Comparative effectiveness and child health

Abstract

Comparative effectiveness research is expected to play an important role in future clinical and policy decision making in the US; however, the application of comparative effectiveness methodologies to child health requires special attention to aspects of health and healthcare that are specific to children. These special considerations include the role of parent/caregiver as joint decision maker and co-participant in many types of interventions, how the effectiveness of an intervention varies by age and developmental stage, and the difficulties in translating short-term data from childhood into projected effectiveness over the lifespan. Each aspect of comparative effectiveness, such as conducting new studies, synthesizing existing evidence, emphasizing real-world settings, considering multiple decision makers, and measuring patient-relevant outcomes, will require expanded definitions when considered in the context of child health. This paper discusses how comparative effectiveness methods and concepts will differ when applied to child health and suggests a potential role for decision analysis as a method to synthesize data and project long-term outcomes. The initiation of comparative effectiveness studies for children represents an exciting opportunity to provide evidence that can guide clinical and policy decisions for child health.