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Les agents biologiques representent un segment important des depenses en medicaments au Canada : alors qu'ils ne constituaient que 1,5% du volume des ordonnances, les agents biologiques ont genere 27,3% [...]

Biologics represent a large segment of drug spending in Canada: although they constituted just 1.5% of prescription volumes, biologics accounted for 27.3% of expenditures in 2018. (1) As of 2018, [...]

The objective was to understand how the expansion of rheumatology supply and the introduction of multidisciplinary care was associated with access to rheumatology services. We accessed Population Data BC, a longitudinal database with de‐identified individual‐level health data on all residents of British Columbia, Canada, to analyze physician visits and prescribing from 2010–2011 to 2019–2020. We calculated access as the time from referral to first rheumatologist visit and, for people with rheumatoid arthritis (RA), time to first disease‐modifying antirheumatic drug (DMARD). Associations between lag time, patient characteristics, and system variables were explored using quantile regression. Over the study period, there were 149,902 new rheumatologist visits, with 31% more visits in 2019–2020 than in 2010–2011. The proportion of first visits for patients with inflammatory arthritis increased from 28% to 51%. The median time from referral to first visit decreased by 22 days (35%) from 63 days (interquartile range 21–120 days) in 2010–2011. For people with RA, time from referral to DMARD decreased by 4 days (6%) to 62 days. Male sex, living in metropolitan areas, and having a rheumatologist who used a multidisciplinary care assessment code were associated with shorter times from referral to first DMARD. Access to rheumatology care improved, and the increased proportion of patients with IA in the first visits case‐mix indicates that rheumatologist supply and incentives for multidisciplinary care may have improved referral patterns. However, time to DMARDs for people with RA remained long, and we found signals of unequal access for female patients and people living outside of metropolitan areas.

ObjectiveTo explore the experiences and perspectives of patients and rheumatologists on decision aid (DA)-led tapering of advanced therapy in rheumatoid arthritis (RA).MethodsSemistructured interviews were completed with patients and rheumatologists, embedded within a pilot study of DA-led tapering (ie, dose reduction) of biologic disease-modifying antirheumatic drugs (bDMARDs) and targeted synthetic DMARDs (tsDMARDs) in RA. All patients were in sustained (≥ 6 mos) remission and had chosen to reduce their therapy after a DA-led shared decision with their rheumatologist. The rheumatologists included those participating in the pilot (n = 4), and those who were not (n = 8). Reflexive thematic analysis of audiotaped and transcribed interviews identified themes in the group experiences.ResultsPatients (n = 10, 6 female) unanimously found the DA easy to understand and felt confident in shared decision making about treatment tapering and managing flares. Rheumatologists’ (n = 12, 5 female) perspectives on tapering bDMARDs and tsDMARDs varied widely, from very supportive to completely opposed, and influenced their views on the DA. Rheumatologists expressed concerns about patient comprehension, destabilizing a stable situation, risks of flare, and extending appointment times. Despite their initial reservations about sending the DA to all eligible patients ahead of appointments, 3 of 4 participating rheumatologists adopted this approach during the pilot, which had the benefit of facilitating patient-led conversations.ConclusionA DA-led strategy for tapering advanced therapy in RA was acceptable to patients and feasible in practice. Sending patients a DA ahead of their appointment facilitated patient-led conversations about tapering.

The Public Health Agency of Canada has estimated that a quarter of adult Canadians are at risk for obstructive sleep apnea (OSA). (1) Despite a large potential disease burden, insufficient [...]

