The Food and Drug Administration (FDA) issued draft guidance on the development of anti-infective drug products for the pediatric population. The guidance is intended to provide recommendations on the development of these products and addresses the initiation of clinical pediatric clinical studies, enrollment strategies, extrapolation of efficacy, and other considerations.
In the draft guidance, the FDA emphasizes that it is important to conduct clinical studies of anti-infective drugs in the pediatric population to accurately assess safety and to inform dosing. Additionally, the FDA recommends that sponsors consider the following when developing anti-infective drugs:
- Efficacy extrapolation – The FDA notes that efficacy results from adequate and well-controlled clinical trials can be extrapolated to pediatric populations if: 1) the course of the infectious disease is similar in adult and the pediatric populations; and 2) the effects of the drug product are sufficiently similar in adult and pediatric populations. The agency does clarify that while efficacy can be extrapolated, pediatric data will be needed to assess the safety of the drug product.
- Age cohorts – The guidance details that cohorts of pediatric studies should be determined based on the incidence of the disease and any specific considerations for the drug product evaluation. The FDA also encourages the enrollment of some age cohorts in parallel for drug products that do not have any specific safety concerns that warrant a different approach. Pediatric patients could also be enrolled in Phase 3 adult clinical trials.
- Safety data – The agency recommends that safety data be collected in all pediatric age ranges for which the drug product will be indicated, using the intended dose and duration of the drug product. Additionally, the guidance notes that the size of the recommended pediatric safety database of a drug product depends on the prevalence of the disease, adverse event profile of the drug product, and expected use of the drug product in the pediatric population.
Comments are due August 29.