Background: There is increasing research focus on intervention for rheumatoid arthritis (RA) at the earliest stages of disease development, including treatment to prevent RA in at-risk groups. Novel cellular therapies are in development, and the effectiveness of existing immunomodulatory agents to prevent RA in those at risk is under investigation. Quantitative evidence of likely uptake of preventive treatments, and preferences for benefits and risks of such treatments is limited.
Objectives: To quantify preferences for preventive therapies for RA.
Methods: A web-based survey (n = 2959) was administered to an age- and gender- stratified sample of adults in the general population from online survey panels in the UK, Germany, and Romania. After receiving information about RA, questions to check comprehension of background information, an introduction to the survey tasks and warm-up questions, participants were asked to imagine that they were experiencing arthralgia (without swelling) and had positive autoantibody tests indicating a 60% chance of developing RA in the next two years. Using a discrete choice experiment with a Bayesian D-efficient design, participants were offered a series of 15 choices between no treatment and two unlabeled hypothetical treatments to lower risk of RA development. Treatments were defined by six attributes with varying levels including benefits, risks, and frequency/route of administration (Table 1). One choice task with fixed levels described treatments representative of those under investigation for RA prevention (abatacept, hydroxychloroquine, atorvastatin and tolerogenic cell-based therapy). Attribute selection and presentation was informed by previous qualitative research, ranking surveys, systematic literature review, and expert opinion. Survey design was informed by patient research partners. The survey was pre-tested during qualitative interviews and revised. A pilot of the final survey with 100 respondents was conducted to obtain priors for the final experimental design. Random parameters logit (RPL) models were used to estimate relative importance of treatment attributes and likely treatment uptake rates in each country.
Results: Across all three countries, effectiveness was the treatment attribute that had most impact on treatment choice (Figure 1). Method of administration was second most important for respondents from the UK and Romania but less important for German respondents. Risks of serious infection and serious side effects were more important determinants of treatment choice for respondents in Romania than they were in the UK and Germany. Percentage choice of fixed profiles reflecting abatacept, atorvastatin, hydroxychloroquine, tolerogenic cell-based therapy and no treatment differed across countries (χ2=78.90; p<0.001): 28.3%, 20.6%, 22.2% 18.5% and 10.4% respectively in the UK; 31.3%, 18.8%, 11.2%, 23.4% and 15.3% in Germany; and 27.6%, 20.5%, 15.8%, 21.7% and 14.4% in Romania.
Conclusion: This study suggests that effective preventive treatments for RA are acceptable to members of the general population told to assume up a 60% chance of developing RA. The relative importance of treatment attributes and likely uptake of fixed treatment profiles differed across countries. These findings are informative for the design of prevention trials, and the development of informational resources and efficient preventive strategies for those at risk of developing RA.
Acknowledgements: On behalf of the PREFER project. PREFER received funding from the IMI 2 Joint Undertaking (grant No. 115966), which receives support from the EU’s Horizon 2020 research and innovation program and European Federation of Pharmaceutical Industries and Associations (EFPIA). K. Raza is supported by the NIHR Birmingham Biomedical Research Centre.
Disclosure of Interests: Gwenda Simons: None declared, Jorien Veldwijk: None declared, Rachael Di Santostefano Shareholder of: Johnson & Johnson, Employee of: Janssen R&D (of Johnson & Johnson), Matthias Englbrecht Speakers bureau: AbbVie, Chugai, Eli Lilly, Novartis, Roche, Sanofi, Mundipharma, Paid instructor for: AbbVie, Chugai, Roche, Consultant of: AbbVie, Novartis, Roche, Sanofi, Grant/research support from: Roche, Chugai, Christine Radawski Shareholder of: Eli Lilly & Company, Employee of: Eli Lilly & Company, Larissa Valor: None declared, Karim Raza Consultant of: Personal fees from Abbvie, Pfizer, Sanofi, Lilly, Bristol Myers Squibb, UCB, Janssen, and Roche Chugai, Grant/research support from: Abbvie and Pfizer, M. Falahee: None declared
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