Dose Ranging in Pediatric Drug Development Trials Submitted to the US FDA 2012-2020.

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Tác giả: Gelareh Abulwerdi, Gilbert J Burckart, Dionna J Green, Francis G Green, Connor M I Kelley, Eugenia Kwon, Carlton K K Lee, Jennifer Pham, Sherbet Samuels

Ngôn ngữ: eng

Ký hiệu phân loại: 571.876 +Development in distinct stages

Thông tin xuất bản: England : Journal of clinical pharmacology , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 687870

 Dose selection is a critical process within pediatric drug development and dose-ranging studies are integral to establish a reasonable dose. The objective of this analysis was to examine the dose-ranging trials utilized in pediatric drug development and to determine (1) the dose-ranging strategies that were used in all available pediatric dose-ranging studies, (2) the success of achieving pediatric labeling in those submissions to the US Food and Drug Administration, and (3) ethical aspects of providing a prospect of direct benefit to pediatric patients in dose-ranging studies. Of the 275 programs that previously surveyed pediatric drug development programs from 2012 to 2020, it was determined that dose-ranging studies were used for 97 (35.3%) programs. The three categorizations of these 97 programs included the parallel dose design (n = 66
  68%), the dose-escalation design (n = 18
  18.6%), and the crossover design (n = 13
  13.4%). In the 66 that used a parallel design, 41 of these products were approved for use in pediatric patients. In 13 out of the 41 drugs (31.7%) approved for pediatric use using parallel dose ranging, the lowest parallel dose (patient on the dose for the entire study) was lower than the approved dose. Dose ranging remains an important strategy for optimizing dosing, but ethical considerations and the need to optimize benefit for individual patients should drive decisions about dosing approaches in pediatric patients. The inclusion of adaptive designs is one possible approach to optimizing dose-ranging studies for pediatric patients.
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