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Unmet need in rheumatology: reports from the Targeted Therapies meeting 2019
  1. Kevin L Winthrop1,
  2. Michael E Weinblatt2,
  3. Joan Bathon3,
  4. Gerd R Burmester4,
  5. Philip J Mease5,
  6. Leslie Crofford6,
  7. Vivian Bykerk7,
  8. Maxime Dougados8,
  9. James Todd Rosenbaum1,9,
  10. Xavier Mariette10,
  11. Joachim Sieper11,
  12. Fritz Melchers12,
  13. Bruce N Cronstein13,
  14. Ferry C Breedveld14,
  15. Joachim Kalden15,
  16. Josef S Smolen16,
  17. Daniel Furst5,17,18
  1. 1 Oregon Health Sciences University, Portland, Oregon, USA
  2. 2 Brigham and Womens Hospital, Boston, Massachusetts, USA
  3. 3 Columbia University, College of Physicians & Surgeons, New York City, New York, USA
  4. 4 Charité University Hospital, Berlin, Germany
  5. 5 Swedish Medical Center, University of Washington, Seattle, Washington, USA
  6. 6 Vanderbilt University, Nashville, Tennessee, USA
  7. 7 Hospital for Special Surgery, New York City, New York, USA
  8. 8 Hôpital Cochin, Paris, France
  9. 9 Legacy Devers Eye Institute, Portland, Oregon, USA
  10. 10 Paris-Sud University, APHP Université Paris-Saclay, Hôpital Bicêtre, Le Kremlin Bicêtre, France
  11. 11 Department of Gastroenterology, Infectious Diseases and Rheumatology, Campus Benjamin Franklin, Charité, Berlin, Germany
  12. 12 Max Planck Institute for Infection Biology, Berlin, Germany
  13. 13 NYU School of Medicine, New York City, New York, USA
  14. 14 Leiden University Medical Center, Leiden, The Netherlands
  15. 15 University of Erlangen, Erlangen, Germany
  16. 16 Division of Rheumatology, Department of Medicine 3, Medical University of, Vienna, Vienna, Austria
  17. 17 University of California, Los Angeles Medical Center, Los Angeles, CA, USA
  18. 18 University of Florence, Florence, Italy
  1. Correspondence to Dr Kevin L Winthrop, Oregon Health Sciences University, Portland, OR 97239, USA; winthrop{at}ohsu.edu

Abstract

Objectives To detail the greatest areas of unmet scientific and clinical needs in rheumatology.

Methods The 21st annual international Advances in Targeted Therapies meeting brought together more than 100 leading basic scientists and clinical researchers in rheumatology, immunology, epidemiology, molecular biology and other specialties. During the meeting, breakout sessions were convened, consisting of 5 disease-specific groups with 20–30 experts assigned to each group based on expertise. Specific groups included: rheumatoid arthritis, psoriatic arthritis, axial spondyloarthritis, systemic lupus erythematosus and other systemic autoimmune rheumatic diseases. In each group, experts were asked to identify unmet clinical and translational research needs in general and then to prioritise and detail the most important specific needs within each disease area.

Results Overarching themes across all disease states included the need to innovate clinical trial design with emphasis on studying patients with refractory disease, the development of trials that take into account disease endotypes and patients with overlapping inflammatory diseases, the need to better understand the prevalence and incidence of inflammatory diseases in developing regions of the world and ultimately to develop therapies that can cure inflammatory autoimmune diseases.

Conclusions Unmet needs for new therapies and trial designs, particularly for those with treatment refractory disease, remain a top priority in rheumatology.

  • rheumatoid arthritis
  • psoriatic arthritis
  • ankylosing spondylitis
  • spondyloarthritis
  • systemic lupus erythematosus
  • systemic sclerosis
  • Sjögren’s syndrome
  • inflammatory myopathies
  • vasculitis

This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, appropriate credit is given, any changes made indicated, and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/.

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Footnotes

  • Contributors All coauthors contributed to this manuscript’s creation in all aspects including data gathering, analysis, writing and critical revision of the manuscript.

  • 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.

  • Competing interests KLW reports personal fees from Pfizer, grants and personal fees from BMS, personal fees from AbbVie, personal fees from UCB, personal fees from Lilly, personal fees from Galapagos, personal fees from GSK, personal fees from Roche outside the submitted work. JB reports personal fees from AbbVie outside the submitted work. MD reports grants and personal fees from Pfizer, grants and personal fees from Abbvie, grants and personal fees from Lilly, grants and personal fees from Novartis, grants and personal fees from UCB, grants and personal fees from Merck, grants and personal fees from Roche, during the conduct of the study. JTR reports grants from Pfizer, personal fees from Abbvie, personal fees from Gilead, personal fees from Santen, personal fees from Roche, personal fees from Novartis, personal fees from UCB, personal fees from Corvus, personal fees from Horizon, personal fees from Celldex, personal fees from Eyevensys, personal fees from UpToDate, personal fees from Janssen outside the submitted work. XM reports personal fees from BMS, personal fees from GILEAD, personal fees from PFIZER, personal fees from SAMSUNG, personal fees from UCB outside the submitted work. BNC has multiple patents, none of which are relevant. In addition he has multiple grants from NIH. JSS reports grants and personal fees from AbbVie, personal fees from Amgen, personal fees from AstraZeneca, personal fees from Astro, personal fees from Bristol-Myers Squibb, personal fees from Celgene, personal fees from Celltrion, personal fees from ILTOO, grants and personal fees from Janssen, grants and personal fees from Lilly, personal fees from MSD, grants and personal fees from Novartis-Sandoz, personal fees from Novo-Nordisk, grants and personal fees from Roche, personal fees from Samsung Bioepis, personal fees from Sanofi, personal fees from UCB, grants and personal fees from Pfizer outside the submitted work. DF reports grant/research support from Actelion, grant/research support from Amgen, grant/research support from BMS, grant/research support from Corbus, grant/research support from Galapagos GSK, grant/research support from NIH, grant/research support from Novartis, grant/research support from Pfizer, grant/research support from Sanofi, grant/research support from Roche/Genentech, personal fees from Actelion, personal fees from Amgen, personal fees from BMS, personal fees from Corbus, personal fees from Galapagos, personal fees from Novartis, personal fees from Pfizer outside the submitted work.

  • Patient consent for publication Not required.

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

  • Data availability statement All data relevant to the study are included in the article or uploaded as supplementary information.