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Comparative Study
. 2025 Jul 3;19(7):jjaf111.
doi: 10.1093/ecco-jcc/jjaf111.

Comparison of the FDA and EMA guidance on drug development in ulcerative colitis: an expert panel review

Sophie Vieujean  1   2 , Bruce E Sands  3 , Remo Panaccione  4 , David T Rubin  5 , Vipul Jairath  6 , Silvio Danese  7 , Laurent Peyrin-Biroulet  2 , Stefan Schreiber  8 , Séverine Vermeire  9 , Geert D'Haens  10 , Axel Dignass  11 , Parambir S Dulai  12 , Neeraj Narula  13 , Walter Reinisch  14
Affiliations
Comparative Study

Comparison of the FDA and EMA guidance on drug development in ulcerative colitis: an expert panel review

Sophie Vieujean et al. J Crohns Colitis. .

Abstract

Background and aims: The Food and Drug Administration (FDA) and European Medicines Agency (EMA) ensure the safety, efficacy, and security of treatments, including therapies for immune-mediated disorders such as inflammatory bowel disease (IBD). Their clinical trial guidelines aid sponsors in designing robust studies. While the EMA updated its guidelines for ulcerative colitis (UC) in 2018, the FDA issued new recommendations in April 2022. This paper compares these guidelines, assesses their implications for IBD clinical trials, and proposes strategies to improve alignment and trial efficiency.

Methods: A comparative analysis of the FDA's 2022 guidelines and the EMA's 2018 guidelines for UC clinical trials was conducted. Key elements reviewed include trial population criteria, study design, assessment tools, endpoints, and safety considerations. Recommendations for optimization were developed in consultation with an expert panel.

Results: The FDA's 2022 updates emphasize balanced participant representation, the use of full colonoscopy for endoscopic severity assessment, and introduce "maintenance of remission" as a new concept. Other novelties include updated statistical guidance and stricter safety requirements. While these updates enhance trial robustness, they also pose challenges for implementation.

Conclusions: Harmonizing FDA and EMA guidelines is essential to streamline global IBD clinical trials, reduce redundancies, and improve patient outcomes. Recommendations include adopting less invasive assessments, standardizing remission definitions, and prioritizing patient-centered endpoints. These measures could reduce trial complexity, increase inclusivity, and accelerate the development of effective therapies for UC.

Keywords: European Medicines Agency; Food and Drug Administration; ulcerative colitis.

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Conflict of interest statement

