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Challenges and opportunities for IBD drug development: from early stage to regulatory approval
  1. Silvio Danese1,2,
  2. Elmer Schabel3,
  3. Mark Andrew Ainsworth4,5,
  4. Laurent Peyrin-Biroulet6
  1. 1 Department of Gastroenterology, IBD Center, Humanitas Clinical and Research Center- IRCCS, Milan, Italy
  2. 2 Department of Biomedical Sciences, Humanitas University, Milan, Italy
  3. 3 Licensing Division 2, Federal Institute for Drugs and Medical Devices, Bonn, Germany
  4. 4 Department of Medical Evaluation and Biostatistics, Danish Medicines Agency, Copenhagen, Denmark
  5. 5 Department of Gastroenterology and Hepatology, Odense University Hospital, Odense, Denmark
  6. 6 Department of Hepato-Gastroenterology and Inserm U954, University Hospital of Nancy, Lorraine University, Vandoeuvre-lès-Nancy, France
  1. Correspondence to Dr Silvio Danese, Istituto Clinico Humanitas, Milan 20089, Italy; sdanese{at}

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Increased understanding of the immunopathology of inflammatory bowel disease (IBD) has resulted in the development of novel therapies such as vedolizumab or ustekinumab, and the investigation of new agents including Janus kinase inhibitors, anti-mucosal vascular addressin cell adhesion molecule-1 agents, anti-interleukin-12/23 monoclonal antibody and sphingosine-1-phosphate receptor-1 selective agonists.1 Over the last years, new approaches to mentoring drug research and testing have been developed. Among these methods, the fast-track drug designation and subsequent approval of safe regimens represent an emerging drug development approach in IBD treatment.2 Since 2001, the European Commission has started a fast-track approval programme for the European Medicines Agency (EMA). The Committee for Human Medicinal Products, established under the EMA, is responsible for such an accelerated review process.2 The requirements and time frames for approval of a drug under the accelerated review process are similar to those used by the United States Food and Drug Administration authorities (FDA). Eligibility for fast-track (in the USA) or accelerated approval (EMA) is based on whether a product will be of major public health interest, particularly from the point of view of therapeutic innovation3 and on endpoints that predict substantial clinical improved outcomes.2

In IBD, this can be difficult to define and the gold standard would probably be complete mucosal or histological healing.2 The cost of IBD care is rising worldwide as IBD incidence and prevalence are rapidly increasing.4 One key reason for the rising cost of IBD treatment is the significant costs of new therapeutic molecules brought into the market, particularly biologics.2 4 Indeed, evidence suggests that the cost of biologic agents now accounts for the main expenditure in treating patients with IBD.5 6 To reduce the cost of IBD drugs, efforts must be made to optimise the drug development process and …

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  • Correction notice This article has been corrected since it published Online First. The affiliations for the first author have been updated.

  • Contributors SD and LP-B planned and wrote the first draft of the paper. ES and MAA critically reviewed the paper. SD and LP-B are guarantors of the article.

  • Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

  • Disclaimer ES and MAA: the views expressed in this article are primarily those of the authors and do not represent those of the Federal Institute for Drugs and Medical Devices, the Danish Medicines Agency or the European Medicines Agency

  • Competing interests SD has served as a speaker, consultant and advisory board member for Schering-Plough, Abbott (AbbVie) Laboratories, Merck and Co, UCB Pharma, Ferring, Cellerix, Millenium Takeda, Nycomed, Pharmacosmos, Actelion, α Wasserman, Genentech, Grunenthal, Pfizer, AstraZeneca, Novo Nordisk, Cosmo Pharmaceuticals, Vifor and Johnson and Johnson. LP-B has received consulting fees from Merck, AbbVie, Janssen, Genentech, Mitsubishi, Ferring, Norgine, Tillots, Vifor, Therakos, Pharmacosmos, Pilège, BMS, UCB Pharma, Hospira, Celltrion, Takeda, Biogaran, Boerhinger Ingelheim, Lilly, Pfizer, HAC-Pharma, Index Pharmaceuticals, Amgen and Sandoz; and lecture fees from Merck, AbbVie, Takeda, Janssen, Takeda, Ferring, Norgine, Tillots, Vifor, Therakos, Mitsubishi and HAC-Pharma.

  • Patient consent for publication Not required.

  • Provenance and peer review Not commissioned; externally peer reviewed.

  • Data availability statement There are no data in this work.