NCHR’s Public Comments on the FDA’s Draft Guidance Regarding the Development of Anti-Infective Drug Products for the Pediatric Population

August 31, 2020

National Center for Health Research’s Comments on the Draft Guidance Regarding the Development of Anti-Infective Drug Products for the Pediatric Population

[Docket Number FDA-2020-D-1518]

We are writing to express our views on the Food and Drug Administration’s (FDA) draft guidance regarding the development of anti-infective drugs for the pediatric population. 

The National Center for Health Research is a nonprofit think tank that conducts, analyzes, and scrutinizes research on a range of health issues, with particular focus on which prevention strategies and treatments are most effective for which patients and consumers. We do not accept funding from companies that make products that are the subject of our work, so we have no conflicts of interest.

To ensure that drugs are safe and effective, it is of utmost importance that sponsors collect data on safety and effectiveness for all groups of people for whom the drug will be indicated. We support the recommendations in the draft guidance ensuring that anti-infective drugs are proven to be safe and effective for pediatric populations in order to include children of various ages in the indication. 

For that reason, we agree that safety data should be collected for all pediatric age ranges for which a drug will be indicated, using the intended dose and the intended duration of the drug. However, we are concerned about the statement that sponsors can extrapolate results from an adult clinical trial if both the course of the disease is similar between adult and pediatric populations, and if the effects of the drug are similar between both populations. Children are not small adults, and the impact of medication can differ substantially in children of different ages.  For that reason, data on children should be required prior to approval. It is not possible to ensure the accuracy of extrapolating results from adult to pediatric populations or older to younger pediatric populations. Well-controlled studies are essential to provide the information needed. Since adolescents can understand their role as patients in a clinical trial, these well-controlled studies should include adolescents ages 12 and older in all pivotal trials.  

We agree with the recommendation that the cohorts in pediatric studies should be determined based on the incidence of the disease as well as any specific considerations under evaluation, such as weight-based criteria. We also support the recommendation that sponsors should limit the number of samples obtained and the number of invasive procedures for pediatric laboratory testing.