Clinical Trials in Children

Abstract

Randomized controlled clinical trials are felt by the medical community to provide the best evidence. Participation in trials involves the possibility of obtaining benefits but also of suffering some risks. Those risks are often considered unacceptable for children but if clinical trials are not conducted in children, clinicians are forced to extrapolate study data from adults. In 1968 H. Shirkey termed children "therapeutic orphans" because of the lack of adequately tested and labeled drugs available in appropriate formulations. Research involving children entails specific difficulties as the need to study children of different ages, the small number of children affected by certain diseases or ethical issues. This paper considers aspects of pediatric clinical pharmacology and children's responses to drugs. It also reviews some of the current situations in pediatric clinical trials, covering aspects such as: the benefits and risks of trial participation; the specificity of pediatric trial design; the ethical issues such as consent; the use of placebo or the participation of healthy children; and the current legal situation in Europe and in the USA.

Drug utilization and off-label drug use among Spanish emergency room paediatric patients

“…Clinicians are therefore forced to extrapolate study data from adults. As a consequence, many drugs used in paediatric patients are prescribed without full knowledge on the optimal dosage and potential adverse drug reactions [1,2].…”

Child vs Adult Randomized Controlled Trials in Specialist Journals

“…1 However, there are logistical, financial, and ethical barriers to conducting trials involving children. 2 Despite financial and legislative incentives to promote conducting pediatric trials, there is evidence that the gap between the number of important pediatric and adult trials is widening. The number of adult trials published annually in high-impact general medical journals has doubled during the past 20 years, with virtually no change in pediatric trials.…”

DigiKnowIt News: Educating youth about pediatric clinical trials using an interactive, multimedia educational website

“…Additionally, some families might be unaware of the benefits of clinical trials or opportunities to engage in research (Natale et al, 2017). Some benefits to participating in pediatric clinical trials include direct access to and benefitting from new treatments that are not yet available, learning more about one’s illness, better medical care, and close monitoring of health (Caldwell et al, 2004; Morales-Olivas and Morales-Carpi, 2006; Walders-Abramson et al, 2016). In a qualitative study, personal and health benefits emerged as important reasons that children chose to participate in a clinical trial (Luchtenberg et al, 2015).…”