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Original Investigation
November 19, 2018

Duration of Pediatric Clinical Trials Submitted to the US Food and Drug Administration

Author Affiliations
  • 1Duke Clinical Research Institute, Duke University School of Medicine, Durham, North Carolina
  • 2Department of Pediatrics, Duke University School of Medicine, Durham, North Carolina
  • 3Office of Pediatric Therapeutics, US Food and Drug Administration, Silver Spring, Maryland
JAMA Pediatr. Published online November 19, 2018. doi:10.1001/jamapediatrics.2018.3227
Key Points

Question  What are the durations of pediatric clinical trials recently submitted to the US Food and Drug Administration, and how can this knowledge inform discussions of safety pharmacovigilance follow-up for drugs that might be used for long-term therapy in the pediatric population?

Findings  This study found that nearly two-thirds of pediatric clinical trials submitted to support the approval of drugs with potential long-term use in the pediatric population are shorter than 52 weeks.

Meaning  Pediatric clinical trials that are sufficient to support US Food and Drug Administration drug approval may require additional strategies to ensure data availability for understanding long-term drug safety in children.

Abstract

Importance  The increasing prevalence of pediatric chronic disease has resulted in increased exposure to long-term drug therapy in children. The duration of recently completed drug trials that support approval for drug therapy in children with chronic diseases has not been systematically evaluated. Such information is a vital first step in forming safety pharmacovigilance strategies for drugs used for long-term therapy in children.

Objective  To characterize the duration of clinical trials submitted to the US Food and Drug Administration (FDA) for pediatric drug approvals, with a focus on drugs used for long-term therapy.

Design and Setting  A review was performed of all safety and efficacy clinical trials conducted under the Best Pharmaceuticals for Children Act or the Pediatric Review Equity Act and submitted to the FDA from September 1, 2007, to December 31, 2014, to support the approval of drugs frequently used for long-term therapy in children. Statistical analysis was performed from July 1, 2015, to December 31, 2017.

Main Outcomes and Measures  Maximum duration of trials submitted to support FDA approval of drugs for children.

Results  A total of 306 trials supporting 86 drugs intended for long-term use in children were eligible for the primary analysis. The drugs most commonly evaluated were for treatment of neurologic (25 [29%]), pulmonary (16 [19%]), and anti-infective (14 [16%]) indications. The median maximum trial duration by drug was 44 weeks (minimum, 1.1 week; maximum, 364 weeks). For nearly two-thirds of the drugs (52 [61%]), the maximum trial duration was less than 52 weeks. For 10 of the drugs (12%), the maximum trial duration was 3 years or more. Maximum duration of trials did not vary by therapeutic category, minimum age of enrollment, calendar year, or legislative mandate.

Conclusions and Relevance  Pediatric clinical trials designed to sufficiently investigate drug safety and efficacy to support FDA approval are of relatively limited duration. Given the potential long-term exposure of patients to these drugs, the clinical community should consider whether new approaches are needed to better understand the safety associated with long-term use of these drugs.

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