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

Details

• Alternative Title:
  JAMA Pediatr
• Personal Author:
  Zimmerman, Kanecia O. ; Smith, P. Brian ; McMahon, Ann W. ; Temeck, Jean ; Avant, Debbie ; Murphy, Dianne ; McCune, Susan
• Description:
  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.
  
  
• Subjects:
  Article
• Source:
  JAMA Pediatr. 173(1):60-67
• Pubmed ID:
  30452504
• Pubmed Central ID:
  PMC6526087
• Document Type:
  Journal Article
• Funding:
  R18 FD005292/FDA HHS/Food and Drug Administration/United States ; HHSN275201000003I/NICHD NIH HHS/National Institute of Child Health & Human Development/United States ; R21 HD080606/NICHD NIH HHS/National Institute of Child Health & Human Development/United States ; K23 HD091398/NICHD NIH HHS/National Institute of Child Health & Human Development/United States ; FD999999/Intramural FDA HHS/Intramural FDA HHS/United States ; U2C OD023375/ODCDC CDC HHS/Office of the Director/United States ; HHSN275201000003C/NICHD NIH HHS/National Institute of Child Health & Human Development/United States ; UL1 TR001117/NCATS NIH HHS/National Center for Advancing Translational Sciences/United States ; KL2 TR001115/NCATS NIH HHS/National Center for Advancing Translational Sciences/United States
• Volume:
  173
• Issue:
  1
• Collection(s):
  CDC Public Access
• Main Document Checksum:
  urn:sha256:87823d0f7a1c4ce21704e02bd5e39bd960e790f599476dd36e1687a717f848d3
• Download URL:
  https://stacks.cdc.gov/view/cdc/80495/cdc_80495_DS1.pdf
• File Type:
  [PDF - 237.02 KB ]