Article Text
Abstract
Background The rheumatoid arthritis (RA) treatment landscape is diverse, with multiple therapies available that differ in several attributes such as mode of administration and benefit-risk profile. Patients and prescribers face challenging trade-offs during treatment selection to accommodate patients’ circumstances in order to ensure comprehensive disease management. EULAR recommendations for RA management emphasize the need to recognize patient preferences in shared decision-making (SDM). Therefore, it is essential to understand how preferences differ in the RA patient population.
Objectives This study elicited trade-offs that RA patients were willing to make during treatment selection while accounting for preference heterogeneity.
Methods An online discrete choice experiment (DCE) was conducted from September to October 2021 in which RA patients were required to elicit their preferences for attributes of treatments for RA (Figure 1) and make trade-offs between them. Attributes were selected and defined based on literature review and qualitative patient interviews, and were then tested in a quantitative pilot. Main data collection consisted of an online survey in which participants were asked to repeatedly choose between hypothetical treatments. Eligible patients were ≥18 years old, diagnosed with RA, currently received systemic disease-modifying anti-rheumatic drug therapy for RA, and were residents of France, Germany, Italy, Spain, United Kingdom, or United States. Male patients were oversampled to support subgroup analysis of preferences for effects on sperm parameters. Data were analyzed using a correlated mixed logit model and differences in preferences between sex and age were explored. Relative attribute importance (RAI) scores and maximum acceptable risk (MAR) measures were derived from the estimates.
Results A total of 2,090 patients participated; 42% were female with predefined oversampling of male patients, with a mean age of 45.2 years (range 18–83). Estimated effects were significant for all attributes (p<0.001), implying that they all influenced treatment choices and suggesting preferences differed between participants. Average RAI scores revealed different priorities (p<0.001) between males and females (Figure 1). While reducing pain and negative effect on semen parameters was most important to male patients, female patients were most concerned by risk of blood clots and serious infections. The remaining attributes were of lower importance but were still relevant. However, no single attribute explained treatment preferences by more than 30%. Preferences were also affected by patients’ age: patients aged 18-44 years placed less importance on frequency and mode of treatment administration (p<0.05) than older age groups. Patients were willing to make benefit-risk trade-offs; they accepted extra risks of blood clots (male: 1.8%; female: 0.8%), serious infections (male: 2.5%; female: 1.0%), or negative effects on sperm (male: 7.4%) for an oral pill every day instead of injection once a week. They also accepted extra risks of blood clots (male: 2.3%; female: 1.2%), serious infections (male: 3.2%; female: 1.6%), or negative effects on sperm (male: 10.4%) for reducing amount of pain from 30% to 10%. Similar observations were made for improved performance of daily activities. However, acceptable trade-offs varied between patients (p<0.05).
Conclusion Preferences of RA patients were driven by benefits and risks of RA treatments, with no single attribute dominating the decision making. Patients were willing to accept higher risk of serious infections and blood clots in exchange for improvements in pain, daily activities, or administration convenience. These findings emphasize the importance of considering the entire treatment profile, including benefits, risks, and administration to support SDM between providers and patients.
Preference drivers: males – pain, blood clots; females – blood clots, infections, pain. Sperm risk data are based on male responses only.
Acknowledgements This study was funded by Galapagos NV (Mechelen, Belgium). Publication coordination was provided by Fabien Debailleul, PhD, of Galapagos NV. Medical writing support was provided by Brooke Middlebrook (Evidera) and publications management was provided by Aspire Scientific Ltd (Bollington, UK), funded by Galapagos NV.
Disclosure of Interests Rieke Alten Consultant of: AbbVie, Amgen, Biogen, BMS, Celltrion, Gilead, Janssen, Lilly, Medac, MSD, Mylan, Novartis, Pfizer, Roche, Sandoz, Sanofi-Genzyme, UCB, Viatris, Juan Carlos Nieto González Speakers bureau: AbbVie, Amgen, Biogen, BMS, Celgene, FAES Farma, Gebro, Janssen, Lilly, MSD, Nordic Pharma, Novartis, Pfizer, Roche, Sandoz, Sanofi, UCB Pharma, Consultant of: AbbVie, Amgen, GSK, Galapagos, Janssen, Lilly, MSD, Peggy Jacques: None declared, Carlomaurizio Montecucco Speakers bureau: AbbVie, BMS, Boehringer, Eli Lilly, Galapagos, Pfizer, Roche, Sanofi, Consultant of: AbbVie, BMS, Gilead, Robert Moots Speakers bureau: Amgen, Galapagos, Consultant of: Ferring, Grant/research support from: Novartis, Helga Radner Speakers bureau: Gilead Sciences, Janssen, MSD, Pfizer Corporation Austria, Sebastian Heidenreich Employee of: Evidera Inc, which is part of Thermo Fisher Scientific’s Clinical Research Group. Evidera received payment for conducting the work outlined in this work., Chiara Whichello Employee of: Evidera Inc, which is part of Thermo Fisher Scientific’s Clinical Research Group. Evidera received payment for conducting the work outlined in this work., Nicolas Krucien Employee of: Evidera Inc, which is part of Thermo Fisher Scientific’s Clinical Research Group. Evidera received payment for conducting the work outlined in this work., Monia Zignani Shareholder of: Galapagos, Employee of: Galapagos, Harald Vonkeman Speakers bureau: AbbVie, Boehringer Ingelheim, Galapagos, Janssen, Novartis, Pfizer, UCB, Grant/research support from: AbbVie, Boehringer Ingelheim, Galapagos, Janssen, Novartis, Pfizer, UCB, Katrien Van Beneden Shareholder of: Galapagos, Employee of: Galapagos.
- Rheumatoid arthritis
- Disease-modifying drugs (DMARDs)