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Research Article: Need for Representation of Pediatric Patients with Obesity in Clinical Trials

Date Published: 2023-09-30

Abstract:
Clinical trials are an integral aspect of drug development. Tremendous progress has been made in ensuring drug products are effective and safe for use in the intended pediatric population, but there remains a paucity of information to guide drug dosages in pediatric patients with obesity. This is concerning because obesity may influence the disposition of drug products. When pediatric patients with obesity are not enrolled in clinical trials, dosing options for use in this subpopulation may be suboptimal. Reliance on physiological-based dosing strategies that are not informed by evaluation of the pharmacokinetics of the drug product could lead to under- or over-dosing with ensuing therapeutic failure or toxicity consequences. Thus, representation of pediatric patients with obesity in clinical trials is crucial to understand the benefit-risk profile of drug products in this subpopulation. It is important to acknowledge that this is a challenging endeavor, but not one that is insurmountable. Collective efforts from multiple stakeholders including drug developers and regulators to enhance diversity in clinical trials can help fill critical gaps in knowledge related to the influence of obesity on drug disposition.

Introduction:
Conducting clinical trials in the pediatric population is complex due in part to maturational and physiological development across age groups, ethical considerations, and feasibility. Despite these challenges, clinical trials are integral to advance pediatric drug development. In the United States (US), legislative endeavors have substantially increased the number of pediatric drug studies conducted across a wide range of US Food and Drug administration (FDA) therapeutic areas [1,2]. Since 1998, over 1000 drug…

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