S.V. lecture and/or consulting fees from Abbvie, Celltrion, Ferring, Janssen, Takeda, and Lilly. B.E.S. reports personal fees and nonfinancial support from AbbVie, personal fees and nonfinancial support from Abivax, personal fees from Adiso Therapeutics, personal fees from Agomab, personal fees from Alimentiv, personal fees from Amgen, personal fees from AnaptysBio, personal fees and nonfinancial support from AstraZeneca, personal fees from Biolojic Design, personal fees from Biora Therapeutics, personal fees from Boehringer Ingelheim, grants, personal fees, and nonfinancial support from Bristol Myers Squibb, personal fees and nonfinancial support from Celltrion, personal fees from Ensho Therapeutics, personal fees from Equilium, personal fees from Enthera, personal fees from Enveda Biosciences, personal fees from Evommune, personal fees from Ferring, personal fees from Fzata, personal fees from Galapagos, personal fees from Genentech, personal fees from Gilead Sciences, personal fees from GlaxoSmithKline, personal fees from GossamerBio, personal fees from Imhotex, personal fees from Index Pharmaceuticals, personal fees from Innovation Pharmaceuticals, grants, personal fees, and nonfinancial support from Janssen, personal fees and nonfinancial support from Johnson & Johnson, personal fees from Kaleido, personal fees from Kallyope, personal fees and nonfinancial support from Lilly, personal fees and nonfinancial support from Merck, personal fees from Microba, personal fees from Microbiotica, personal fees from Mitsubishi Tanabe Pharma, personal fees from Mobius Care, personal fees from Morphic Therapeutics, personal fees from MRM Health, personal fees from Nexus Therapeutics, personal fees from Immunyx Therapeutics, personal fees from Nimbus Discovery, personal fees from Odyssey Therapeutics, personal fees from Palisade Bio, personal fees and nonfinancial support from Pfizer, personal fees from Progenity, personal fees and nonfinancial support from Prometheus Biosciences, personal fees from Prometheus Laboratories, personal fees from Protagonist Therapeutics, personal fees from Q32 Bio, personal fees from Rasayana Therapeutics, personal fees from Recludix Therapeutics, personal fees from Reistone Biotherapeutics, personal fees from Sorriso Pharmaceuticals, personal fees from Spyre Therapeutics, personal fees from Surrozen, personal fees from Target RWE, personal fees and nonfinancial support from Takeda, personal fees from Teva, personal fees from Theravance Biopharma, personal fees from TLL Pharmaceutical, personal fees from TR1X, personal fees from Union Therapeutics, personal fees and nonfinancial support from Ventyx Biosciences, outside the submitted work. R.P.: Consultant for: Abbott, AbbVie, Abbivax, Alimentiv (formerly Robarts), Amgen, AnaptysBio, Arena Pharmaceuticals, AstraZeneca, Biogen, Boehringer Ingelheim, Bristol Myers Squibb, Celgene, Celltrion, Cosmos Pharmaceuticals, Eisai, Elan, Eli Lilly, Ferring, Galapagos, Fresenius Kabi, Genentech, Gilead Sciences, GlaxoSmithKline, JAMP Bio, Janssen, Merck, Mylan, Novartis, Oppilan Pharma, Organon, Pandion Pharma, Pendopharm, Pfizer, Progenity, Prometheus Biosciences, Protagonist Therapeutics, Roche, Sandoz, Satisfai Health, Shire, Sublimity Therapeutics, Spyre Therapeutics, Takeda Pharmaceuticals, Theravance Biopharma, Trellus, Union Biopharma, Viatris, Ventyx, UCB. Speaker’s fees for: AbbVie, Amgen, Arena Pharmaceuticals, Bristol Myers Squibb, Celgene, Eli Lilly, Ferring, Fresenius Kabi, Gilead Sciences, Janssen, Merck, Organon, Pfizer, Roche, Sandoz, Shire, Takeda Pharmaceuticals. Advisory boards for: AbbVie, Alimentiv (formerly Robarts), Amgen, Arena Pharmaceuticals, AstraZeneca, Biogen, Boehringer Ingelheim, Bristol Myers Squibb, Celgene, Eli Lilly, Ferring, Fresenius Kabi, Genentech, Gilead Sciences, GlaxoSmithKline, JAMP Bio, Janssen, Merck, Mylan, Novartis, Oppilan Pharma, Organon, Pandion Pharma, Pfizer, Progenity, Protagonist Therapeutics, Roche, SandozShire, Sublimity