The FDA is committed to addressing scientific and regulatory issues unique to therapeutics development in children including leveraging of existing data to facilitate efficient and successful pediatric drug development programs.

Moreover, the Division of Pediatrics and Maternal Health (DPMH) in the Office of New Drugs engages in regulatory science research to address challenges in pediatric drug development, especially with development of therapeutics targeting rare pediatric diseases. DPMH, in collaboration with other Divisions and disciplines at the FDA, publishes papers in scientific journals that advance pediatric therapeutics development. A primary focus of DPMH’s regulatory science research in pediatrics involves research questions to address specific challenges encountered in the development of drugs for pediatric patients, including extrapolation of efficacy, use of complex innovative trial designs, application of biomarkers and surrogate endpoints, predicting and analyzing clinical pharmacology information in children, developing clinical trial endpoints, safety assessment, and analysis of negative trials. Additionally, DPMH has access to a large database of clinical trial data from over 1,600 pediatric clinical trials that can be used to address these challenges. DPMH establishes pediatric research collaborations using many effective mechanisms, including research collaboration agreements, memoranda of understanding, and Broad Agency Announcements. Moreover, the Division leverages innovative tools, including crowdsourcing challenges, to engage external stakeholders in identifying regulatory science research questions that can advance pediatric drug development.

The Office of Pediatric Therapeutics (OPT) is mandated by statute to help ensure access for children to innovative, safe and effective medical products as well as coordinate and facilitate activities across FDA that may have any effect on a pediatric population, the practice of pediatrics, or involve pediatric issues.

This responsibility is particularly significant given that, historically, many medical products have not been tested for use in children. OPT provides leadership and policy direction regarding issues of pediatric health, research, and product development, and has developed a number of interrelated programs to support FDA efforts to improve the development of products for the pediatric population spanning from the neonatal period through adolescence. OPT collaborates internally with FDA centers, as well as externally with government agencies, academia, and other stakeholders to facilitate cross-cutting research projects and scientific forums to advance FDA’s understanding of pediatric health and children’s unique needs. In addition, OPT supports extramural research through BAAs and grants.

OPT’s activities advance regulatory science through coordinating, facilitating, and conducting research projects intended to evaluate and answer important regulatory science questions relevant to informing pediatric product development, regulatory review, and safe use of pediatric products in children. OPT actively reviews and analyzes pediatric data submitted in product submissions to FDA, as well as real-world data, to identify lessons learned, develop best practices, and inform regulatory decision-making.