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EULAR points to consider when analysing and reporting comparative effectiveness research using observational data in rheumatology
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  1. Delphine Sophie Courvoisier1,
  2. Kim Lauper1,2,
  3. Joanna Kedra3,4,
  4. Maarten de Wit5,
  5. Bruno Fautrel6,7,
  6. Thomas Frisell8,
  7. Kimme L Hyrich2,
  8. Florenzo Iannone9,
  9. Pedro M Machado10,11,12,
  10. Lykke Midtbøll Ørnbjerg13,
  11. Ziga Rotar14,15,
  12. Maria Jose Santos16,17,
  13. Tanja A Stamm18,19,
  14. Simon R Stones20,
  15. Anja Strangfeld21,
  16. Sytske Anne Bergstra22,
  17. Robert B M Landewé23,24,
  18. Axel Finckh1
  1. 1 Division of Rheumatology, University of Geneva, Geneva, Switzerland
  2. 2 Centre for Musculoskeletal Research, University of Manchester, Manchester, UK
  3. 3 Institut Pierre Louis d'Epidémiologie et de Santé Publique (iPLESP), UMR S 1136, Sorbonne Universite, Paris, France
  4. 4 APHP, Rheumatology Department, Hopital Universitaire Pitie Salpetriere, Paris, France
  5. 5 EULAR Patient Research Partner, Amsterdam, Netherlands
  6. 6 Rheumatology, Assistance Publique - Hopitaux de Paris, Paris, France
  7. 7 GRC08 - IPLESP, UPMC Faculte de Medecine, Paris, France
  8. 8 Clinical Epidemiology Unit, Karolinska Institutet, Stockholm, Sweden
  9. 9 School of Medicine, University of Bari, Bari, Italy
  10. 10 Centre for Rheumatology & Department of Neuromuscular Diseases, University College London, London, UK
  11. 11 Department of Rheumatology, Northwick Park Hospital, London North West University Healthcare NHS Trust, London, UK
  12. 12 National Institute for Health Research (NIHR) University College London Hospitals Biomedical Research Centre, University College London Hospitals NHS Foundation Trust, London, UK
  13. 13 Copenhagen Center for Arthritis Research, Center for Rheumatology and Spine Diseases, Rigshospitalet Glostrup, Glostrup, Denmark
  14. 14 Department of Rheumatology, University Medical Centre, Ljubljana, Slovenia
  15. 15 Faculty of Medicine, University of Ljubljana, Ljubljana, Slovenia
  16. 16 Rheumatology, Hospital Garcia de Orta EPE, Almada, Portugal
  17. 17 Rheumatology Research Unit, Instituto de Medicina Molecular, Lisboa, Portugal
  18. 18 Section for Outcomes Research, Medical University of Vienna, 1090, Austria
  19. 19 Ludwig Boltzmann Institute for Arthritis and Rehabilitation, Vienna, Austria
  20. 20 EULAR Patient Research Partner, Manchester, UK
  21. 21 Forschungsbereich Epidemiologie, Deutsches Rheuma-Forschungszentrum Berlin, Berlin, Germany
  22. 22 Rheumatology, Leiden University Medical Center, Leiden, Netherlands
  23. 23 Amsterdam Rheumatology Center, AMC, Amsterdam, Netherlands
  24. 24 Rheumatology, Zuyderland MC, Heerlen, Netherlands
  1. Correspondence to Dr Delphine Sophie Courvoisier, University of Geneva, Geneva, Switzerland; delphine.courvoisier{at}hcuge.ch

Abstract

Background Comparing treatment effectiveness over time in observational settings is hampered by several major threats, among them confounding and attrition bias.

Objectives To develop European Alliance of Associations for Rheumatology (EULAR) points to consider (PtC) when analysing and reporting comparative effectiveness research using observational data in rheumatology.

Methods The PtC were developed using a three-step process according to the EULAR Standard Operating Procedures. Based on a systematic review of methods currently used in comparative effectiveness studies, the PtC were formulated through two in-person meetings of a multidisciplinary task force and a two-round online Delphi, using expert opinion and a simulation study. Finally, feedback from a larger audience was used to refine the PtC. Mean levels of agreement among the task force were calculated.

Results Three overarching principles and 10 PtC were formulated, addressing, in particular, potential biases relating to attrition or confounding by indication. Building on Strengthening the Reporting of Observational Studies in Epidemiology guidelines, these PtC insist on the definition of the baseline for analysis and treatment effectiveness. They also focus on the reasons for stopping treatment as an important consideration when assessing effectiveness. Finally, the PtC recommend providing key information on missingness patterns.

Conclusion To improve the reliability of an increasing number of real-world comparative effectiveness studies in rheumatology, special attention is required to reduce potential biases. Adherence to clear recommendations for the analysis and reporting of observational comparative effectiveness studies will improve the trustworthiness of their results.

  • epidemiology
  • patient reported outcome measures
  • outcome assessment
  • health care

https://doi.org/10.1136/annrheumdis-2021-221307

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    Footnotes

    • Handling editor Dimitrios T Boumpas

    • Twitter @delcourvoisier, @pedrommcmachado

    • Contributors All authors were members of the taskforce and made substantial contributions to the developement and interpretation of the points to consider. They also contributed to revising the manuscript critically for important intellectual content. All authors approved the final version to be published, and agree to be accountable for all aspects of the work.

    • Competing interests DSC has received consulting fees from Abbvie, MSD, and Pfizer outside submitted work. KL has received speaker fees from Gilead-Galapagos and grant/research support from AbbVie outside submitted work. ZR has received consulting and speaker fees from Abbvie, Eli Lilly, Novartis, MSD, Pfizer, Roche, Sandoz outside submitted work. PMM has received consulting/speaker’s fees from Abbvie, BMS, Celgene, Eli Lilly, Janssen, MSD, Novartis, Orphazyme, Pfizer, Roche and UCB, unrelated to the work presented in this manuscript, and is supported by the National Institute for Health Research (NIHR), University College London Hospitals (UCLH), Biomedical Research Centre (BRC). Disclaimer: The views expressed here are those of the authors and do not necessarily represent the views of the (UK) National Health Service (NHS), the National Institute for Health Research (NIHR), or the (UK) Department of Health, or any other organisation. FI has received consulting/speaker’s fees from Abbvie, BMS, Celgene, Eli Lilly, Galapagos, Janssen, MSD, Novartis, Pfizer, SOBI, Roche and UCB, unrelated to the work presented in this manuscript. TAS has received grant/research support from AbbVie and Roche, has been a consultant for AbbVie and Sanofi Genzyme, and has been a paid speaker for AbbVie, Roche, Sanofi and Takeda. AF has received grant/research support from AbbVie, BMS, Eli-Lilly, Galapagos, and Pfizer, has been a paid speaker for AbbVie, BMS, Eli-Lilly, Novartis, on Novartis. MdW operating for Stichting Tools has received fees for lectures or consultancy provided by MdW from Celgene, Eli Lilly, Pfizer and UCB, over the last three years, unrelated to the work presented in this manuscript. AS has received speaker’s fees from AbbVie, BMS, Celltrion, MSD, Pfizer, and Roche, unrelated to the work presented in this manuscript. SRS has received consulting/speaker’s fees from 67 Health, Ampersand Health, Envision Pharma Group, Janssen and On The Pulse Consultancy, and is an employee of Envision Pharma Group, unrelated to the work presented in this manuscript. LMØ has received grant/research support from Novartis, unrelated to the work presented in this manuscript.

    • Patient and public involvement Patients and/or the public were involved in the design, or conduct, or reporting, or dissemination plans of this research. Refer to the Methods section for further details.

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