Ethical Considerations in Pediatric Research

The conduct of research in pediatric subjects is crucial to optimizing clinical care in this diverse patient population. Although the demand for pediatric research remains high, the logistical implementation is complex. Many factors have led to a lack of pediatric research, including lack of agreed-on end points, informed consent issues, and the general perception that pediatric patients are vulnerable study subjects. This perceived vulnerability of children is based on several factors: (1) their decision-making capacity may be immature, (2) their lives are still subject to the authority of others, (3) their underlying dissent may be masked for fear of upsetting authority figures, and (4) their rights and interests may be undervalued by society. To ensure that pediatric patients are provided the best clinical care, it is important that thoughtful, efficient research be conducted that has the scientific merit to answer important clinical questions. The fundamental challenge to pediatric research is risk: what is the risk of conducting (or not conducting) the research, and who has the right to decide the level of risk exposure to the child? What “say” does a child have in agreeing to participate in research? What “say” does the legal guardian have in agreeing for a child to participate in research? What is the ultimate goal of the research? Finally, does the goal of the research justify the risks associated with the study? This chapter outlines the ethical factors affecting the successful conduct of pediatric research. Ethical considerations and their relationship within the context of informed consent and assent are discussed. The discussion of clinical research design focuses on ethical challenges inherent to researchers, especially those unique to pediatric patients. Finally, additional confounders to pediatric research, such as compensation and the role of the pharmaceutical industry, are discussed. ABBREVIATIONS IN THIS CHAPTER CFR Code of Federal Regulations IRB Institutional review board NI Noninferiority