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Background <p>Legislative initiatives have spurred an increase in pediatric drug development programs. However, some drug products studied in the pediatric population have not received an approved pediatric indication, and efforts have been made to improve the outcomes of a greater percentage of pediatric trials.</p> Objective <p>This analysis evaluated the rate of failure and the factors associated with those unsuccessful outcomes of recent pediatric drug development programs in comparison to earlier pediatric drug development programs.</p> Methods <p>Publicly available information for non-oncology pediatric drug development programs submitted to the FDA between 2015 and 2022 were reviewed.</p> Results <p>FDA reviews and drug product labeling for 211 drug products were examined. Of these, 32 (32/211, 15%) drug products for which pediatric trials were conducted did not receive an approved pediatric indication. The reasons for these unsuccessful outcomes were failure to demonstrate effectiveness only (18/32, 56%), failure to demonstrate safety only (7/32, 22%), or failure to demonstrate both effectiveness and safety (7/32, 22%). The psychiatry (8/32, 25%) and pain (5/32, 17%) therapeutic areas had the highest number of drug products that did not receive a pediatric indication.</p> Conclusion <p>The findings from this review suggest that, although this represents an improvement from pre-2012 pediatric drug development programs, that basic problems are still encountered in pediatric trial designs and dose selection. The 15% failure rate may represent close to a best-case scenario for pediatric drug development presently, but an increased use of pediatric extrapolation could change that.</p>

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A Review of Trials for Unsuccessful Pediatric Drug Development Programs Submitted to the US Food and Drug Administration 2015–2022

  • 期刊论文

  • 发表日期：

    2025年10月4日

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  • Sherbet Samuels,
  • Mark Seaton,
  • Susan M. Abdel-Rahman,
  • Gilbert J. Burckart

摘要

Background

Legislative initiatives have spurred an increase in pediatric drug development programs. However, some drug products studied in the pediatric population have not received an approved pediatric indication, and efforts have been made to improve the outcomes of a greater percentage of pediatric trials.

Objective

This analysis evaluated the rate of failure and the factors associated with those unsuccessful outcomes of recent pediatric drug development programs in comparison to earlier pediatric drug development programs.

Methods

Publicly available information for non-oncology pediatric drug development programs submitted to the FDA between 2015 and 2022 were reviewed.

Results

FDA reviews and drug product labeling for 211 drug products were examined. Of these, 32 (32/211, 15%) drug products for which pediatric trials were conducted did not receive an approved pediatric indication. The reasons for these unsuccessful outcomes were failure to demonstrate effectiveness only (18/32, 56%), failure to demonstrate safety only (7/32, 22%), or failure to demonstrate both effectiveness and safety (7/32, 22%). The psychiatry (8/32, 25%) and pain (5/32, 17%) therapeutic areas had the highest number of drug products that did not receive a pediatric indication.

Conclusion

The findings from this review suggest that, although this represents an improvement from pre-2012 pediatric drug development programs, that basic problems are still encountered in pediatric trial designs and dose selection. The 15% failure rate may represent close to a best-case scenario for pediatric drug development presently, but an increased use of pediatric extrapolation could change that.