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Alternate endpoints and clinical outcome assessments in pediatric ulcerative colitis registration trials

Haihao Sun1, Jessica J Lee, Elektra J Papadopoulos

  • 1*Office of Pediatric Therapeutics, Office of the Commissioner †Division of Gastroenterology and Inborn Errors Products ‡Study Endpoint and Labeling Development (SEALD) §Pediatrics and Maternal Health Staff, Office of New Drug, Center for Drug Research and Evaluation, Food and Drug Administration, Silver Spring, MD.

Journal of Pediatric Gastroenterology and Nutrition
|October 15, 2013
PubMed

Insights

Clinical outcome measures for pediatric ulcerative colitis (UC) lack consensus. This review of UC drug trials found inconsistent definitions for clinical response, hindering pediatric drug development.

Area of Science:

  • Pediatric Gastroenterology
  • Clinical Trial Design
  • Ulcerative Colitis Therapeutics

Background:

  • Assessing clinical outcomes in pediatric ulcerative colitis (UC) lacks standardized endpoint measures.
  • This variability complicates the evaluation of treatment efficacy in young patients.

Purpose of the Study:

  • To review the primary efficacy endpoints utilized in pivotal registration trials for drugs approved for pediatric UC.
  • To identify inconsistencies in outcome measures and definitions of clinical response.

Main Methods:

  • A comprehensive review of Food and Drug Administration (FDA)-approved drug trials for pediatric and adult UC from 1950 to 2008.
  • Analysis focused on primary efficacy endpoints and their specific definitions.

Main Results:

  • Two drugs, Colazal and Remicade, have been approved for pediatric UC, using clinical response as a primary endpoint.
  • Definitions of clinical response varied between trials, utilizing different indices like the Sutherland UC Activity Index and Mayo score, with modifications for pediatric studies.

Conclusions:

  • Existing pediatric UC trials employed specific endpoints, but significant inconsistencies in outcome measures and clinical response definitions were observed.
  • Establishing consensus on successful treatment outcomes (response/remission) and developing standardized, reliable measures are crucial for advancing pediatric UC drug development.
Abstract

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