Uptake of biosimilars has been suboptimal in North America. This study was undertaken to quantify the impact of various policy interventions (namely, new start and switching policies) on uptake and spending on biosimilar infliximab and etanercept in British Columbia (BC), Canada. We used administrative claims data to identify BC residents ≥18 years of age with rheumatoid arthritis, ankylosing spondylitis, psoriatic arthritis, and/or plaque psoriasis who qualified for public drug coverage from January 2013 to November 2020. Using interrupted time series analysis, we studied the change in proportion spent on and prescriptions dispensed of biosimilar infliximab and etanercept out of the total amount per agent after new start and biosimilar switching policies were implemented. Our study included 208,984 individuals living with rheumatoid arthritis, ankylosing spondylitis, plaque psoriasis, and/or psoriatic arthritis, corresponding to 5,884 patients taking infliximab and etanercept. After the new start policy, we detected a small gradual increase in the proportion of dispensed biosimilar etanercept prescriptions of 0.65% per month (95% confidence interval [95% CI] 0.44, 0.85). The trend related to the proportion of total spending on biosimilar etanercept also increased (0.51% [95% CI 0.28, 0.73]). After the switching policy, there was a sustained increase in the proportion of dispensed biosimilar etanercept and infliximab prescriptions of 76.98% (95% CI 75.56, 78.41) and 58.43% (95% CI 52.11, 64.75), respectively. Similarly, there was a persistent increase in monthly spending on biosimilar etanercept and infliximab of 78.22% (95% CI 76.65, 79.79) and 71.23% (95% CI 66.82, 75.65), respectively. We found that mandatory switching policies were much more effective than new starting policies for increasing the use of biosimilar medications.

We evaluated DECIDE, an online pretest decision-support tool for diagnostic genomic testing, in nongenetics specialty clinics where there are no genetic counselors (GCs).

Contemporary management of COPD relies on exacerbation history to risk-stratify patients for future exacerbations. Multivariable prediction models can improve the performance of risk stratification. However, the clinical utility of risk stratification can vary from one population to another.

ObjectiveTo understand how people with chronic immune-mediated inflammatory diseases (IMIDs) trade off the benefits and risks of coronavirus disease 2019 (COVID-19) vaccine options.MethodsWe conducted an online discrete-choice experiment in people with IMIDs to quantify the relative importance (RI) of attributes relevant to COVID-19 vaccination. Participants were recruited between May and August 2021 through patient groups and clinics in Canada, and completed 10 choices where they selected 1 of 2 hypothetical vaccine options or no vaccine. The RI of each attribute was estimated and heterogeneity was explored through latent class analysis.ResultsThe survey was completed by 551 people (89% female, mean age 46 yrs) with a range of IMIDs (inflammatory bowel disease [48%], rheumatoid arthritis [38%], systemic lupus erythematosus [16%]). Most had received 1 (94%) or 2 (64%) COVID-19 vaccinations. Across the ranges of levels considered, vaccine effectiveness was most important (RI = 66%), followed by disease flare (21%), rare but serious risks (9%), and number/timing of injections (4%). Patients would accept a risk of disease flare requiring a treatment change of ≤ 8.8% for a vaccine with a small absolute increase in effectiveness (10%). Of the 3 latent classes, the group with the greatest aversion to disease flare were more likely to be male and have lower incomes, but this group still valued effectiveness higher than other attributes.ConclusionPatients perceived the benefits of COVID-19 vaccination to outweigh rare serious risks and disease flare. This supports COVID-19 vaccine strategies that maximize effectiveness, while recognizing the heterogeneity in preferences that exists.

Background: Long-term control or remission of rheumatoid arthritis (RA) may be possible with very early treatment. However, no optimal first therapeutic strategy has been determined. Objective: To assess the potential cost-effectiveness of major therapeutic strategies for very early RA. Design: Decision analytic model with probabilistic sensitivity analyses. Data Sources: Published data, the National Data Bank for Rheumatic Diseases, and actual 2007 hospital costs. Target Population: U.S. adults with very early RA (symptom duration [less than or equal to] 3 months). Time Horizon: Lifetime. Perspective: Health care provider and societal. Intervention: 3 management strategies were compared: a symptomatic or 'pyramid' strategy with initial nonsteroidal anti-inflammatory drugs, patient education, pain management, and low-dose glucocorticoids, and disease-modifying antirheumatic drugs (DMARDs) at 1 year for nonresponders; early DMARD therapy with methotrexate; and early therapy with biologics and methotrexate. Outcome Measures: Cost per quality-adjusted life-year (QALY) gained. Results of Base-Case Analysis: By reducing the progression of joint erosions and subsequent functional disability, both early intervention strategies increase quality-adjusted life more than the pyramid strategy and save long-term costs. When the cost of very early intervention is factored in, the cost-effectiveness ratio of the early DMARD strategy is $4849 per QALY (95% Cl, $0 to $16 354 per QALY) compared with the pyramid strategy, whereas the benefits gained through the early biologic strategy come at a substantial incremental cost. The early DMARD strategy maximizes the effectiveness of early DMARDs and reserves the use of biologics for patients with more treatment-resistant disease of longer duration, for which the incremental benefit of biologics is greater. Results of Sensitivity Analysis: The early biologic strategy becomes more cost-effective if drug prices are reduced, risk for death is permanently lowered through biologic therapy, patients experience drug-free remission, responders can be selected before therapy initiation, or effective alternative antirheumatic agents are available for patients for whom several biologics have failed. Limitations: Data on the long-term effect of very early therapeutic interventions on the natural progression in disability and joint erosions are limited. The study considered only tumor necrosis factor inhibitors and not the newer biologics. Conclusion: According to the most objective measures of RA progression, very early intervention with conventional DMARDs is cost-effective. The cost-effectiveness of very early intervention with biologics remains uncertain.

