MULTIDISCIPLINARY ASSESSMENT OF HEALTHCARE PROFESSIONALS' ADOPTION WILLINGNESS, BARRIERS, AND FACILITATORS FOR DUCHENNE MUSCULAR DYSTROPHY GENE THERAPY IN SPAIN
Author(s)
Rocio Garcia Uzquiano, MD1, Jose Luis Poveda Andres, PharmD2, Julita Medina Castillo, MD3, Beatriz Palop Garcia, PharmD4, M ANGELES TERRANCLE JUAN, PharmD5, Elisa Guillen, PharmD5, maria brañas pampillon, PharmD5, Jorge Maurino, MD5.
1Hospital General Universitario Gregorio Marañón, Madrid, Spain, 2Hospital Universitari I Politècnic la Fe, Valencia, Spain, 3Hospital Sant Joan de Deu, Barcelona, Spain, 4Hospital Vall d’Hebrón, Barcelona, Spain, 5Roche Farma SA, Madrid, Spain.
1Hospital General Universitario Gregorio Marañón, Madrid, Spain, 2Hospital Universitari I Politècnic la Fe, Valencia, Spain, 3Hospital Sant Joan de Deu, Barcelona, Spain, 4Hospital Vall d’Hebrón, Barcelona, Spain, 5Roche Farma SA, Madrid, Spain.
OBJECTIVES: OBJECTIVES: To evaluate the willingness of a multidisciplinary cohort of healthcare professionals (HCPs) to adopt gene therapy for Duchenne Muscular Dystrophy (DMD) and to identify clinical, behavioral, and predictive factors influencing clinical integration.
METHODS: METHODS: This non-interventional, cross-sectional web-based survey included Spanish pediatric neurologists, rehabilitation physicians, and hospital pharmacists recruited through their respective national scientific societies. Professional attitudes toward innovation were quantified using the Evidence-Based Practice Attitude Scale (EBPAS), while professional engagement was assessed via the Utrecht Work Engagement Scale (UWES). High willingness was defined by a composite of risk-benefit calibration and resilience to administrative or economic burdens. Factors independently associated with adoption were identified using a multivariate logistic regression model.
RESULTS: RESULTS: The cohort comprised 53 HCPs (37 pediatric neurologists, 11 rehabilitation physicians, and 5 hospital pharmacists). High enthusiasm was observed, with 89.2% of pediatric neurologists viewing gene therapy as a transformative shift in the standard of care. Key clinical barriers included adverse event concerns and long-term efficacy uncertainty, whereas logistical burdens and patient resistance were less impactful. Based on EBPAS scores, 73.0% of pediatric neurologists were open to innovation. High professional vigor (UWES) facilitated adoption; 61.9% of participants with high vigor reported ease in interpreting complex clinical trial evidence versus 18.8% with lower scores. Behavioral intention remained robust despite obstacles; 94.6% intended to adopt therapy regardless of economic impact. In the multivariate analysis, confidence in the 6-Minute Walk Test (6MWT) as an objective and sensitive tool was the only independent predictor of high adoption willingness (Odds Ratio: 15.23; p=0.018).
CONCLUSIONS: CONCLUSIONS: Spanish HCPs demonstrate a strong predisposition toward adopting DMD gene therapy. Successful integration requires standardized professional guidelines and robust evidence to mitigate safety concerns. Furthermore, establishing consensus on sensitive monitoring tools is critical, as confidence in assessments like the 6MWT significantly drives adoption willingness.
METHODS: METHODS: This non-interventional, cross-sectional web-based survey included Spanish pediatric neurologists, rehabilitation physicians, and hospital pharmacists recruited through their respective national scientific societies. Professional attitudes toward innovation were quantified using the Evidence-Based Practice Attitude Scale (EBPAS), while professional engagement was assessed via the Utrecht Work Engagement Scale (UWES). High willingness was defined by a composite of risk-benefit calibration and resilience to administrative or economic burdens. Factors independently associated with adoption were identified using a multivariate logistic regression model.
RESULTS: RESULTS: The cohort comprised 53 HCPs (37 pediatric neurologists, 11 rehabilitation physicians, and 5 hospital pharmacists). High enthusiasm was observed, with 89.2% of pediatric neurologists viewing gene therapy as a transformative shift in the standard of care. Key clinical barriers included adverse event concerns and long-term efficacy uncertainty, whereas logistical burdens and patient resistance were less impactful. Based on EBPAS scores, 73.0% of pediatric neurologists were open to innovation. High professional vigor (UWES) facilitated adoption; 61.9% of participants with high vigor reported ease in interpreting complex clinical trial evidence versus 18.8% with lower scores. Behavioral intention remained robust despite obstacles; 94.6% intended to adopt therapy regardless of economic impact. In the multivariate analysis, confidence in the 6-Minute Walk Test (6MWT) as an objective and sensitive tool was the only independent predictor of high adoption willingness (Odds Ratio: 15.23; p=0.018).
CONCLUSIONS: CONCLUSIONS: Spanish HCPs demonstrate a strong predisposition toward adopting DMD gene therapy. Successful integration requires standardized professional guidelines and robust evidence to mitigate safety concerns. Furthermore, establishing consensus on sensitive monitoring tools is critical, as confidence in assessments like the 6MWT significantly drives adoption willingness.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HSD36
Topic
Health Service Delivery & Process of Care
Disease
Neurological Disorders