Expediting drug development for pediatric inflammatory bowel disease

A discussion with stakeholders

Andrew E. Mulberg, Athos Bousvaros, Richard Colletti, Jeffrey Hyams, Kerry Jo Lee, Joachim Musaus, Anne M. Robinson, Joel Rosh, Shehzad Saeed, Perdita Taylor-Zapata, Eric Zuckerman, Harland S. Winter

Research output: Contribution to journalArticle

Abstract

Lack of scientific consensus on efficacy endpoints and outcome assessments presents a hurdle for global drug development in pediatric inflammatory bowel disease (IBD). Multiple stakeholders participated in a meeting November 2015, sponsored by The Pediatric IBD Foundation, which is a 501c3 organization formed by parents of children with IBDwhose mission is to improve the lives of children with Crohn disease and ulcerative colitis by supporting innovative research and educational programs (www.pedsibd.org). With representatives of the Food and Drug Administration, European Medicines Agency, pediatric gastroenterologists, and representatives of the pharmaceutical industry, this meeting was organized to harmonize present thinking about various aspects of global drug development in pediatric IBD. The meetingwas designed to be interactive, allowing participants from the pharmaceutical industry, regulatory agencies, academia, and clinical practice an opportunity to collaborate in a public forum and to identify potential strategies to expedite drug evaluation in children. Before the meeting, a questionnaire focused on the hurdles hindering approval of medications used to treat children with IBD was sent to all participants and other pediatric gastroenterologists in North America and Europe with expertise in IBD. Responses were reviewed by the steering committee and results presented at the meeting. Following the presentation of the survey, participants were divided into small groups composed of representatives from academia, industry, regulatory agencies, and members of the Pediatric IBD Foundation and assigned the task of working together to find solutions to the hurdles that had been identified. Hurdles hindering approval included pediatric trials start later in the development process; lack of enrollment in pediatric trials; lack of monitoring safety registries that might expedite approval; different priorities among stakeholders. This 1-day meeting discussed how to expedite pediatric drug development in IBD therapy. Hurdles for achieving approvals of pediatric indications for treatments of IBD were identified.

Original languageEnglish (US)
Pages (from-to)95-99
Number of pages5
JournalJournal of pediatric gastroenterology and nutrition
Volume66
Issue number1
DOIs
StatePublished - Jan 1 2018
Externally publishedYes

Fingerprint

Inflammatory Bowel Diseases
Pediatrics
Pharmaceutical Preparations
Drug Industry
Drug Evaluation
United States Food and Drug Administration
North America
Ulcerative Colitis
Crohn Disease
Registries
Consensus
Industry
Parents
Outcome Assessment (Health Care)
Organizations
Safety
Therapeutics

All Science Journal Classification (ASJC) codes

  • Pediatrics, Perinatology, and Child Health
  • Gastroenterology

Cite this

Mulberg, Andrew E. ; Bousvaros, Athos ; Colletti, Richard ; Hyams, Jeffrey ; Lee, Kerry Jo ; Musaus, Joachim ; Robinson, Anne M. ; Rosh, Joel ; Saeed, Shehzad ; Taylor-Zapata, Perdita ; Zuckerman, Eric ; Winter, Harland S. / Expediting drug development for pediatric inflammatory bowel disease : A discussion with stakeholders. In: Journal of pediatric gastroenterology and nutrition. 2018 ; Vol. 66, No. 1. pp. 95-99.
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Mulberg, AE, Bousvaros, A, Colletti, R, Hyams, J, Lee, KJ, Musaus, J, Robinson, AM, Rosh, J, Saeed, S, Taylor-Zapata, P, Zuckerman, E & Winter, HS 2018, 'Expediting drug development for pediatric inflammatory bowel disease: A discussion with stakeholders', Journal of pediatric gastroenterology and nutrition, vol. 66, no. 1, pp. 95-99. https://doi.org/10.1097/MPG.0000000000001824

Expediting drug development for pediatric inflammatory bowel disease : A discussion with stakeholders. / Mulberg, Andrew E.; Bousvaros, Athos; Colletti, Richard; Hyams, Jeffrey; Lee, Kerry Jo; Musaus, Joachim; Robinson, Anne M.; Rosh, Joel; Saeed, Shehzad; Taylor-Zapata, Perdita; Zuckerman, Eric; Winter, Harland S.

In: Journal of pediatric gastroenterology and nutrition, Vol. 66, No. 1, 01.01.2018, p. 95-99.

Research output: Contribution to journalArticle

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