ObjectiveTo provide the initial installment of a living guideline that will provide up-to-date guidance on the pharmacological management of patients with rheumatoid arthritis (RA) in Canada.MethodsThe Canadian Rheumatology Association (CRA) formed a multidisciplinary panel composed of rheumatologists, researchers, methodologists, and patients. In this first installment of our living guideline, the panel developed a recommendation for the tapering of biologic and targeted synthetic disease-modifying antirheumatic drug (b/ts DMARD) therapy in patients in sustained remission using the GRADE (Grading of Recommendations Assessment, Development, and Evaluation) approach, including a health equity framework developed for the Canadian RA population. The recommendation was adapted from a living guideline of the Australia & New Zealand Musculoskeletal Clinical Trials Network.ResultsIn people with RA who are in sustained low disease activity or remission for at least 6 months, we suggest offering stepwise reduction in the dose of b/tsDMARD without discontinuation, in the context of a shared decision, provided patients are able to rapidly access rheumatology care and reestablish their medications if needed. In patients where rapid access to care or reestablishing access to medications is challenging, we conditionally recommend against tapering. A patient decision aid was developed to complement the recommendation.ConclusionThis living guideline will provide contemporary RA management recommendations for Canadian practice. New recommendations will be added over time and updated, with the latest recommendation, evidence summaries, and Evidence to Decision summaries available through the CRA website (www.rheum.ca).

Biologic drugs are well-known for both revolutionizing the treatment of rheumatoid arthritis and their high costs. (1) Recently, a randomized noninferiority trial, partly funded and conducted in Canada, showed that [...]

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The Hypertension Canada 2018 clinical guidance for pregnancy hypertension recommends antihypertensive therapy for raised blood pressure in pregnancy to a target diastolic blood pressure (BP) of 85 mm Hg (ie, “tight” control). Although evidence shows this approach reduces the incidence of severe maternal hypertension without increasing risk to the baby, we do not know how pregnant women feel about this approach, particularly as they are generally medication averse. An online survey assessed pregnant women’s preferences for management of pregnancy hypertension and explored decisional needs. The survey included information provision and knowledge assessment, a preference elicitation task, and a decisional needs assessment. Survey responses were analysed descriptively, by latent class analysis to identify treatment priority subgroups, and by logistic regression to assess predictors of treatment preference. For the 183 pregnant respondents, 3 treatment priority subgroups were identified, with most respondents expressing equal prioritization of treatment outcomes and components (eg, taking medication). Participants who preferred tight control (49%) were more often white (odds ratio [OR]: 2.38; 95% confidence interval [CI]: 1.18-4.55), with a university education/professional qualification (OR 1.95; 95% CI: 1.02-3.7), and had greater knowledge about pregnancy hypertension and pregnancy complications (OR 1.37; 95% CI: 1.15-1.65). Participants reported diverse decisional needs, but most preferred to make final treatment decisions themselves (70%), with (48%) or without (22%) physician input. The diversity of priorities, preferences, and decisional needs for management of pregnancy hypertension identified in this study emphasises the importance of an individualized approach to treatment recommendations.

