This chapter has provided an overview of developmental pharmacology as a basis for designing, conducting, and evaluating pediatric drug studies. It has discussed how children’s growth and development may require alterations in research strategies that are commonly used in conducting drug studies with adults.

The exclusivity incentive and other features of BPCA and PREA explicitly recognize and accommodate some distinctive features of pediatric research. Notably, with direction from Congress and on its own initiative, FDA has applied additional expertise in pediatrics and pediatric research to the oversight of pediatric study requests or requirements, discussions with sponsors about acceptable research designs, and appropriate review of submitted pediatric data (see Chapters 3 and 4). By employing sufficient expertise in developmental pharmacology and pediatric clinical research from the early stages of pediatric plan discussion through the review of submitted studies, FDA increases the likelihood that studies will generate useful information to guide and improve clinical care for children of all ages.

The next chapter moves from developmental variability and pediatric research to public policy. It builds on the overview provided in Chapter 1 to discuss BPCA and PREA in more detail.

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