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Duration of Pediatric Clinical Trials Submitted to the US Food and Drug Administration
Kanecia O Zimmerman1,2, P Brian Smith1,2, Ann W McMahon3
1Duke Clinical Research Institute, Duke University School of Medicine, Durham, North Carolina.
Insights
Most pediatric drug trials supporting long-term use are short, often under a year. This limited duration may not fully assess safety for children requiring chronic medication.
Area of Science:
- Pediatric pharmacology
- Clinical trial design
- Drug safety evaluation
Background:
- Rising rates of chronic diseases in children necessitate long-term drug therapies.
- Systematic evaluation of pediatric drug trial durations is crucial for pharmacovigilance.
- Understanding trial lengths informs safety strategies for chronic pediatric medication use.
Purpose of the Study:
- To analyze the duration of clinical trials submitted for US Food and Drug Administration (FDA) approval of pediatric drugs.
- To focus on trials for medications intended for long-term use in children.
Main Methods:
- Review of safety and efficacy trials submitted to the FDA (2007-2014) under the Best Pharmaceuticals for Children Act and Pediatric Research Equity Act.
- Analysis focused on drugs frequently used for chronic conditions in pediatric populations.
- Statistical analysis conducted between July 2015 and December 2017.
Main Results:
- 306 trials for 86 drugs indicated for long-term pediatric use were analyzed.
- Median maximum trial duration was 44 weeks; 61% of drugs had trials under 52 weeks.
- Neurologic, pulmonary, and anti-infective drugs were most commonly evaluated.
Conclusions:
- Pediatric clinical trials supporting FDA approval are often of limited duration.
- The short duration may be insufficient for assessing long-term safety in children.
- New approaches are needed to evaluate the safety of chronic drug use in pediatric patients.
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 Research 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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