Designing and Implementing a Web‐Based Platform for Accurate and Reliable Clinical Outcome Measures and Global Certification for Evaluating Charcot–Marie–Tooth disease

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BACKGROUND AND AIMS: Accurate, reliable and sensitive clinical outcome measures in rare neurologic conditions, such as Charcot-Marie-Tooth disease (CMT), are essential for monitoring disease progression and evaluating treatment efficacy. Ensuring measures such as the CMTPedS, CMTInfS and CMT-FOM are easily accessible removes a barrier to implementation. The aims of this project were to: (1) design a web-based platform to enable real-time scoring of CMT outcome measures; (2) implement co-designed training and quality assurance resources; and (3) establish a Global Certification Standard for clinical evaluators. METHODS: A consultation process informed the design of the web-based platform and included a process evaluation of current users (n = 65). Training resources were co-designed with key stakeholders (n = 51) including CMT physicians and scientists, clinical evaluators, pharmaceutical representatives, and patients through a mixed-methods approach. A Global Certification Standard was developed through the co-design process and Master Trainer expertise. RESULTS: A web-based platform, www.ClinicalOutcomeMeasures.org was designed and implemented in June 2020 as a freely available trial readiness resource for clinicians and researchers. The platform now has > 1400 registered users from > 45 countries. Clinical evaluators identified a lack of accessible training resources as the top barrier to accurate and reliable administration of CMT outcome measures. Video demonstrations, online workshops, and labelled photographs were ranked as the top training methods. Five guiding principles for the Global Certification Standard for CMT outcome measures were established. INTERPRETATION: The web-based platform provides real-time scoring of CMT outcome measures, access to standardized training, and a Global Certification Standard to support accurate and reliable assessment of disease severity, progression, and treatment efficacy.

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