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Drug Development for Pediatric Populations: Regulatory Aspects

Jochen Zisowsky1, Andreas Krause2, Jasper Dingemanse2

  • 1Actelion Pharmaceuticals Ltd, Clinical Pharmacology, Gewerbestrasse 16, CH-4123 Allschwil, Switzerland. jochen.zisowsky@actelion.com.

Pharmaceutics
|October 11, 2016
PubMed

Insights

Regulatory requirements for pediatric drug development are increasingly enforced, leading to more trials in children. This review outlines industry strategies for pediatric study planning, data generation, and the use of modeling and simulation (M&S) for pharmacokinetics (PK) and pharmacodynamics (PD).

Area of Science:

  • Pharmacology
  • Regulatory Science
  • Pediatric Medicine

Background:

  • Pediatric drug development is now integrated early in the drug creation pipeline.
  • Regulatory agencies like the European Medicines Agency (EMA) and Food and Drug Administration (FDA) have strengthened requirements for pediatric data.
  • This has led to a rise in clinical trials specifically designed for pediatric populations.

Approach:

  • This review synthesizes regulatory requirements and industry strategies for developing drugs for children.
  • It details essential aspects including study planning, first-dose considerations, sampling techniques, and data analysis for pharmacokinetic (PK) and pharmacodynamic (PD) assessments.
  • The crucial role of Modeling and Simulation (M&S) in pediatric drug development, supported by regulatory guidelines, is emphasized with practical examples.

Key Points:

  • Key elements of pediatric drug development include regulatory compliance, strategic study design, and robust data collection.
  • Pharmacokinetic (PK) and pharmacodynamic (PD) profiling in children requires specialized approaches to sampling and analysis.
  • Modeling and Simulation (M&S) offers a powerful tool to bridge data gaps and support regulatory submissions for pediatric drug applications.

Conclusions:

  • Adherence to EMA and FDA guidelines is paramount for successful pediatric drug development.
  • Effective pediatric drug development necessitates a comprehensive strategy encompassing planning, execution, and advanced analytical methods.
  • Modeling and Simulation (M&S) is integral to understanding drug behavior in children and meeting regulatory expectations.

Keywords:
Modeling and SimulationPIPPK/PD  modelingchildrenclinical studieshealth authoritiespediatric drug developmentpediatric legislationpediatricsregulatory guidelines

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