Question

What are the ethical concerns of pediatric clinical trials?

Answer 1

Research that involves children has always been ethically problematic. The fundamental reason is straightforward. Research, by its very nature, uses subjects as a means to the end of creating generalizable knowledge. In adults, the solution to this fundamental ethical problem is to get the voluntary, informed consent of the research subject. Children cannot consent on their own behalf. Instead, researchers, parents, and regulators must determine whether the risk-benefit ratio is acceptable in order to permit the research to go forward.

The regulations governing research conduct in the United States have always included special requirements and considerations for pediatric research subjects and other vulnerable populations. These special requirements call for a higher level of scrutiny and more stringent thresholds of protection than for less vulnerable populations.

While the reasons for extra scrutiny of pediatric research are straightforward, the arguments for the necessity of doing research in children are also compelling. Children, and particularly infants, respond differently to drugs and other medical treatments than do adults. There are many stories of drugs that, while safe in adults, have serious and even fatal side effects in children. Studies in children often require longer follow-up than do studies in adults in order to determine whether innovative treatments have any long-term developmental effects.

The current regulatory guidelines for pediatric research define four levels of riskiness in research studies, each of which is subject to a different level of regulation and oversight. The lowest level of risk is “minimal risk.” Minimal risk is defined as ‘the probability and magnitude of physical or psychological harm that is normally encountered in the daily lives, or in the routine medical or psychological examination, or health children’ (National Commission – Research Involving Children). Studies that involve only minimal risk can be carried out, even if they do not offer any prospect of direct benefit to the research subjects. For such studies, researchers only need the permission of one parent and the assent of the child – if the child is old enough and cognitively capable of giving assent.