Walter Reinisch1, Klaus Gottlieb2, Jean-Frederic Colombel3, Silvio Danese4, Remo Panaccione5, Julian Panes6, Laurent Peyrin-Biroulet7, David Rubin8, Bruce E Sands3, Stefan Schreiber9, Severine Vermeire10, Andrew Mulberg11, Bill Sandborn12. 1. Division of Gastroenterology and Hepatology, Department of Internal Medicine III, Medical University of Vienna, Vienna, Austria. Electronic address: walter.reinisch@meduniwien.ac.at. 2. Eli Lilly Research Laboratories, Indianapolis, Indiana. 3. Dr Henry D. Janowitz Division of Gastroenterology, Icahn School of Medicine at Mount Sinai, New York, New York. 4. Inflammatory Bowel Disease Center, Humanitas University, Milano, Italy. 5. Division of Gastroenterology and Hepatology, University of Calgary, Calgary, Canada. 6. Department of Gastroenterology, Faculty of Medicine, University of Barcelona, Barcelona, Spain. 7. Inserm U954, Department of Gastroenterology, Nancy University Hospital, Lorraine University, Nancy, France. 8. Department of Gastroenterology, Hepatology and Nutrition, University of Chicago Medicine, Chicago, Illinois. 9. Klinik für Innere Medizin I, Christian-Albrechts-Universität Kiel, Kiel, Germany. 10. Department of Clinical and Experimental Medicine, KU Leuven, Leuven, Belgium. 11. Amicus Therapeutics, Cranbury, New Jersey. 12. University of California San Diego, La Jolla, California.
Abstract
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 We sought to compare and contrast the EMA draft guideline with the FDA draft guidance to facilitate further discussion and perhaps harmonization between the 2 guidelines when they are finalized.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.
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.
Authors: Christopher Ma; William J Sandborn; Geert R D'Haens; Guangyong Zou; Larry W Stitt; Siddharth Singh; Ashwin N Ananthakrishnan; Parambir S Dulai; Reena Khanna; Vipul Jairath; Brian G Feagan Journal: Clin Gastroenterol Hepatol Date: 2019-09-20 Impact factor: 11.382
Authors: Klaus Gottlieb; Marco Daperno; Keith Usiskin; Bruce E Sands; Harris Ahmad; Colin W Howden; William Karnes; Young S Oh; Irene Modesto; Colleen Marano; Ryan William Stidham; Walter Reinisch Journal: Gut Date: 2020-07-22 Impact factor: 23.059