Background/aimsTo validate a preference-based Diabetic Retinopathy Utility Index (DRU-I) using discrete choice experiment (DCE) methods and assess disutilities associated with vision-threatening DR (VTDR: severe non-proliferative DR, proliferative DR and clinically significant macular oedema) and associated vision impairment.MethodsThe DRU-I comprises five quality-of-life dimensions, including Visual symptoms, Activity limitation/mobility, Lighting and glare, Socio-emotional well-being and Inconvenience, each rated as no, some, or a lot of difficulty. The DRU-I was developed using a DCE comprising six blocks of nine choice sets which, alongside the EuroQoL-5D (EQ-5D-3L) and Vision and Quality of Life (VisQoL) utility instruments, were interviewer-administered to participants. To ensure the DRU-I was sensitive to severe disease, we oversampled patients with VTDR. Data were analysed using conditional logit regression.ResultsOf the 220 participants (mean±SD age 60.1±11.3 years; 70.9% men), 57 (29.1%) and 139 (70.9%) had non-VTDR and VTDR, respectively, while 157 (71.4%), 20 (9.4%) and 37 (17.3%) had no, mild or moderate/severe vision impairment, respectively. Regression coefficients for all dimensions were ordered as expected, with worsening levels in each dimension being less preferred (theoretical validity). DRU-I utilities decreased as DR severity (non-VTDR=0.87; VTDR=0.80; p=0.021) and better eye vision impairment (none=0.84; mild=0.78; moderate/severe=0.72; p=0.012) increased. DRU-I utilities had low (r=0.39) and moderate (r=0.58) correlation with EQ-5D and VisQoL utilities, respectively (convergent validity).DiscussionThe DRU-I can estimate utilities associated with vision-threatening DR and associated vision impairment. It has the potential to assess the cost-effectiveness of DR interventions from a patient perspective and inform policies on resource allocation relating to DR.

Objective.To summarize patients’ preferences for disease-modifying antirheumatic drug (DMARD) therapy in rheumatoid arthritis (RA).Methods.We conducted a systematic review to identify English-language studies of adult patients with RA that measured patients’ preferences for DMARD or health states and treatment outcomes relevant to DMARD decisions. Study quality was assessed using a published quality assessment tool. Data on the importance of treatment attributes and associations with patient characteristics were summarized across studies.Results.From 7951 abstracts, we included 36 studies from a variety of countries. Most studies were in patients with established RA and were rated as medium- (n = 19) or high-quality (n = 12). The methods to elicit preferences varied, with the most common being discrete choice experiment (DCE; n = 13). Despite the heterogeneity of attributes in DCE studies, treatment benefits (disease improvement) were usually more important than both non-serious (6 of 8 studies) and serious adverse events (5 of 8), and route of administration (7 of 9). Among the non-DCE studies, some found that patients placed high importance on treatment benefits, while others (in patients with established RA) found that patients were quite risk averse. Subcutaneous therapy was often but not always preferred over intravenous therapy. Patient preferences were variable and commonly associated with the sociodemographic characteristics.Conclusion.Overall, the results showed that many patients place a high value on treatment benefits over other treatment attributes, including serious or minor side effects, cost, or route of administration. The variability in patient preferences highlights the need to individualize treatment choices in RA.

Millions of dollars are spent on the development of new personalized medicine technologies. While these research costs are often supported by public research funds, many diagnostic tests and biomarkers are not adopted by the healthcare system due to lack of evidence on their cost–effectiveness. We describe a stepwise approach to conducting cost–effectiveness analyses that are performed early in the technology's development process and can help mitigate the potential risks of investment. Decision analytic modeling can identify the key drivers of cost effectiveness and provide minimum criteria that the technology needs to meet for adoption by public and private healthcare systems. A value of information analysis can quantify the added value of conducting more research to provide further evidence for policy decisions. These steps will allow public research funders to make better decisions on their investments to maximize the health benefits and to minimize the number of suboptimal technologies.

