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Exposure–Response Assessment in Pediatric Drug Development Studies Submitted to the US Food and Drug Administration
Author(s) -
Zhang Yifei,
Wang Yaning,
Khurana Mona,
Sachs Hari Cheryl,
Zhu Hao,
Burckart Gilbert J.,
Alexander John,
Yao Lynne P.,
Wang Jian
Publication year - 2020
Publication title -
clinical pharmacology and therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.941
H-Index - 188
eISSN - 1532-6535
pISSN - 0009-9236
DOI - 10.1002/cpt.1809
Subject(s) - medicine , food and drug administration , drug , dosing , drug development , clinical trial , context (archaeology) , leverage (statistics) , clinical study design , pharmacology , intensive care medicine , computer science , paleontology , machine learning , biology
Exposure–response (E–R) modeling provides a quantitative tool to leverage adult data to support pediatric trial design and drug approval. The pediatric E–R studies submitted to US Food and Drug Administration (FDA) between 2007 and 2018 were surveyed in the context of various types of trial designs supporting drug approval in the pediatric population. The applications of E–R evaluation in pediatric drug development programs are mainly focused on three areas: (i) supporting pediatric extrapolation when the E–R relationships are similar between the pediatric and adult populations; (ii) dose selection to balance the risk–benefit profile based on the change in efficacy and safety response with different exposure levels; and (iii) approval of a new formulation, new dosing regimen, or new route of administration, where E–R evaluation helps quantify the change in clinical response between the old and new strategies. E–R modeling will continue to play an expanded role in pediatric drug development in the future.