A Review of Trials for Unsuccessful Pediatric Drug Development Programs Submitted to the US Food and Drug Administration 2015-2022.

Sherbet Samuels, Mark Seaton, Susan M Abdel-Rahman, Gilbert J Burckart

Abstract readReview

In one paragraph

Review in Therapeutic innovation & regulatory science, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it

0cells of the map it votes in

2citing papers in PubMed

–field-weighted citation impact

1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

From Small Data to Big Decisions: How Clinical Pharmacology Shapes Rare Disease Development.Journal of clinical pharmacology · 2026
Review
Accelerating Pediatric Oncology Drug Development: Advances in Clinical Pharmacology, Trial Design, Non-Clinical Evidence, and Regulatory Science.Journal of clinical pharmacology · 2026
Review

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors.

Sherbet SamuelsOffice of Clinical Pharmacology, Center for Drug Evaluation and Research, Food and Drug Administration, Bldg. 51 Rm 3184, 10903 New Hampshire Ave, Silver Spring, MD, 20993, USA.

Mark SeatonOffice of Study Integrity and Surveillance, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, MD, USA.

Susan M Abdel-RahmanOffice of Clinical Pharmacology, Center for Drug Evaluation and Research, Food and Drug Administration, Bldg. 51 Rm 3184, 10903 New Hampshire Ave, Silver Spring, MD, 20993, USA.

Gilbert J BurckartOffice of Clinical Pharmacology, Center for Drug Evaluation and Research, Food and Drug Administration, Bldg. 51 Rm 3184, 10903 New Hampshire Ave, Silver Spring, MD, 20993, USA. Gilbert.Burckart@fda.hhs.gov.

Funding

Intramural FDA HHS FD999999

6 · The paper itself

Abstract

backgroundLegislative initiatives have spurred an increase in pediatric drug development programs. However, some drug products studied in the pediatric population have not received an approved pediatric indication, and efforts have been made to improve the outcomes of a greater percentage of pediatric trials.

objectiveThis analysis evaluated the rate of failure and the factors associated with those unsuccessful outcomes of recent pediatric drug development programs in comparison to earlier pediatric drug development programs.

methodsPublicly available information for non-oncology pediatric drug development programs submitted to the FDA between 2015 and 2022 were reviewed.

resultsFDA reviews and drug product labeling for 211 drug products were examined. Of these, 32 (32/211, 15%) drug products for which pediatric trials were conducted did not receive an approved pediatric indication. The reasons for these unsuccessful outcomes were failure to demonstrate effectiveness only (18/32, 56%), failure to demonstrate safety only (7/32, 22%), or failure to demonstrate both effectiveness and safety (7/32, 22%). The psychiatry (8/32, 25%) and pain (5/32, 17%) therapeutic areas had the highest number of drug products that did not receive a pediatric indication.

conclusionThe findings from this review suggest that, although this represents an improvement from pre-2012 pediatric drug development programs, that basic problems are still encountered in pediatric trial designs and dose selection. The 15% failure rate may represent close to a best-case scenario for pediatric drug development presently, but an increased use of pediatric extrapolation could change that.

Indexed as

Clinical Trials as TopicDrug DevelopmentChildDrug ApprovalHumansPediatricsUnited StatesUnited States Food and Drug AdministrationDrug approvalDrug labelingPediatricPediatric drug developmentPediatric trial

Identifiers

PMID41046306

DOI10.1007/s43441-025-00864-1

PMCPMC12951222

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.