMed, 5 syntheses or guidelines pooled it.
- Pooled it
- Practical Considerations for Using Online Methods to Engage Patients in Guideline Development.The patient · 2018Pooled it
- Application of discrete choice experiments to enhance stakeholder engagement as a strategy for advancing implementation: a systematic review.Implementation science : IS · 2017Pooled it
- Using Best-Worst Scaling to Investigate Preferences in Health Care.PharmacoEconomics · 2016Pooled it
- Life with a Primary Immune Deficiency: a Systematic Synthesis of the Literature and Proposed Research Agenda.Journal of clinical immunology · 2016Pooled it
- 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
- The Impact of the Risk Functional Form Assumptions on Maximum Acceptable Risk Measures.The patient · 2021Article
- Characterizing Enrollment in Observational Studies of Duchenne Muscular Dystrophy by Race and Ethnicity.Journal of neuromuscular diseases · 2020Article
- The Patient Perspective: Putting the Patient at the Center of the Translational Innovation Process.Clinical pharmacology and therapeutics · 2020Article
- 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.Orphanet journal of rare diseases · 2019Article
- Article
- Nonclinical Exon Skipping Studies with 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn-/- Mice Inspired by Clinical Trial Results.Nucleic acid therapeutics · 2019Article
- Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents.PloS one · 2019Article
- Symposium Title: Preference Evidence for Regulatory Decisions.The patient · 2018Article
- How important is social support in determining patients' suitability for transplantation? Results from a National Survey of Transplant Clinicians.Journal of medical ethics · 2018Article
- The Ball is in Your Court: Agenda for Research to Advance the Science of Patient Preferences in the Regulatory Review of Medical Devices in the United States.The patient · 2017Article
- Moving from Patient Advocacy to Partnership: A Long and Bumpy Road.The patient · 2017Article
- Article
- Patient-centered Treatment Decisions for Urethral Stricture: Conjoint Analysis Improves Surgical Decision-making.Urology · 2017Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundThrough Patient-Focused Drug Development, the US Food and Drug Administration (FDA) documents the perspective of patients and caregivers and are currently conducting 20 public meetings on a limited number of disease areas. Parent Project Muscular Dystrophy (PPMD), an advocacy organization for Duchenne muscular dystrophy (DMD), has demonstrated a community-engaged program of preference research that would complement the FDA's approach.
objectiveOur objective was to compare two stated-preference methods, best-worst scaling (BWS) and conjoint analysis, within a study measuring caregivers' DMD-treatment preferences.
methodsWithin one survey, two preference-elicitation methods were applied to 18 potential treatments incorporating six attributes and three levels. For each treatment profile, caregivers identified the best and worst feature and intention to use the treatment. We conducted three analyses to compare the elicitation methods using parameter estimates, conditional attribute importance and policy simulations focused on the 18 treatment profiles. For each, concordance between the results was compared using Spearman's rho.
resultsBWS and conjoint analysis produced similar parameter estimates (p < 0.01); conditional attribute importance (p < 0.01); and policy simulations (p < 0.01). Greatest concordance was observed for the benefit and risk parameters, with differences observed for nausea and knowledge about the drug-where a lack of monotonicity was observed when using conjoint analysis.
conclusionsThe observed concordance between approaches demonstrates the reliability of the stated-preference methods. Given the simplicity of combining BWS and conjoint analysis on single profiles, a combination approach is easily adopted. Minor irregularities for the conjoint-analysis results could not be explained by additional analyses and needs to be the focus of future research.
Indexed as
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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.