Institution-based prospective inception cohort studies in neonatal rare disease research

SummaryIn rare diseases, wherein the conduct of randomized controlled clinical trials (RCTs) is challenging, cohort studies can offer important, and, in certain instances, high quality (e.g. the prospective inception cohort study) evidence on relationships among risk factors, treatments, and outcomes. The objective of this perspective article is to provide an overview of salient issues in the design and application of institution-based prospective inception cohort studies in neonatal rare disease research, with emphasis on quality assurance measures. Rigorous implementation of the prospective inception cohort study is challenging, and application to neonates renders it even more difficult. However, when performed collaboratively among institutions employing uniform methods and quality assurance mechanisms, institution-based prospective inception cohort studies can provide optimal observational evidence to inform the design and execution of RCTs in this special pediatric population.