Keywords: HIV, pediatrics, neonates
Improvement of HIV treatments in recent years has resulted in a dramatic decline in the incidence of new pediatric HIV infections worldwide. Nonetheless, significant unmet medical need for development of pediatric age- and weight-appropriate dosing formulations exists. From a statistical perspective, many methodological / operational difficulties and opportunities arise: selecting an appropriate strategy for evaluating pharmacokinetic, safety, and activity/efficacy parameters; prospectively defining decision criteria based on interim data analyses to support enrollment in additional/expanded dosing cohorts; enhancement of study design via historical borrowing of pediatric prior data; extrapolation from adults to pediatric study populations via modeling/simulation; prevention of HIV transmission from HIV+ pregnant women to newborns/neonates. Additionally, given that pediatric HIV clinical trial network sponsored trials often serve as the basis for registration, establishing collaborative working models with pediatric HIV clinical trial networks is of critical importance. This session will address the aforementioned topics, identifying both challenges and opportunities for novel and efficient pediatric HIV study design and analysis through close collaboration between industry and pediatric clinical trial networks.
Key questions to guides the discussion include the following:
1) What are appropriate strategies for evaluating PK, safety, and efficacy parameters in HIV pediatric studies? 2) What opportunities arise for enhancement of study design via historical borrowing of prior data and extrapolation from adult to pediatric settings? 3) How are design and analysis issues of neonate studies of HIV- participants different than those based on HIV+ pediatric participants? 4) What are some challenges and opportunities in non-industry sponsored collaborative research to support registration of pediatric formulations.