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# Progress Seen in Pediatric Studies
- URL: https://www.fdaweb.com/progress-seen-in-pediatric-studies/
- Published: 2016-11-15T12:00:00.000Z
- Updated: 2026-09-14T21:48:00.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5137338

A 2016 update of a 2007 Tufts Center for the Study of Drug Development assessment of drug developers’ experiences with FDA’s two pediatric studies initiatives shows that pediatric regulatory science and clinical trial networks hold promise for pediatric studies. Experts were [surveyed](http://csdd.tufts.edu/reports/purchase%5Fsingle%5Fimpact%5Freport?issue=Vol.%2018%20No.%306&ref=fdaweb.com) (purchase required) on progress being made under the Best Pharmaceuticals for Children Act of 2002 (BPCA) and the Pediatric Research Equity Act of 2003 (PREA), as well as the changes in the pediatric studies environment since the center’s 2007 report.

The survey respondents indicated that progress is being made toward achieving BPCA and PREA goals. Most said that since 2008, progress toward the laws’ goals has been either “good” or “moving in the right direction,” especially in increasing pediatric dosing information in labeling, something that FDA said was among the most important goals. The report says that making pediatric studies a routine part of drug development, considered to be the most ambitious goal, had one of the highest scores, with 36% of respondents agreeing that “a great deal of progress” had been made, and the rest saying it had been “good” or “moving in the right direction.”

Pediatric study costs have increased, the respondents said. Some 72% said the increases were due to patient recruitment and retention costs, while 81% attributed them to modifications/amendments to study plans after study starts. Formulation development/testing and long-term safety studies were cited by 64% and 46% of respondents, respectively, as major factors in cost increases.

Most respondents also said that pediatric study complexity has increased more than 50% since 2008\. Factors cited included lack of age-appropriate validated endpoints, lack of sufficient safety information to start studies, and differences between pediatric and adult manifestations of the same disease. Less often named were commercial decisions and reimbursement obstacles.

Tufts says that the availability of increased resources for pediatric drug development generated the greatest variability among survey responses, likely reflecting different starting points for building capacity in those research and development activities, as well as changes in therapeutic area focus at respondents’ companies.

Survey respondents said that the research environment has improved slowly but steadily since 2008\. They note that conducting neonatal studies, developing age-appropriate formulations, and undertaking studies of diseases rare in children were vexing problems and the focus of changes in the FDA Amendments Act and the FDA Safety and Innovation Act. Thus, they believe that real progress has been made with availability of child-friendly formulations allowing more accurate dosing, ease of administration, and better treatment adherence. The drug development environment for pediatric rare diseases, including pediatric oncology, continues to “move in the right direction,” survey respondents said. However, they added, including neonates in routine drug development will likely remain challenging in the near term.

When asked what holds the most promise for improving the reliability of pediatric drug development, respondents cited pediatric regulatory science, including more accurate methods of extrapolating evidence from older age groups to younger ones, as well as modeling and simulation techniques. They also said that improved pediatric clinical trial networks promise more efficient and effective management of trial resources, including investigators, patients, and study sites, especially for multi-country studies.