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Analysis and reporting of pediatric growth and development assessment from clinical trials: overview and challenges

YounJeong Choi1, Nathalie Barbier2, Sabine Fürst-Recktenwald3

  • 1Data and Statistical Sciences, Genentech Inc, South San Francisco, California, USA.

Journal of Biopharmaceutical Statistics
|December 21, 2022
PubMed

Insights

Evaluating pediatric drug safety requires assessing normal growth and development changes. This paper offers practical recommendations for analyzing these crucial pediatric clinical trial endpoints.

Area of Science:

  • Pediatric clinical pharmacology
  • Developmental pediatrics
  • Clinical trial methodology

Background:

  • Pediatric drug development faces unique challenges in distinguishing drug effects from normal growth and maturation.
  • Children's development varies significantly by age, impacting how investigational drugs affect them.
  • Assessing the risk-benefit profile of pediatric medications necessitates careful consideration of developmental changes.

Purpose of the Study:

  • To provide practical considerations and recommendations for assessing growth and development in pediatric clinical trials.
  • To guide the evaluation of endpoints related to growth, sexual maturation, and neurocognitive development in pediatric populations.
  • To recommend basic analysis approaches for pediatric clinical trial data.

Main Methods:

  • Review of practical considerations for assessing growth and development in pediatric clinical trials.
  • Discussion of relevant endpoints and measures for growth, sexual maturation, and neurocognitive development.
  • Recommendation of basic statistical analysis approaches for pediatric data.

Main Results:

  • Highlights the importance of differentiating expected developmental changes from drug-induced effects.
  • Emphasizes age-specific considerations, such as sexual maturation in older children.
  • Provides a framework for evaluating the impact of investigational drugs on pediatric development.

Conclusions:

  • Effective assessment of pediatric drug safety relies on robust evaluation of growth and development.
  • Standardized methods and age-appropriate endpoints are crucial for pediatric clinical trials.
  • Recommendations are provided to improve the analysis of developmental data in pediatric drug studies.

Keywords:
Pediatricsanalysisdevelopmentgrowthmaturation

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