Guidelines for the Ethical Conduct of Studies to Evaluate Drugs in Pediatric Populations

Abstract

To be acceptable, protocols for clinical pharmacologic studies in children must meet high ethical and scientific standards. Any study that is unacceptable medically or promises no significant scientific benefit in its aims or construction is unethical by definition. Studies believed to be medically and scientifically acceptable must then be scrutinized carefully to determine that the risks are minimal when compared with the benefits and that the individual child is as well protected as is reasonable. Only then may a study be considered accepted as ethical and approved for implementation. Investigators who wish to carry out drug studies in children should be acquainted with the ethical requirements of such investigations in children. Informed consent should be obtained from an adult acting on behalf of the minor in all instances prior to the enrollment of a child into a study. The investigator's zeal should not be permitted to result in misrepresentation of the nature of the study. Assent by the child is essential for children more than approximately 7 years of age. The child should be old enough or intellectually mature enough to be capable of understanding appropriately presented data about the study and the procedures required if he/she enrolls in it. These children should be given the right to say no, even when an adult acting on their behalf has given consent. Minors 13 years of age or older may be enrolled in a study only after they have been appropriately informed and have given their consent. Frequent review of the progress of the activities of the institutional review committee and of the implementation of approved proposals is required for the protection and welfare of children enrolled in studies. Similar review is required when drug investigation studies are carried out in children in the absence of institutional supervision. Continuing review provides a mechanism to maintain the highest ethical standards. These guidelines are designed to provide a format allowing for maximal protection of child subjects of drug research. The guidelines also acknowledge both the "need to know" and the "unethical" nature of the situation in which pediatric therapeutics finds itself today. It is hoped that these guidelines will pave the way for an increase in the number of drug investigations carried out in children, and thereby improve health care for all children in the future.