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Sponsor Initiated Requests to Delay Pediatric Postmarketing Studies

Susan M Abdel-Rahman1,2, Yutao Gong3, Caleb Choi3

  • 1Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA. susan.abdel-rahman@fda.hhs.gov.

Pharmaceutical Medicine
|May 10, 2026
PubMed

Insights

Delays in mandated pediatric postmarketing studies (PMRs) are increasing due to clinical trial conduct issues, particularly affecting smaller companies. These delays prolong off-label drug use in children, highlighting a need for collaborative solutions.

Area of Science:

  • Pediatric pharmacology
  • Drug development regulation
  • Clinical trial management

Background:

  • Off-label drug use in children poses significant safety and effectiveness risks due to insufficient pediatric data.
  • Mandated pediatric postmarketing requirement (PMR) studies are crucial for addressing these data gaps.

Purpose of the Study:

  • To analyze the frequency and reasons for delays in completing mandated pediatric PMR studies.
  • To identify factors contributing to extended timelines in pediatric drug development.

Main Methods:

  • Utilized US Food and Drug Administration (FDA) data from 2012-2024 to examine pediatric PMRs and deferral extension (DE) requests.
  • Analyzed PMR completion dates, durations, DE request rationale, sponsor size, and trial enrollment status.

Main Results:

  • Identified 1160 pediatric PMRs, with 459 associated with 1176 DE requests.
  • Observed a decline in issued PMRs but an increase in DE requests, alongside shorter negotiated timelines.
  • Clinical trial conduct issues were the predominant reason for delays (48%), with smaller companies showing higher DE rates and lower success rates.

Conclusions:

  • Increasing DE requests indicate persistent challenges in timely pediatric trial completion.
  • Clinical trial conduct difficulties are the main driver of delays, though root causes require further investigation.
  • Cooperative efforts among regulators, sponsors, and stakeholders are needed to expedite pediatric drug development and reduce off-label use.
Abstract

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