Introduction: Considerable time and resources are dedicated to accurate and reliable data collection in clinical trials and cohort studies. The aim of this study was to compare different training pathways to accurately and reliably collect clinical outcome assessments (CMTPedS and CMT-FOM) in children and adults with Charcot-Marie-Tooth disease (CMT).
Methods: Twenty clinical evaluators from various healthcare professions and years of CMT experience participated in a global certification study to compare three training pathways with decreasing face-to-face training components i.e., gold standard (in-person, hands-on training with a Master Trainer) vs. high-fidelity (online-only training with a Master Trainer) vs. low-fidelity (online-only, self-directed training). All clinical evaluators completed e-learning at www.ClinicalOutcomeMeasures.org and were allocated to one of three training pathways stratified for healthcare profession and years of experience assessing patients with CMT. All clinical evaluators performed muscle strength accuracy testing on 5 healthy controls and then attended a 2-day training summit to perform reliability testing on three individuals with CMT or healthy controls against Master Trainers.
Results: Following online training, clinical evaluators across the three training pathways were equivalent for CMT experience, confidence and knowledge. Muscle strength measures in healthy controls showed the highest accuracy for clinical evaluators in the gold standard followed by high-fidelity training pathways (measures <1SD and <1.5 SDs respectively of normative reference data from the 1000 Norms Project) with unacceptably low accuracy in the low-fidelity pathway. For CMTPedS and CMT-FOM item z-scores and total scores, clinical evaluators trained by the gold standard and high-fidelity training pathways demonstrated higher reliability against Master Trainers compared to the low-fidelity pathway. Clinical evaluators trained by the gold standard pathway measured identical CMTPedS and CMT-FOM total scores as Master Trainers.
Conclusion: Accurate and reliable collection of CMT clinical outcome assessments requires training of clinical evaluators using the gold standard or high-fidelity training pathways.