Background:Patient education facilitates construction of a correct illness representation, improves beliefs about medications, and improves knowledge, factors that have been associated with better adherence. Objective:Our objective was to characterize the published literature about atrial fibrillation (AF) patients’ disease and medication knowledge to identify knowledge gaps and misconceptions to inform AF patient education strategies. Methods:Following PRISMA guidelines, we searched PubMed, EMBASE, CINAHL, and PsychINFO from inception to May 2018 for studies that assessed AF patients’ knowledge about their condition and medications. For quantitative studies, we extracted the proportion of participants who provided correct answers to the questions asked about their condition, medications, or risk of stroke. We classified data for related questions into knowledge domains. A random-effects meta-analysis was conducted for each knowledge domain. A domain was considered a knowledge gap if the pooled mean proportion of participants who demonstrated knowledge of it was ≤50%, regardless of CI. Qualitative data were summarized narratively. Results:A total of 21 studies were included. AF- and stroke-related knowledge gaps and misconceptions included the following: AF can be asymptomatic, AF can predispose to heart failure, women are at a higher risk of stroke, the definition of ischemic stroke, and patients’ awareness of their diagnosis. Medication-related knowledge gaps were antithrombotic-drug interactions, antithrombotic-food interactions, vitamin K content of foods, the term INR(international normalized ratio) and its interpretation, and the required actions in case of a missed dose. Conclusion and Relevance:This systematic review identified several AF patient knowledge gaps about their condition and its treatment that can inform the development of AF patient education programs.

Background:Stroke prevention therapy decisions for patients with atrial fibrillation (AF) are complex and require trade-offs, but few validated patient decision aids (PDAs) are available to facilitate shared decision making. Objective:To evaluate the effects of a novel PDA on decision-making parameters for AF patients choosing stroke prevention therapy. Methods:We developed an evidence-based individualized online AF PDA for stroke prevention therapy and evaluated it in a prospective observational pilot study. The primary outcome was decisional conflict. Secondary outcomes were knowledge, usability/acceptability, patient preferences, effects on therapy choices, and participant feedback. Results:37 participants completed the PDA. The PDA could be completed independently and was well accepted. It significantly decreased the mean decisional conflict score (P< 0.001) and all its subscales and increased participant AF knowledge (P= 0.02). 76% of participants indicated that their individualized therapy attribute ranking was congruent with their values. The PDA-generated best-match therapy was chosen by 70% of participants in decision 1 (no therapy, aspirin, or oral anticoagulant), and 17% for decision 2 (choice of anticoagulant). Among AF patients, 60% chose a different drug than that currently prescribed to them. Conclusion and Relevance:Our PDA was effective for reducing decisional conflict, increasing patient knowledge, eliciting patients’ values, and presenting therapy options that aligned with patients’ values and preferences. Using the PDA revealed that many patients have therapy preferences different from their currently prescribed treatment. The PDA is a practical and potentially valuable tool to facilitate decision making about stroke prevention therapy for AF.

The Canadian Inflammatory Myopathy Study (CIMS) is a multicenter prospective cohort recruiting in 8 centers across Canada. One of the aims of CIMS is to conduct and participate in clinical trials in autoimmune inflammatory myopathies (AIM). Conducting clinical trials in rare diseases such as AIM presents challenges. During this symposium, experts in the field presented different solutions to successfully conduct clinical trials in AIM, including the importance of collaboration and careful trial design, as well as training and mentoring of young investigators.

To assess the extent to which ANSWER‐2, an interactive online patient decision aid, reduces patients’ decisional conflict and improves their medication‐related knowledge and self‐management capacity. We used a pre–post study design. Eligible participants had a diagnosis of rheumatoid arthritis (RA), had been recommended to start using a biologic agent or small‐molecule agent or to switch to a new one, and had internet access. Access to ANSWER‐2 was provided immediately after enrollment. Outcome measures included 1) the Decisional Conflict Scale (DCS), 2) the Medication Education Impact Questionnaire (MeiQ), and 3) the Partners in Health Scale (PIHS). A paired t‐test was used to assess differences pre‐ and postintervention. The majority of the 50 participants were women (n = 40), and the mean ± SDage of participants was 49.6 ± 12.2 years. The median disease duration was 5 years (25th, 75th percentile: 2, 10 years). The mean ± SD DCSscore was 45.9 ± 25.1 preintervention and 25.1 ± 21.8 postintervention (mean change of −21.2 of 100 [95% confidence interval (95% CI) −28.1, −14.4], P< 0.001). Before using ANSWER‐2, 20% of participants had a DCSscore of <25, compared to 52% of participants after the intervention. Similar results were observed in the PIHS(mean ± SD25.3 ± 14.8 preintervention and 20.4 ± 13.0 postintervention; mean change of −3.7 of 88 [95% CI−6.3, −1.0], P= 0.009). Findings from the MeiQ were mixed, with statistically significant differences found only in the self‐management subscales. Patients’ decisional conflict decreased and perceived self‐management capacity improved after using ANSWER‐2. Future research comparing the effectiveness of ANSWER‐2 with that of educational material on biologic agents will provide further insight into its value in RAmanagement.

