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Developing guidelines for ultrarare rheumatic disorders: a bumpy ride
  1. David Piskin1,2,3,
  2. Micol Romano3,4,
  3. Daniel Aletaha5,
  4. Brian M Feldman6,
  5. Raphaela Goldbach-Mansky7,
  6. Loreto Carmona8,
  7. Erkan Demirkaya2,3,4
  1. 1 Lawson Health Research Institute, London, Ontario, Canada
  2. 2 Department of Epidemiology and Biostatistics, Western University, London, Ontario, Canada
  3. 3 Canadian Behcet and Autoinflammatory Disease Center (CAN-BE-AID), Western University, London, Ontario, Canada
  4. 4 Division of Pediatric Rheumatology, Department of Pediatrics, Schulich School of Medicine & Dentistry, Western University, London, Ontario, Canada
  5. 5 Division of Rheumatology, Department of Internal Medicine III, Medical University of Vienna, Vienna, Austria
  6. 6 Division of Rheumatology, Hospital for Sick Children, Toronto, Ontario, Canada
  7. 7 National Institute of Allergy and Infectious Diseases, Bethesda, Maryland, USA
  8. 8 Instituto de Salud Musculoesquelética (INMUSC), Madrid, Spain
  1. Correspondence to Dr David Piskin, Lawson Health Research Institute, London, ON N6C 2R5, Canada; david.piskin{at}lhsc.on.ca

Abstract

Clinical practice guidelines are useful tools for both patients and physicians. Several standardised operating procedures are in existence to describe tasks step by step to develop guidelines/recommendations. The end product consists of data synthesis from the systematic literature search and patient/physician’s inputs. For the prevalent diseases, the process for developing guidelines is straightforward; it is based on physicians’/patients’ experiences and abundance of the literature. When it comes to the realm of ultrarare diseases, there are few physicians who are familiar with a disease, and there is a scarcity of literature. In this viewpoint, we describe challenges from the methodological perspectives that occurred during the process of developing recommendations for autoinflammatory disorders with the goal of finding solutions that facilitate the development of guidelines for ultrarare diseases in the future.

  • Interleukin 1 Receptor Antagonist Protein
  • Cryopyrin-Associated Periodic Syndromes
  • Epidemiology

https://doi.org/10.1136/ard-2022-222538

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    Footnotes

    • Handling editor Gerd-Rüdiger R Burmester

    • Twitter @carmona_loreto

    • DP and MR contributed equally.

    • Contributors All authors contributed to this viewpoint conception and design. The first draft of the manuscript was written by DP and MR. All the authors commented on previous versions of the manuscript and read and approved the final 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 None declared.

    • Patient and public involvement Patients and/or the public were not involved in the design, conduct, reporting or dissemination plans of this research.

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