Willingness to adopt gene therapy for Duchenne muscular dystrophy: barriers, facilitators, and multidisciplinary readiness
6 affiliations
- 1Department of Pediatric Neurology, Hospital General Universitario Gregorio Marañón, Madrid, Spain
- 2Medical Department, Roche Farma, Madrid, Spain
- 3Department of Rehabilitation, Hospital Sant Joan de Déu, Barcelona, Spain
- 4Department of Pharmacy, Hospital Universitari Vall D'Hebron, Barcelona, Spain
- 5Health Economics and Outcome Research Unit, Roche Farma, Madrid, Spain
- 6Management Department, Hospital Universitari i Politècnic La Fe, Valencia, Spain
Abstract
Introduction: Management of Duchenne muscular dystrophy (DMD) is shifting toward genotype-specific, disease-modifying interventions, including adeno-associated virus-mediated micro-dystrophin gene transfer. Grounded in established technology adoption frameworks, this study evaluated healthcare professionals' (HCPs) willingness to adopt advanced gene therapies, identified critical systemic barriers and facilitators, and mapped multidisciplinary familiarity and quality perceptions regarding functional outcome measures.
Methods: We conducted a cross-sectional, web-based study evaluating 53 HCPs actively involved in DMD care across Spain: 37 pediatric neurologists, 11 rehabilitation physicians, and 5 hospital pharmacists. Using ad-hoc questionnaires and validated psychometric instruments, we evaluated technical knowledge, functional scale perceptions (including sensitivity to change and specificity), behavioral intent, and implementation challenges. Multivariate logistic regression identified independent predictors of high adoption willingness.
Results: Receptivity was high, with 67.6% of pediatric neurologists demonstrating strong willingness to adopt gene therapy. Clinicians ranked acute post-infusion safety concerns (mean rank: 2.30; lower values indicate greater relevance) and uncertainty regarding long-term efficacy (mean rank: 2.70) as primary clinical barriers, followed by institutional cost constraints (mean rank: 3.38). Conversely, the availability of robust clinical trial evidence was the primary operational facilitator (86.5%). In the multivariate model, a clinician′s perception of the Six-Minute Walk Test as an objective and sensitive instrument emerged as the sole independent predictor of high adoption intent (OR = 15.23, 95%CI: 1.58-146.67, p = 0.018).
Conclusions: HCPs demonstrate high theoretical receptivity to DMD gene therapy. However, clinical translation remains constrained by operational bottlenecks, acute safety risks, and technical gaps in functional outcome mastery. Because clinician readiness depends on diagnostic confidence in outcome measures, health authorities must prioritize standardized safety surveillance protocols, interprofessional training on scale psychometrics, and transparent reimbursement frameworks prior to broader deployment.
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BibTeX
@article{garciauzquiano2026willingness,
title = {{Willingness to adopt gene therapy for Duchenne muscular dystrophy: barriers, facilitators, and multidisciplinary readiness}},
author = {Rocío García-Uzquiano and María Branas-Pampillón and Julita Medina-Cantillo and Beatriz García-Palop and Elena Ruiz-Beato and Jorge Maurino and José Luis Poveda-Andrés},
journal = {Frontiers in Medicine},
year = {2026},
month = oct,
doi = {10.3389/fmed.2026.1985172},
url = {https://doi.org/10.3389/fmed.2026.1985172},
note = {CC BY 4.0},
}
RIS
TY - JOUR
TI - Willingness to adopt gene therapy for Duchenne muscular dystrophy: barriers, facilitators, and multidisciplinary readiness
AU - Rocío García-Uzquiano
AU - María Branas-Pampillón
AU - Julita Medina-Cantillo
AU - Beatriz García-Palop
AU - Elena Ruiz-Beato
AU - Jorge Maurino
AU - José Luis Poveda-Andrés
PY - 2026
DA - 2026/10/21
JO - Frontiers in Medicine
DO - 10.3389/fmed.2026.1985172
UR - https://doi.org/10.3389/fmed.2026.1985172
AB - Introduction: Management of Duchenne muscular dystrophy (DMD) is shifting toward genotype-specific, disease-modifying interventions, including adeno-associated virus-mediated micro-dystrophin gene transfer. Grounded in established technology adoption frameworks, this study evaluated healthcare professionals' (HCPs) willingness to adopt advanced gene therapies, identified critical systemic barriers and facilitators, and mapped multidisciplinary familiarity and quality perceptions regarding functional outcome measures. Methods: We conducted a cross-sectional, web-based study evaluating 53 HCPs actively involved in DMD care across Spain: 37 pediatric neurologists, 11 rehabilitation physicians, and 5 hospital pharmacists. Using ad-hoc questionnaires and validated psychometric instruments, we evaluated technical knowledge, functional scale perceptions (including sensitivity to change and specificity), behavioral intent, and implementation challenges. Multivariate logistic regression identified independent predictors of high adoption willingness. Results: Receptivity was high, with 67.6% of pediatric neurologists demonstrating strong willingness to adopt gene therapy. Clinicians ranked acute post-infusion safety concerns (mean rank: 2.30; lower values indicate greater relevance) and uncertainty regarding long-term efficacy (mean rank: 2.70) as primary clinical barriers, followed by institutional cost constraints (mean rank: 3.38). Conversely, the availability of robust clinical trial evidence was the primary operational facilitator (86.5%). In the multivariate model, a clinician′s perception of the Six-Minute Walk Test as an objective and sensitive instrument emerged as the sole independent predictor of high adoption intent (OR = 15.23, 95%CI: 1.58-146.67, p = 0.018). Conclusions: HCPs demonstrate high theoretical receptivity to DMD gene therapy. However, clinical translation remains constrained by operational bottlenecks, acute safety risks, and technical gaps in functional outcome mastery. Because clinician readiness depends on diagnostic confidence in outcome measures, health authorities must prioritize standardized safety surveillance protocols, interprofessional training on scale psychometrics, and transparent reimbursement frameworks prior to broader deployment.
SN - 2296-858X
ER -