Accelerating Pediatric Drug Development

As noted in the Therapeutic Innovation & Regulatory Science special section on “Innovation and Opportunities in Pediatric Therapeutic Development” published in 2019, progress has been made over the past 15 years with respect to labeling drugs for pediatric patients. Unfortunately, there continues to be an approximately 9-year delay from drug approval in adults to labeling for pediatrics.

Efficient, innovative trials incorporating the voice of the global, ethnically diverse patient/parent population need to be initiated earlier in the drug development paradigm to reduce the off-label use of therapies where information about safety, dosing, and efficacy is lacking or limited in children. The next phase of accelerating pediatric drug development involves the fullest possible use of more complex trial designs and extrapolation while coordinating global pediatric networks, incorporating patients/parents into protocol development, and ensuring ethnic diversity and inclusivity in pediatric trials.

In this pediatric special section of Therapeutic Innovation & Regulatory Science, a series of articles propels us into the next phase of pediatric drug development that aims at decreasing the development timeline from adult approval to pediatric labeling. These efforts are focused on innovative trial designs; issues related to rare diseases and pregnant individuals; advances in pediatric formulation development; development of digital technologies in pediatric trials, including the digital capture of clinically meaningful endpoints; an update on pediatric global networks; and a discussion of stakeholder engagement with a focus on health equity and the voices of children and their families.

Once again, the mandate is for a public health requirement that ensures the availability of safety and efficacy data for all drugs prescribed for children.

Submissions to this special issue are by invitation only.

For questions regarding this special section, please email Dr. Sandra Blumenrath, managing editor of TIRS, at


  • Ronald J. Portman, MD

    Dr. Portman is head of Pediatric Clinical Development, Pediatric Center of Excellence at Novartis and a board-certified pediatrician and pediatric nephrologist. He joined Bristol Myers Squibb and Novartis after a career as professor and director of the Division of Pediatric Nephrology & Hypertension, University of Texas Medical School, Houston. His focus is on facilitating pediatric drug development in industry, emphasizing early drug development, extrapolation and innovative study design, adolescent/pediatric inclusion in adult trials, efficiencies in studies of drugs with multiple indications, and global pediatric clinical trial networks.

  • Susan McCune

    Dr. McCune is a board-certified pediatrician and neonatologist. She joined the FDA (2003) after an academic career at Children’s National Medical Center and Johns Hopkins Medicine. Dr. McCune was the director of the Office of Pediatric Therapeutics in the Office of the Commissioner, FDA (2017-2021), and joined Thermo Fisher in 2021. She is focused on facilitating pediatric drug development with a particular emphasis on global trials for neonates and rare diseases, development of biomarkers for pediatric studies, and inclusion of the voice of the pediatric patient in trial designs.

Articles (9 in this collection)