On March 14, 2018, FDA issued draft guidance on Pediatric HIV Infection: Drug Development for Treatment. The draft guidance provides general guidance on the development of drug products and clinical trial designs for treating pediatric HIV patients. This guidance does not address the full scope of considerations for drug development or clinical trial designs for treating adult or pediatric HIV patients.
The draft guidance identifies several major considerations for sponsors developing pediatric HIV drugs for global use. Regarding clinical trial designs, FDA recommends sponsors to: (1) include adolescents in its initial efficacy (phase III) trials or conduct a separate adolescent study in parallel with its adult phase III trials because dosing recommendations for antiretroviral drugs for adults and adolescents have been historically similar for the two groups, (2) base cohort enrollment and dose selections on weight-bands, predefined by the World Health Organization, rather than age during nonadolescent pediatric clinical studies, (3) enroll nonadolescent pediatric population cohorts within clinical studies in parallel rather than in series, unless the drug product in the studies warrants a different approach, and (4) use pharmacokinetic modeling using the adult and adolescent data for initial dose selection to initiate parallel enrollment of cohorts across the different weight groups in the nonadolescent pediatric population. Regarding drug development, FDA further advises sponsors to: (1) begin pediatric formulation development as soon as an adult dose is selected based on results from phase II trials, (2) obtain approval of new pediatric formulations by using comparable bioavailability/bioequivalence studies in adults, and (3) have early discussions with the WHO, FDA, and other organizations regarding facilitating drug development and meeting the needs of pediatric patients.
Readers are encouraged to read the draft guidance, also available on FDA’s website.