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Innovations in Pediatric Therapeutics Development: Principles for the Use of Bridging Biomarkers in Pediatric
Thomas R Fleming1, Christine E Garnett2, Laurie S Conklin3
1Department of Biostatistics, University of Washington, Seattle, WA, USA. tfleming@u.washington.edu.
Therapeutic Innovation & Regulatory Science
|September 3, 2022
Summary
Bridging biomarkers can help extrapolate adult drug effectiveness to children. Validated biomarkers ensure pediatric drug safety and efficacy, improving children's healthcare options.
Area of Science:
- Pediatric pharmacology
- Biomarker development
- Clinical trial design
Background:
- Pediatric populations require safe and effective medical interventions.
- Drug approval processes necessitate robust evidence of efficacy and safety.
- Pediatric extrapolation, using adult data, can streamline evaluating interventions for children.
Purpose of the Study:
- To explore the role of bridging biomarkers in pediatric extrapolation.
- To define criteria for effective bridging biomarkers in drug development.
- To enhance the availability of evaluated therapies for pediatric use.
Main Methods:
- Review of existing literature on pediatric extrapolation and biomarkers.
- Definition of criteria for bridging biomarker validation.
- Conceptual framework for using bridging biomarkers in pediatric drug evaluation.
Main Results:
- Bridging biomarkers can link adult study results to pediatric populations.
- Three key criteria proposed for validating bridging biomarkers.
- Validated biomarkers can support extrapolating efficacy from adults to children.
Conclusions:
- Bridging biomarkers are crucial for efficient pediatric drug evaluation.
- Proper use of biomarkers can increase access to approved pediatric therapies.
- This approach empowers informed healthcare choices for children and families.
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