Children are not little adults, and protocols for pediatric pain studies must take into account the unique needs of children to meet regulatory and ethical standards and protect this vulnerable population from untreated pain.
Sponsors of pediatric analgesia clinical trials are tasked with designing studies that are both realistic to execute and sufficiently rigorous to support efficacy, safety, and dosing in children.
Performing research studies to evaluate analgesics in children is critical for determining the safety and efficacy of these medications in pediatric populations. According to the National Institutes of Health, 70 percent of the medicines given to children have only been tested in adults, so there remains a significant need for pediatric-specific analgesic prescribing information based on clinical evidence to support efficacy, safety, and dosing in children. With the permanent extension of the Pediatric Research Equity Act of 2003 (PREA) in July 2012 and similar legislation in the European Union, an increasing number of sponsors are confronted with the task of conducting pediatric studies as an integral part of the analgesic development pathway.
Pediatric clinical trials typically require a different approach than adult clinical trials, as the differences between children and adults extend beyond anatomical and physiological differences to communication barriers and emotional considerations. There are also significant developmental differences among children of different age groups. In pediatric analgesia studies, the intrinsic scientific, ethical, and practical challenges of conducting clinical trials in children are further complicated by the need to protect these vulnerable patients from exposure to untreated pain.
Writing protocols for pediatric pain studies requires sponsors to do much more than simply cut and paste the protocol from adult trials. In order to meet regulatory and ethical standards, all aspects of the protocol – from study design and procedures to endpoints and informed consent – must be customized for children. A well-written protocol that takes into account the unique needs of children can facilitate regulatory approval, study enrollment, retention, and data quality. This paper provides insight on optimizing protocol writing for pediatric analgesia clinical trials of acute pain, with a focus on innovative study designs and outcome measures specific for children.