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FDA Releases Draft Guidance on Ethics for Pediatric Involvement in Clinical Trials

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The inclusion of children in medical research is important to the development of more effective treatments, however, the ethnics of doing so may raise eyebrows if not careful.

Prior to formal legislation, the use of children as subjects in medical research or drug clinical trials was rare or nonexistent and unregulated until after the 1980s. Up until this time, therapy for children with a disease that affected both adults and children was to simply use a lower dose of a preferred therapy. However, some diseases, such as genetic diseases and metabolic disorders are unique or fatal in childhood, meaning that these patients never live long enough to see adulthood and give informed consent to be studied. Additionally, simply lowering a dose for children based on body weight or surface area was not always equally effective and sometimes toxic due to the unique absorption, elimination, and metabolic system of a child.

Evaluation of children in clinical trials, in result, is important to the development of potentially more effective treatment regimens and can result in improved pediatric patient outcomes. However, what are the ethnical responsibilities of including pediatric patients in clinical trials when they are unable to give informed consent? Children are among the most vulnerable members of our society and, in the context of medical research, not considered capable or competent of making an informed consent, which is a requirement of today’s clinical trial process in order to participate. In these populations, the informed consent of participating in a trial is obtained from the parents, and the children provide an “assent”. Due to these assertions, the FDA has put additional safeguards in place to protect the interest of a child participating in medical research.

Recently, the FDA announced an updated guidance draft that, when finalized, will provide “the agency's perspective on the ethical considerations for including and protecting children in clinical trials.” In general, FDA guidance documents do not establish legally enforceable responsibilities but instead describe the agency’s current thinking on a topic and should be viewed as recommendations. The draft, according to the FDA, is intended to assist industry, sponsors, and institutional review boards when considering the enrollment of children in clinical investigations of drugs, biological products, and medical devices.

Recommendations in the drafted guidance describe the ethical framework for protecting children, including risk and benefit considerations, and outlines and explains fundamental concepts to be considered when reviewing or conducting these pediatric trials. Included in the risk and benefit considerations for using children in clinical trials are the scientific necessity of conducting a clinical investigation in children. Additionally, researchers should evaluate whether risk categories for interventions or procedures do or do not offer a prospect of direct benefit to the child. Those wishing to conduct a study including children should conduct a component analysis of the risks of intervention or procedures, and in all children-included studies, obtain parental or guardian permission and child assent. Researchers should also understand the potential for review, under a regulatory provision, of research that is not otherwise approvable by an institutional review board.

Since the guidance is a draft, the public can provide comments for 90 days. This is in order for the agency to be able to consider them when finalizing the guidance.

"Children need access to safe and effective medical products and healthcare professionals need data to make evidence-based decisions when treating children," said Dionna Green, MD, director of the FDA's Office of Pediatric Therapeutics. “However, children are a vulnerable population who can't provide consent for themselves and are afforded additional safeguards when participating in a clinical investigation. The best way to provide children with safe and effective treatment options is by including them in clinical research and providing these additional safeguards to protect them during clinical trials."

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