Abstract

BackgroundThere is a growing research focus on the development of interventions to reduce risk of rheumatoid arthritis (RA) in at-risk individuals.(1) A recent survey of the general population asked to assume a 60% risk of RA established that hypothetical preventive treatments were acceptable to most participants.(2) However the preferences of individuals who actually have an elevated risk of RA, such as first-degree relatives (FDRs) of RA patients, are not well understood.ObjectivesTo quantify FDRs’ preferences for preventive treatments for RA.MethodsAdult FDRs in the UK and Germany were invited to take part in a web-based survey via patients with clinician-confirmed RA either during a rheumatology clinic visit or by mail. In addition, FDRs taking part in a UK-based prospective cohort (PREVeNT-RA) were invited via email. Participants received information about RA followed by questions to check comprehension, and an introduction to the survey including warm-up questions. They were asked to imagine they were experiencing arthralgia and had positive autoantibody tests indicating a 60% chance of developing RA in the next two years. Using a discrete choice experiment, participants were offered a series of 15 choices between no treatment and two unlabeled hypothetical treatments to reduce risk of RA. Treatments were defined by six attributes with varying levels, describing benefits, risks, and frequency/route of administration (Table 1). Attribute selection and presentation was informed by qualitative research, ranking surveys, systematic literature review, and expert opinion. Survey layout was informed by patient research partners and qualitative pre-testing. A two-class latent class analysis was used to estimate preferences and calculate relative importance of treatment attributes and predicted uptake. A panel mixed logit model was used to obtain maximum acceptable risk estimates.Table 1.Treatment attributes and levelsAttributeLevelsChance of developing RA reduced from 60% to10%; 20%; 30%; 40%How the treatment is takenA shallow injection under the skinA drip into the veinOne or two tabletsHow often the medication has to be takenDailyWeeklyMonthlyEvery 6 monthsChance of mild side effects2%; 5%; 10%Chance of a serious infection due to treatment0%; 1%; 5%Chance of a serious side effect that is potentially irreversible1 in 100,000 people20 in 100,000 people100 in 100,000 peopleResults356 FDRs (252 female, 289 in the UK) responded. While treatment effectiveness was the most important attribute in both classes (Figure 1), the importance of other attributes differed between classes, with method and frequency of treatment administration being more important in class 2 and risk of mild side effects only impacting treatment choice in class 1. Perceived risk of developing RA predicted class assignment; those with higher perceived risk were more likely to belong to class 1. On average, the predicted uptake of treatment profiles estimating prevention candidates: abatacept; atorvastatin; hydroxychloroquine; tolerogenic cell-based therapy; and no treatment would be 50%, 15%, 9%, 18% and 0%, respectively. Finally, the maximum acceptable risk participants were willing to accept were 81%, 25% and 3% point increases in risk of mild side effects, serious infection, and serious side effects, respectively, for medicines that would reduce their risk of developing RA in the upcoming two years from 60% to 20%.ConclusionEffective preventive treatments for RA were acceptable to FDRs asked to assume a 60% chance of developing RA. Mode and frequency of treatment administration had a greater impact on treatment choices for participants with a lower perceived risk of RA. These findings are informative for target product profile development, endpoint selection, benefit-risk assessment, regulatory approval, and development of informational resources for those at risk of RA.

Full Text
Published version (Free)

Talk to us

Join us for a 30 min session where you can share your feedback and ask us any queries you have

Schedule a call