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Adaptive paediatric investigation plans, a small step to improve regulatory decision making in drug development for

Peter Bauer1, Franz König2

  • 1Center for Medical Statistics, Informatics, and Intelligent Systems, Medical University of Vienna, Vienna, Austria. peter.bauer@meduniwien.ac.at.

Pharmaceutical Statistics
|July 13, 2016
PubMed

Insights

Drug developers must plan for pediatric drug development early, as mandated by EU regulations. Adaptive plans allow re-evaluation of extrapolation from adults to children, improving regulatory decisions and reducing trial burdens.

Area of Science:

  • Pharmaceutical development
  • Regulatory science
  • Pediatric drug safety

Background:

  • European Union regulations mandate early agreement on Paediatric Investigation Plans (PIPs) during adult drug development.
  • Extrapolation of data from adults to children is common practice to minimize pediatric trials but carries inherent uncertainties.
  • Early regulatory decisions regarding the extent of extrapolation can be highly uncertain.

Discussion:

  • The current regulatory process for pediatric drug development should incorporate flexibility.
  • Adaptive Paediatric Investigation Plans (PIPs) are proposed to allow re-evaluation of initial decisions.
  • This adaptivity enables informed adjustments based on emerging data from adult studies or other sources.

Key Insights:

  • Uncertainty in early extrapolation decisions necessitates a more adaptive regulatory approach.
  • Allowing for re-evaluation of extrapolation in pediatric drug development enhances decision quality.
  • Learning from accumulated data is crucial for optimizing the use of information borrowed from adult studies.

Outlook:

  • Implementing adaptive PIPs can lead to more robust and reliable pediatric drug approval pathways.
  • Future regulatory frameworks should prioritize flexibility and iterative decision-making.
  • This approach aims to improve the scientific basis for drug use in pediatric populations while maintaining safety and efficacy.

Keywords:
European regulationadaptiveclinical trialsdrug developmentextrapolationpaediatric medicine

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