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Methods Used for Pediatric Dose Selection in Drug Development Programs Submitted to the US FDA 2012-2020
Francis G Green1, Kyunghun Park1, Gilbert J Burckart1
1Office of Clinical Pharmacology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.
Journal of Clinical Pharmacology
|June 29, 2021
Summary
Pediatric drug development faces dosing challenges. This study analyzed 275 programs (2012-2020), finding a 76.4% labeling success rate, with "titration to target response" emerging as a key strategy.
Area of Science:
- Pharmacology
- Pediatric Drug Development
- Clinical Trial Design
Background:
- Pediatric drug dosing is complex, often leading to trial failures and lack of FDA labeling.
- A structured approach is needed to improve pediatric dose selection success rates.
Purpose of the Study:
- To analyze pediatric dosing strategies from 2012-2020.
- To identify initial dose selection methods and pivotal dosing strategies for pediatric clinical trials.
Main Methods:
- Characterized 275 pediatric drug development programs for dosing strategies.
- Analyzed initial and pivotal dose selection approaches.
- Included data up to September 2020.
Main Results:
- Achieved a 76.4% success rate for pediatric drug labeling.
- Common initial strategies: prior product experience, allometric scaling, adult exposure matching.
- Most frequent pivotal strategies: titration to target response (33%), pharmacokinetic/pharmacodynamic studies (30%), exposure matching (20%).
- Model-informed drug development used in ~50% of programs.
Conclusions:
- Titration to target response may indicate a move towards precision medicine in pediatrics.
- A structured, evidence-based approach for pediatric dose selection is recommended.
- Continued research is vital for optimizing pediatric drug dosing.