Abstract
Scale development and evaluation is particularly challenging in rare diseases, due to small samples and heterogenous clinical profiles. Recent recommendations support a flexible approach which includes multiple sources, iterative mixed methods and repeated measurements to estimate the psychometric characteristics of an instrument when sample size is small. However, detailed methodological accounts of COA examination in rare disease are still rare. This study describes an iterative mixed method approach for the evaluation, modification and measurement maximization of the INAD-RS, a 40-item ClinRO developed to assess the disease progression in children with Infantile Neuroaxonal Dystrophy (INAD).
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