Systematic Review of Early Phase Pediatric Clinical Pharmacology Trials
Deejesh Subramanian1, Cintia V Cruz2,3, Facundo Garcia-Bournissen4
1Schulich School of Medicine and Dentistry (DS), University of Western Ontario, London, Ontario, Canada.
Insights
Early phase pediatric clinical trials are increasing and can be conducted safely. Findings show that concerns about child safety in these trials are disproportionate to observed adverse effects, supporting their contribution to pediatric pharmacotherapy.
Area of Science:
- Pediatric Pharmacology
- Clinical Trial Design
- Drug Development
Background:
- Historically, children were excluded from early-phase clinical trials due to safety concerns, not data.
- The need for pediatric-specific therapies and adult drug repurposing necessitates pediatric participation in early trials.
Purpose of the Study:
- To systematically review the history and current status of early-phase pediatric clinical pharmacology trials.
- To analyze safety concerns, trends, and challenges in pediatric trials.
Main Methods:
- Systematic literature search of MEDLINE for pediatric non-oncology phase 1 or phase 1-2 drug and vaccine studies.
- Analysis of trial data to assess safety and trends.
Main Results:
- The number of early-phase pediatric trials is increasing but remains lower than in adults.
- A low incidence of serious adverse effects was observed in these trials.
Conclusions:
- Concerns regarding child safety in early-phase trials appear disproportionate to actual risks.
- Pediatric early-phase clinical trials can be conducted safely and are vital for advancing pediatric pharmacotherapy.
Objective:
Children have generally been excluded from early-stage clinical trials owing to safety concerns based in social expectations and not data. However, the repositioning of adult therapeutics for pediatric use and the increase in the development of therapies for pediatric only conditions require the participation of children in phase 1-2 trials. Therefore, the aim of this article is to systematically review the history and current state of early phase pediatric clinical pharmacology trials in order to understand safety concerns, trends, and challenges in pediatric trials.
Methods:
This review analyzed the nature of early phase pediatric clinical trials conducted for nononcology conditions through a systematic search that was performed for pediatric non-oncologic phase 1 or phase 1-2 drug and vaccine studies in MEDLINE.
Results:
The data show that the number of early phase pediatric clinical trials is still small relative to adults but has been on the rise in the past decade with relatively few serious adverse effects observed.
Conclusions:
The widespread concerns about children's safety when they participate in early phase clinical trials seem disproportionate, based on our findings. The data confirm that these studies can be conducted safely, and that their results can contribute significantly to pediatric pharmacotherapy.
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