Early Development and Translational/Quantitative Pharmacology Strategies in Pediatric Drug Development
“Traditional” approaches to pediatric development of small molecules involves gaining approval or collecting significant clinical data in adults prior to bridging to the pediatric population.
Challenges in pediatric programs, which include selection of starting doses, PK and PD sampling strategies, and study design are amplified when significant adult safety and efficacy data are not available.
This seminar described the application of early development strategies to enable development in children, in cases where limited adult data are available. Extensive use of translational science and quantitative modeling and simulation methods were key underpinnings to facilitate the successful execution of these programs.