Therapeutics, Takeda Pharmaceuticals, Ventyx. D.T.R. has received grant support from Takeda; and has served as a consultant for Abbvie, Altrubio, Abivax SA, Altrubio, Avalo Therapeutics, Bausch Health, Bristol Myers Squibb, Buhlmann Diagnostics Corp, ClostraBio, Connect BioPharma, Douglas Pharmaceuticals, Eli Lilly & Co., Genentech Inc., InDex Pharmaceuticals, Iterative Health, Janssen Pharmaceuticals, Odyssey Therapeutics, Pfizer, Sanofi, and Takeda Pharmaceuticals. He serves on the Board of Trustees for the Crohn’s & Colitis Foundation. V.J. has received consulting/advisory board fees from AbbVie, Alimentiv, Arena pharmaceuticals, Asahi Kasei Pharma, Asieris, Astra Zeneca, Avoro Capital, Bristol Myers Squibb, Celltrion, Eli Lilly, Endpoint Health, Enthera, Ferring, Flagship Pioneering, Fresenius Kabi, Galapagos, Gilde Healthcare, GlaxoSmithKline, Genentech, Gilead, Innomar, JAMP, Janssen, Merck, Metacrine, Mylan, MRM Health, Pandion, Pendopharm, Pfizer, Protagonist, Prometheus Biosciences, Reistone Biopharma, Roche, Roivant, Sandoz, Second Genome, Sorriso, Synedgen, Takeda, TD Securities, Teva, Topivert, Ventyx, Vividion; speaker’s fees from Abbvie, Ferring, Bristol Myers Squibb, Galapagos, Janssen Pfizer Shire, Takeda, and Fresenius Kabi. S.D. declares consultancy/advisory fees from AbbVie, Allergan, Amgen, AstraZeneca, Biogen, Boehringer Ingelheim, Celgene, Celltrion, Ferring Pharmaceuticals, Gilead Sciences, Hospira, Janssen, Johnson & Johnson, MSD, Mundipharma, Pfizer Inc, Roche, Sandoz, Takeda, TiGenix, UCB and Vifor; lecture/speaker fees: AbbVie, Amgen, Ferring Pharmaceuticals, Gilead Sciences, Janssen, Mylan, Pfizer Inc., Takeda; and has directorship/ownership interests in Gastroenterology and Endoscopy. L.P.-B. declares consulting fees from Abbvie, Abivax, Adacyte, Alimentiv, Amgen, Applied Molecular Transport, Arena, Banook, Biogen, BMS, Celltrion, Connect Biopharm, Cytoki Pharma, Enthera, Ferring, Fresenius Kabi, Galapagos, Genentech, Gilead, Gossamer Bio, GSK, IAC Image Analysis, Index Pharmaceuticals, Inotrem, Janssen, Lilly, Medac, Mopac, Morphic, MSD, Nordic Pharma, Novartis, Oncodesign Precision Medicine, ONO Pharma, OSE Immunotherapeuthics, Pandion Therapeuthics, Par’ Immune, Pfizer, Prometheus, Protagonist, Roche, Samsung, Sandoz, Sanofi, Satisfay, Takeda, Telavant, Theravance, Thermo Fischer, Tigenix, Tillots, Viatris, Vectivbio, Ventyx, Ysopia; receives grant from Celltrion, Fresenius Kabi, Medac, MSD, Takeda; gives lecture for Abbvie, Alfasigma, Amgen, Arena, Biogen, Celltrion, Ferring, Galapagos, Genentech, Gilead, Janssen, Kern Pharma, Lilly, Medac, MSD, Nordic Pharma, Pfizer, Sandoz, Takeda, Tillots, Viatris; received support travel from Abbvie, Alfasigma, Amgen, Celltrion, Connect Biopharm, Ferring, Galapagos, Genentech, Gilead, Gossamer Bio, Janssen, Lilly, Medac, Morphic, MSD, Pfizer, Sandoz, Takeda, Thermo Fischer, Tillots. S.S. has received consulting fees from AbbVie, Amgen, Arena, Boehringer Ingelheim, Bristol Myers Squibb, Celltrion, Falk Pharma, Ferring, Galapagos/Gilead, Genentech/Roche, GlaxoSmithKline, IMAB Biopharma, Lilly, MSD, Pfizer, Shire, and Takeda. The remaining authors disclose no conflicts. S.V. received financial support for research from: AbbVie, J&J, Pfizer, Takeda, and Galapagos and received speakers’ and/or consultancy fees from: AbbVie, Abivax, AbolerlsPharma, AgomAb, Alimentiv, Arena Pharmaceuticals, Astrazeneca, BioraTherapeutics, BMS, Boehringer Ingelheim, Celgene, Cytoki Pharma, Dr Falk Pharma, Ferring, Galapagos, Genentech Roche, Gilead, GSK, Hospira, lmidomics, Janssen, J&J, Lilly, Materia Prima, Mestag Therapeutics, Microbiotica, MiroBio, Morphic, MrMHealth, Mundipharma, MSD, Pfizer, Prodigest, Progenity, Prometheus, Robarts Clinical Trials, Surrozen, Takeda, Theravance, Tillots Pharma AG, VectivBio, Ventyx, and Zealand Pharma. G.D. has served as advisor and/or received speaker fees from Abbvie, Agomab Therapeutics, Alimentiv, AstraZeneca, Bristol Meiers