Keywords: Pediatric, Extrapolation
Since the Pediatric Research Equality Act (PREA) was signed into law, followed by other regulatory agencies and general principles outlined in ICH E11 (2000), the rigor required for successful pediatric drug development programs are increasing globally. How to obtain adequate pediatric efficacy and safety data for labeling, while still shortening the time gap between adult approval and incorporation of pediatric information in labeling, and how to find treatment for pediatric-specific diseases, have all become key challenges in the current pediatric drug development era. In addition, to address these questions, a new ICH guideline was proposed in Oct 2017 to build more detailed guidance about how pediatric extrapolation can be used. During this round table session, we will focus on the above questions; and perhaps brainstorm ideas to contribute to the newly proposed guideline.
The issues in pediatric studies were presented in round table sessions previously. However, the innovative quantitative approaches to the review of existing data and a minimized approach to the number of children exposed to treatment are still developing. This session will gather real world evidence and further exchange knowledge among statisticians.
Some questions to facilitate the discussion are as follows: • What type pediatric study design have you used recently? Is it pharmacokinetics, efficacy, safety, or post-marketing information? How was the sample size determined? • Share the experiences you had with global regulatory agencies when submitting your pediatric study. Did you receive review comments that can help with the study design? • Under what circumstances can efficacy data be extrapolated from adequate and well-controlled studies in adults to pediatric patients?