“…a total of 426 drug labels have been revised with new paediatric information and an estimated 50 percent of drugs used in children have now been studied in children as compared to just 20 percent before the enactment of [the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act].” – Bohannon and Sietsema. Pharmaceutical Physician. 2012: 6
In the past 10 years, there have been more studies involving pediatrics than there were in the 50 years prior, according to the 2012 special feature article. This increase is due to US legislation and the realization that clinical trials involving pediatric patients are important. There are ethical and practical challenges to the inclusion of pediatric patients in clinical trials. However, excluding pediatric patients from clinical trials can be harmful.
The FDA and the WHO have deemed pediatric patients a vulnerable population. This vulnerability leads to ethical considerations when including pediatric patients in clinical trials. One issue is that of informed consent or assent. Infants and young children are developmentally unable to understand what it means to participate in a clinical trial or the implications of doing so. Additionally, even children who do (or don’t) wish to participate can be over-ruled by their parents. This also affects patient privacy. Pediatric patients may not legally make their own health decisions. This means their medical information must be shared with their legal guardians.
Practically, there are challenges to including pediatric patients in clinical trials, too. Children have special compliance challenges, including dysphagia and sensitivity to palatability, dependence on caregivers and caregiver schedules and a desire to avoid injections or other uncomfortable drug delivery methods due to lack of understanding the benefits.
Despite these ethical and practical challenges, excluding pediatric patients from clinical trials can do great harm. Pediatric and adult patients have different pharmacokinetics, pharmacodyamics and susceptibility to adverse events. Drugs that have only been tested in adults could cause harmful side effects in children while providing no medical benefit.
In spite of the ethical and practical challenges to including pediatric patients in clinical trials, such inclusion can do great good and prevent harm. Orbis Biosciences develops many drugs that can be adapted for use in children. Pediatric health is a passion of ours, and we feel strongly that an ethical foundation is vital to all areas of medicine.