Pediatric Pharmacotherapy Part 1: The History of Pediatric Drug Therapy: Learning from Errors, Not Trials

Marcel J. Casavant; Jill R. K. Griffith


AccessMedicine from McGraw-Hill 

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Testing Drugs in Children

Before the 1970s, children and pregnant women were routinely excluded from drug studies. Noble reasons concerning the protection of children, a vulnerable population, and their inability to provide legally valid, truly informed consent, are most often cited, although one wonders whether the practical difficulties of enrolling and studying children, and of analyzing the results in a time when little was known about pediatric development, may have contributed. Regardless, by the mid-1970s, the American Academy of Pediatrics made a powerful argument that scientifically sound and ethically acceptable studies of drugs could be conducted in children and, further, that the failure to conduct drug studies in children was itself unethical as it forced physicians to do uncontrolled experiments almost every time they prescribed a drug to a child, and it deprived children of the full benefits of pharmacotherapy enjoyed by other populations.[7]

Guidelines proposed by the American Academy of Pediatrics, published first in 1977 and revised in 1995, called for studies in children to be performed by principal investigators who know about the drug they’re studying and about the study of drugs in children, and for the supervision of these investigators by an institutional review board with expertise about the specific concerns and requirements of the child as a research subject.[8] The Belmont report (1979) and subsequent U.S. regulations further defined requirements for pediatric research. Children can be exposed to risks from research, and to greater risks if the research offers them potential benefit. Varying degrees of permission are required based on the balance of risks and benefits to which the child is exposed: one parent may give permission for a child to participate in a minimal risk study or a study with more than minimal risk but also the prospect of direct benefit, but both parents must consent for a child to participate in a study with more than minimal risk and no direct benefit to the child.


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