Squibb, Cytoki, Celltrion, Eli Lilly, Exeliom Biosciences, Ferring, Galapagos, GlaxoSmithKline, Pfizer, Polpharm, Immunic, Index Pharmacueticals, Johnson and Johnson, Merck, ProciseDx, Roivant, Seres Health, Takeda, Tillotts, and Ventyx. A.D. reports fees for participation in clinical trials, review activities such as data monitoring boards, statistical analysis, and end point committees from Abivax, AbbVie, Arena Pharmaceuticals, Bristol Myers Squibb/Celgene, Dr Falk Foundation, Alfasigma/Galapagos, Gilead, Janssen, and Pfizer; consultancy fees from AbbVie, Alfasigma, Amgen, Arena Pharmaceuticals, Biogen, Boehringer Ingelheim, Bristol Myers Squibb/Celgene, Celltrion, Dr Falk Foundation, Ferring Pharmaceuticals, Fresenius Kabi, Galapagos, Janssen, Lilly, MSD, Pfizer, Pharmacosmos, Roche/Genentech, Sandoz/Hexal, Takeda, Tillotts, and Vifor Pharma; payment from lectures including service on speakers bureaus from AbbVie, Biogen, CED Service GmbH, Celltrion, Falk Foundation, Ferring, Galapagos, Gilead, High5MD, Janssen, Materia Prima, MedToday, MSD, Pfizer, Streamed-Up, Takeda, Tillotts, and Vifor Pharma; payment for manuscript preparation from Abbvie, Falk Foundation, Takeda, Thieme, and UniMed Verlag. P.S.D. consulting Abbvie, Abivax, Adiso, Alimentiv, Bristol Meyer Squibb, Celltrion, Genetech, Geneoscopy, Janssen, Pfizer, Takeda, and Sanofi. N.N. holds a McMaster University AFP Clinician Researcher Award. N.N. has received honoraria from Janssen, Abbvie, Takeda, Pfizer, Sandoz, Novartis, Iterative Health, Innomar Strategies, Fresinius Kabi, Amgen, Organon, Eli Lilly, and Ferring. W.R. has served as a speaker for Abbvie, Aptalis, Astellas, Celltrion, Danone Austria, Elan, Falk Pharma GmbH, Ferring, Immundiagnostik, Medice, Mitsubishi Tanabe Pharma Corporation, MSD, Otsuka, PDL, Pharmacosmos, PLS Education, Schering-Plough, Shire, Takeda, Therakos, Vifor, Yakult, as a consultant for Abbvie, Agomab, Algernon, AltruBio, Amgen, AM Pharma, AMT, AOP Orphan, Arena Pharmaceuticals, Astellas, Astra Zeneca, Avaxia, Roland Berger GmBH, Bioclinica, Biogen IDEC, Boehringer Ingelheim, Bristol Myers Squibb, Calyx, Cellerix, Chemocentryx, Celgene, Celltrion, Covance, Danone Austria, DSM, Elan, Eli Lilly, Ernest & Young, Falk Pharma GmbH, Ferring, Fresenius, Galapagos, Gatehouse Bio Inc., Genentech, Gilead, Grünenthal, ICON, Index Pharma, Inova, Intrinsic Imaging, Janssen, Johnson & Johnson, Kyowa Hakko Kirin Pharma, Landos Biopharma, Lipid Therapeutics, LivaNova, Mallinckrodt, Medahead, MedImmune, Millenium, Mitsubishi Tanabe Pharma Corporation, MSD, Nash Pharmaceuticals, Nestle, Nippon Kayaku, Novartis, Ocera, OMass, Otsuka, Parexel, PDL, Periconsulting, Pharmacosmos, Pfizer, Procter & Gamble, Prometheus, Protagonist, Provention, Quell Therapeutics, Robarts Clinical Trial, Sandoz, Schering-Plough, Second Genome, Seres Therapeutics, Setpointmedical, Sigmoid, Sublimity, Takeda, Teva Pharma, Therakos, Theravance, Tigenix, UCB, Vifor, Zealand, Zyngenia, and 4SC, as an advisory board member for Abbvie, Aesca, Amgen, AM Pharma, Astellas, Astra Zeneca, Avaxia, Biogen IDEC, Boehringer Ingelheim, Bristol Myers Squibb, Cellerix, Chemocentryx, Celgene, Celltrion, Danone Austria, DSM, Elan, Ferring, Galapagos, Genentech, Grünenthal, Inova, Janssen, Johnson & Johnson, Kyowa Hakko Kirin Pharma, Lipid Therapeutics, MedImmune, Millenium, Mitsubishi Tanabe Pharma Corporation, MSD, Nestle, Novartis, Ocera, Otsuka, PDL, Pharmacosmos, Pfizer, Procter & Gamble, Prometheus, Sandoz, Schering-Plough, Second Genome, Setpointmedical, Takeda, Therakos, Tigenix, UCB, Zealand, Zyngenia, and 4SC, and has received research funding from Abbvie, Janssen, MSD, Sandoz, and Takeda.

Figures

Figure 1.
Figure 1.
Points of agreement, divergence, and those addressed exclusively by either the FDA or EMA. Abbreviations: EMA, European Medicines Agency; FDA, Food and Drug Administration.
See this image and copyright information in PMC

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