Cornett Kayla M D, Estilow Tim, Bray Paula, Mandarakas Melissa R, Donlevy Gabrielle A, Baldwin Jennifer N, Eichinger Kate, Finkel Richard S, Burns Joshua, McKay Marnee J
School of Health Sciences, Faculty of Medicine & Health, The University of Sydney, Sydney, New South Wales, Australia.
Sydney Children's Hospitals Network, Sydney, New South Wales, Australia.
J Peripher Nerv Syst. 2025 Sep;30(3):e70062. doi: 10.1111/jns.70062.
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.
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.
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.
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.
在诸如夏科-马里-图斯病（CMT）等罕见神经系统疾病中，准确、可靠且灵敏的临床结局指标对于监测疾病进展和评估治疗效果至关重要。确保诸如CMTPedS、CMTInfS和CMT-FOM等指标易于获取可消除实施障碍。本项目的目的是：（1）设计一个基于网络的平台，以实现CMT结局指标的实时评分；（2）实施共同设计的培训和质量保证资源；（3）建立临床评估人员的全球认证标准。
通过咨询过程为基于网络的平台设计提供参考，其中包括对当前用户（n = 65）的过程评估。培训资源通过混合方法与关键利益相关者（n = 51）共同设计，这些利益相关者包括CMT医生和科学家、临床评估人员、制药代表以及患者。通过共同设计过程和首席培训师的专业知识制定了全球认证标准。
基于网络的平台提供CMT结局指标的实时评分、标准化培训资源以及全球认证标准，以支持对疾病严重程度、进展和治疗效果进行准确可靠的评估。
