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Primary Endpoints in Pediatric Efficacy Trials Submitted to the US FDA

Dionna J Green1, Janelle M Burnham1, Paul Schuette2

  • 1Office of Clinical Pharmacology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA.

Journal of Clinical Pharmacology
|April 18, 2018
PubMed

Insights

Selecting the same primary efficacy endpoint as in adult trials and enrolling both pediatric and adult patients in the same study significantly increases pediatric drug development success rates.

Area of Science:

  • Clinical Pharmacology
  • Pediatric Research
  • Drug Development

Background:

  • Pediatric drug development faces high failure rates, necessitating optimized trial designs.
  • Endpoint selection is a critical factor influencing the success of pediatric clinical trials.
  • US Food and Drug Administration (FDA) submissions provide valuable data on trial design and outcomes.

Purpose of the Study:

  • To identify primary efficacy endpoints in pediatric drug development trials submitted to the FDA.
  • To analyze the relationship between endpoint characteristics and trial success.
  • To determine factors associated with successful pediatric drug development.

Main Methods:

  • Analysis of 234 pediatric pivotal efficacy trials for 138 unique products (September 2007–July 2016).
  • Inclusion criteria: pediatric studies with a corresponding adult trial for the same indication.
  • Comparison of trial success rates based on endpoint similarity to adult trials and patient population (pediatric-only vs. combined).

Main Results:

  • Trials using the same primary efficacy endpoint as adult trials succeeded more often (86.5%) than those with differing endpoints (61.3%).
  • Combined pediatric and adult trials showed higher success rates (85/95) compared to pediatric-only trials (94/139).
  • No significant difference in success rates was observed between subjective and objective endpoints in pediatric trials.

Conclusions:

  • Using the same primary efficacy endpoint as in the corresponding adult trial is associated with increased pediatric trial success.
  • Enrolling both pediatric and adult patients within the same trial is a significant attribute linked to higher success rates.
  • These findings highlight key design elements to improve the likelihood of success in pediatric drug development.

Keywords:
US FDAclinical trialsendpointspediatricsregulatory/scientific affairs

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