FDA Highlights Challenges of Conducting Clinical Trials in Children
(Thursday, September 29, 2022)
Including children in clinical trials poses unique challenges and FDA’s latest guidance document on the topic acknowledges and highlights all the reasons why so few trials are done in pediatric subjects. The guidance confirms the conventional wisdom that trials in children must be adequately justified, properly designed to minimize risk to children, and avoided if alternate means are available for evaluating the safety and efficacy of a new drug in children. Although pediatric studies have been required for more than 20 years for all new drugs, first under the Pediatric Rule, 2002, and then under the Pediatric Research Equity Act (PREA) 2012, most sponsors prefer to avoid pediatric clinical trials to reduce liability. The threshold for risk tolerance in children is much lower than that for adults. Citing this risk to children and the policy that drugs for adults should not be delayed due to lack of studies in children, most new drugs are approved either with waivers from pediatric studies or deferral of such studies to post-market stage. There are several indications for which the incidence rates in children are so low that FDA automatically grants waiver from pediatric studies. And there are some FDA acceptable ways to calculate safe and effective doses for children. Under this regulatory environment, conducting clinical studies in children is much harder than those in adults. The biggest challenges are doing placebo-controlled studies, discussing the potential benefit of the experimental treatment to the participating children and their parents/guardians, or conducting studies in children where there may be higher risk of adverse events either because of the investigational product or the study procedures such as blood draws or scans. The Guidance Document discusses all the above and other issues related to pediatric studies with suggested solutions. In short, while the guidance provides a robust discussion of the various issues, it also highlights the various challenges which would always hold back clinical studies in children. The guidance will be important for discussing justification and design of pediatric clinical trials. The draft guidance is open for public comments. It should help create comprehensive strategies for pediatric clinical trials.
Dr. Mukesh Kumar
Founder & CEO, FDAMap
Linkedin: Mukesh Kumar, PhD, RAC