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Challenges in clinical trials for children and young people

Florian B Lagler1,2, Steven Hirschfeld3, Jenny M Kindblom4,5

  • 1Institute for Inherited Metabolic Diseases and Department of Pediatrics, Paracelsus Medical University, Clinical Research Center Salzburg GmBH, Strubergasse 21, Salzburg, Austria.

Archives of Disease in Childhood
|October 20, 2020
PubMed

Insights

Pediatric clinical trials are crucial for ensuring children receive safe and effective medicines, but face significant challenges. Strengthening pediatric research is essential to bridge this knowledge gap and advance child healthcare.

Area of Science:

  • Pediatric Pharmacology
  • Clinical Trial Design
  • Regulatory Science

Background:

  • A significant knowledge gap exists regarding the efficacy and safety of medications in pediatric populations.
  • Children are frequently treated with medications off-label due to insufficient pediatric-specific data.
  • Legislation in North America and the EU acknowledges this gap, yet pediatric clinical trials remain challenging.

Purpose of the Study:

  • To highlight the challenges and special considerations in conducting pediatric clinical trials.
  • To emphasize the need for robust pediatric research to inform evidence-based treatment for children.
  • To advocate for strengthening pediatric clinical research infrastructure and expertise.

Main Methods:

  • The abstract discusses the inherent complexities of pediatric clinical trials, including scientific, ethical, and logistical aspects.
  • It reviews legislative efforts and their impact on pediatric drug development.
  • Expertise in trial design, planning, coordination, and research infrastructure is identified as critical.

Main Results:

  • Pediatric clinical trials present unique challenges compared to adult trials, such as small sample sizes, heterogeneity, and age-appropriate consent procedures.
  • Despite legislative efforts, conducting effective pediatric clinical trials remains a significant hurdle.
  • There is a continued need for trials across all developmental stages and therapeutic areas.

Conclusions:

  • Strengthening pediatric clinical research is imperative to ensure children benefit from medical advancements equally to adults.
  • Addressing the unique scientific and ethical considerations in pediatric trials is key.
  • Enhanced research infrastructure and collaborative networks are vital for generating high-quality pediatric data.

Keywords:
evidence-based medicinegeneral paediatricspharmacology

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