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Can we improve the performance and reporting of investigator-initiated clinical trials? Rheumatoid arthritis as an example
  1. Robert B M Landewé1,2,
  2. Josef S Smolen3,
  3. Michael E Weinblatt4,
  4. Paul Emery5,6,
  5. Maxime Dougados7,
  6. Roy Fleischmann8,
  7. Daniel Aletaha9,
  8. Arthur Kavanaugh10,
  9. Désirée van der Heijde11
  1. 1Amsterdam Rheumatology & immunology Center, Academic Medical Center/University of Amsterdam, The Netherlands
  2. 2Department of Rheumatology, Atrium Medical Center Heerlen, The Netherlands
  3. 3Division of Rheumatology, Department of Medicine Three, Medical University Vienna & Second Department of Medicine, Hietzing Hospital, Vienna, Austria
  4. 4Division of Rheumatology, Immunology and Allergy,
    Brigham and Women's Hospital
    Harvard Medical School
    , Boston, USA
  5. 5Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, Chapel Allerton Hospital, Leeds, UK
  6. 6NIHR Leeds Musculoskeletal Biomedical Research Unit, Leeds Teaching Hospitals NHS Trust, Leeds, UK
  7. 7Rheumatology Department, Paris Descartes University, Cochin Hospital, Assistance Publique-Hôpitaux de Paris, INSERM (U1153): Clinical Epidemiology and Biostatistics, PRES Sorbonne Paris-Cité, Paris, Fance
  8. 8Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, USA
  9. 9Division of Rheumatology, Department of Medicine Three, Medical University Vienna, Austria
  10. 10Rheumatology, Allergy, Immunology Division, University of California, San Diego, USA
  11. 11Department of rheumatology, Leiden University Medical Center, Leiden, The Netherlands
  1. Correspondence to Professor Robert B M Landewé, Department of Rheumatology & Clinical Immunology, Academic Medical Center, Meibergdreef 9, Amsterdam 1100 DD, The Netherlands; Landewe{at}rlandewe.nl

Abstract

Investigator-initiated trials, some of which have been referred to as comparative effectiveness trials, pragmatic trials, or strategy trials, are sometimes considered to be of greater clinical importance than industry-driven trials, because they address important but unresolved clinical questions that differ from the questions asked in industry-driven trials. Regulatory authorities have provided methodological guidance for industry-driven trials for the approval of new treatments, but such guidance is less clear for investigator-initiated trials. The European League Against Rheumatism (EULAR) task force for the update of the recommendations for the management of rheumatoid arthritis has critically looked at the methodological quality and conduct of many investigator-initiated trials, and has identified a number of concerns. In this Viewpoint paper, we highlight commonly encountered issues that are discussed using examples of well-known investigator-initiated trials. These issues cover three themes: (1) design choice (superiority vs non-inferiority designs); (2) statistical power and (3) convenience reporting. Since we acknowledge the importance of investigator-initiated research, we also propose a shortlist of points-to-consider when designing, performing and reporting investigator-initiated trials.

  • Treatment
  • Rheumatoid Arthritis
  • Anti-TNF

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