Preferences for preventive treatments for rheumatoid arthritis: discrete choice survey in the UK, Germany and Romania

Abstract

Objective To quantify preferences for preventive therapies for rheumatoid arthritis (RA) across three countries. Methods A web-based survey including a discrete choice experiment was administered to adults recruited via survey panels in the UK, Germany and Romania. Participants were asked to assume they were experiencing arthralgia and had a 60% chance of developing RA in the next 2 years and completed 15 choices between no treatment and two hypothetical preventive treatments. Treatments were defined by six attributes (effectiveness, risks and frequency/route of administration) with varying levels. Participants also completed a choice task with fixed profiles reflecting subjective estimates of candidate preventive treatments. Latent class models (LCMs) were conducted and the relative importance of attributes, benefit–risk trade-offs and predicted treatment uptake was subsequently calculated. Results Completed surveys from 2959 participants were included in the analysis. Most participants preferred treatment over no treatment and valued treatment effectiveness to reduce risk more than other attributes. A five-class LCM best fitted the data. Country, perceived risk of RA, health literacy and numeracy predicted class membership probability. Overall, the maximum acceptable risk for a 40% reduction in the chance of getting RA (60% to 20%) was 21.7%, 19.1% and 2.2% for mild side effects, serious infection and serious side effects, respectively. Predicted uptake of profiles reflecting candidate prevention therapies differed across classes. Conclusion Effective preventive pharmacological treatments for RA were acceptable to most participants. The relative importance of treatment attributes and likely uptake of fixed treatment profiles were predicted by participant characteristics.

Acceptable risks of treatments to prevent rheumatoid arthritis among first-degree relatives: demographic and psychological predictors of risk tolerance

“…This study is part of a case study for the Innovative Medicines Initiative project ‘PREFER’ (Patient Preferences in Benefit-Risk Assessments during the Medical Product Lifecycle), which aimed to develop evidence-based recommendations on how and when preference studies can inform decision-making during drug development. 14 The case study protocol 27 and other results from the case study 21 have been published previously.…”

“…17 One study eliciting preferences for preventive treatments for RA which used profile-case best-worst scaling 18 had a relatively small sample size and did not report acceptable benefit-risk trade-offs. A small number of quantitative studies have employed discrete choice experiments (DCEs), [19][20][21] which require large sample sizes to estimate reliable models and to account for both acceptability of treatment risks and heterogeneity among preferences. Preference heterogeneity in a DCE study of 288 self-reported FDRs was not explained by participant characteristics.…”

Can the General Public Be a Proxy for an “At-Risk” Group in a Patient Preference Study? A Disease Prevention Example in Rheumatoid Arthritis

“…Health literacy and perceived risk partly explained preference heterogeneity; however, no other patient characteristics explained heterogeneity. 51 Although scale was investigated, a scaleadjusted latent class model could have been suitable for this purpose but would have further complicated sample comparisons. 52 Despite the limitations, this study provides a starting point for discussion on using the general public as a proxy in the context of disease prevention.…”

‘What is my risk really?’: a qualitative exploration of preventive interventions among individuals at risk of rheumatoid arthritis

“…Approaching RA prevention presents an ongoing challenge in rheumatology. Although early initiation of immunotherapies in at-risk individuals is encouraging, previous studies have shown that many at-risk individuals have concerns about pharmacological side effects [ 13 , 16 , 19 ]. In congruence, our findings suggest that side effects associated with the medication used to treat RA could cause concern for at-risk individuals, regardless of how proactive they might be in other ways.…”

“…There is a pressing need for further understanding of how at-risk individuals weigh up the risk of developing RA vs the benefits of preventive interventions [ 18 ]. Furthermore, determining preferences for lifestyle approaches to reduce the risk of RA development in at-risk groups has been identified as an important topic for further study [ 19 , 20 ].…”