A Pilot Study to Evaluate Cross-Sectional and Longitudinal Supplemental Data Collection on Medical and Nonmedical Costs of Muscular Dystrophy

Abstract

This case study describes the design and implementation of a small-scale, or pilot, study for collecting cross-sectional and longitudinal supplemental data on medical and nonmedical costs associated with selected muscular dystrophies. We recruited individuals with a confirmed diagnosis of Duchenne and Becker or myotonic muscular dystrophies identified by the population-based Muscular Dystrophy Surveillance, Tracking, and Research Network. We later analyzed our pilot study approach for the purpose of identifying successful components for future large-scale data collection. Readers are expected to learn about the challenges of designing and implementing a pilot study. Our evaluation included recruitment success, cross-sectional versus longitudinal data collection, and response patterns.

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