Comparison of the EMA and FDA Guidelines on Ulcerative Colitis Drug Development

Clin Gastroenterol Hepatol. 2019 Aug;17(9):1673-1679.e1. doi: 10.1016/j.cgh.2018.10.032. Epub 2018 Oct 26.


Background & aims: In 2016, the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) released revised EMA and new FDA draft guidelines related to the development of drugs intended for the treatment of ulcerative colitis. We sought to compare and contrast the EMA draft guideline with the FDA draft guidance to facilitate further discussion and perhaps harmonization between the 2 guidelines when they are finalized.

Methods: A concordance document was created by arranging like or similar topics addressed by the guidelines side by side in a tabular format. This concordance table served as a source for writing the narrative. The first draft was subjected to repeated rounds of reviews and revisions by the authors and outside reviewers, all of them familiar with the subject matter from a regulatory science and/or academic perspective.

Results: The FDA guidance focuses on end points, whereas the EMA guideline additionally supplies much useful information for trial design. FDA guidance appears more aspirational, suggesting the development of entirely new patient-reported outcome instruments and the incorporation of a not-yet-validated histology instrument into the definition of mucosal healing.

Conclusions: The guidelines by the FDA and the EMA complement each other and together are aimed to further practical drug development toward more clinically relevant end points in ulcerative colitis. Efforts are needed to harmonize the documents.

Keywords: Drug Development; EMA; FDA; Guidelines; Inflammatory Bowel Disease; Ulcerative Colitis.

Publication types

  • Comparative Study
  • Review

MeSH terms

  • Clinical Trials as Topic
  • Colitis, Ulcerative / drug therapy*
  • Colitis, Ulcerative / pathology
  • Colitis, Ulcerative / physiopathology
  • Drug Development*
  • European Union*
  • Government Agencies*
  • Guidelines as Topic*
  • Humans
  • Intestinal Mucosa / pathology
  • Patient Reported Outcome Measures
  • Treatment Outcome
  • United States
  • United States Food and Drug Administration*