Developing comparative effectiveness studies for a rare understudied pediatric disease: lessons learned from the CARRA juvenile localized scleroderma consensus treatment plan pilot study

摘要

BackgroundWe designed and initiated a pilot comparative effectiveness study for juvenile localized scleroderma (jLS), for which there is limited evidence on best therapy. We evaluated the process we used, in relation to the specific protocol and to the general task of identifying strategies for implementing studies in rare pediatric diseases.