Diversifying Clinical Evaluator Training Resources to Globalize CMT Trial Readiness

Topic:

Other

Poster Number: 344 T

Author(s):

Gabrielle Donlevy, PhD, The University of Sydney School of Health Sciences, Faculty of Medicine and Health, NSW Australia, Kayla Cornett, PhD, University of Sydney, Marnee McKay, PhD, The University of Sydney School of Health Sciences, Faculty of Medicine and Health, NSW Australia, Kate Eichinger, PhD, University of Rochester Medical Centre, Tim Estilow, OTR/L, Children’s Hospital of Philadelphia, Philadelphia PA, Melissa Mandarakas, PhD, Department of Medicine and Health, University of New South Wales, Sydney Australia, Joshua Burns, PhD, St Jude Children's Research Hospital

Introduction: International multicentre clinical trials in rare diseases represent complex global initiatives that face challenges arising from cultural and linguistic diversity, as well as variability in evaluator training and experience. The standardization in data collection can compromise scientific validity and hinder trial outcomes. The online learning platform www.ClinicalOutcomeMeasures.org was developed as a comprehensive resource to facilitate clinical evaluator training and provide quality assurance support for participating sites. The platform provides extensive training materials promoting accurate, reliable administration of commonly used clinical outcome assessments for Charcot-Marie-Tooth disease (CMTPedS, CMTInfS, and CMT-FOM). However, existing materials are available only in English reflecting limited cultural and linguistic diversity.
The aim of this study was to expand and diversify e-training and quality assurance resources, co-designing new materials with input from children, adolescents, and adults living with CMT.
Methods: Participants and their families engaged during interviews or focus groups to ensure an authentic representation of disease severity and lived experience of CMT was represented and captured. A structured questionnaire and follow-up feedback process supported iterative refinement of video content prior to finalization.
In parallel, professional certified translations of training materials and quality assurance resources into Spanish and French have been developed using AI and human verification. Translated materials have undergone verification for clinical accuracy, cognitive debriefing, and assessment of linguistic equivalence by local language specialists and clinicians.
Results: This project will deliver (a) full translation of training manuals, (b) audio translations for video training materials, and (c) consumer co-design led culturally diverse training videos representing disease severity and diversity. Translation of all equipment and training manuals is underway, and participant recruitment for co-design interviews and video capture is complete.
Conclusion: Diversifying training and quality assurance resources will enhance the reach and relativity of CMT outcome measures and support global standardization and certification of CMT evaluators across the lifespan.