EULAR points to consider when analysing and reporting comparative effectiveness research using observational data in rheumatology.

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.

Informations de copyright

© Author(s) (or their employer(s)) 2022. No commercial re-use. See rights and permissions. Published by BMJ.

Déclaration de conflit d'intérêts

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.