Objective.To understand the limitations with current patient-reported outcome measures (PROM) used to generate quality-adjusted life-years (QALY) in rheumatology, and set a research agenda.Methods.Two activities were undertaken. The first was a scoping review of published studies that have used PROM to generate QALY in rheumatology between 2011 and 2016. The second was an interactive “eyeball test” exercise at Outcome Measures in Rheumatology 13 that compared subdomains of widely used generic PROM, as identified through the scoping review, to subdomains of the Assessment of SpondyloArthritis Health Index (ASAS-HI) condition-specific PROM for ankylosing spondylitis.Results.The scoping review included 39 studies. Five different PROM have been used to generate QALY in rheumatology; however, the EQ-5D and Short Form 6 Dimensions (SF-6D) were used most frequently (in 32 and 9 of included studies, respectively). Special interest group participants identified energy/drive and sleep as 2 key subdomains of the ASAS-HI instrument that may be missed by the EQ-5D, and sexual function as potentially missed by the SF-6D. Participants also expressed concerns that aspects of the process of care and non-health outcomes may be missed. Three ways of incorporating additional subdomains were discussed, including using an alternative generic PROM, modifying an existing generic PROM with “bolt-on” subdomain(s), and generating societal weights for a condition-specific PROM.Conclusion.Three priorities for future research were identified: understanding whether the EQ-5D and SF-6D identify what matters to patients with different rheumatic conditions, analyzing how much patients value process or non-health outcomes, and identifying which approaches to incorporating a greater number of subdomains into the QALY are being undertaken in other disease areas.

Objective.To evaluate a classification system to define adherence to axial spondyloarthritis (axSpA) anti-tumor necrosis factor (anti-TNF) use recommendations and examine the effect of adherence on outcomes in the DESIR cohort (Devenir des Spondylarthropathies Indifférenciées Récentes).Methods.Using alternate definitions of adherence, patients were classified as adherent “timely” anti-TNF users, nonadherent “late” anti-TNF users, adherent nonusers (“no anti-TNF need”), non-adherent nonusers (“unmet anti-TNF need”). Multivariate models were fitted to examine the effect of adherence on quality-adjusted life-years (QALY), total costs, and nonbiologic costs 1 year following an index date. Generalized linear regression models assuming a γ-distribution with log link were used for costs outcomes and linear regression models for QALY outcomes.Results.Using the main definition of adherence, there were no significant differences between late anti-TNF users and timely anti-TNF users in total costs (RR 0.86, 95% CI 0.54–1.36, p = 0.516) or nonbiologic costs (RR 0.72, 95% CI 0.44–1.18, p = 0.187). However, in the sensitivity analysis, late anti-TNF users had significantly increased nonbiologic costs compared with timely users (RR 1.58, 95% CI 1.06–2.36, p = 0.026). In the main analysis, there were no significant differences in QALY between timely anti-TNF users and late anti-TNF users, or between timely users and patients with unmet anti-TNF need. In the sensitivity analysis, patients with unmet anti-TNF need had significantly lower QALY than timely anti-TNF users (−0.04, 95% CI −0.07 to −0.01, p = 0.016).Conclusion.The effect of adherence to anti-TNF recommendations on outcomes was sensitive to the definition of adherence used, highlighting the need to validate methods to measure adherence.

The Rheumatology Informatics System for Effectiveness (RISE) is a national electronic health record (EHR)–enabled registry. RISE passively collects data from EHRs of participating practices, provides advanced quality measurement and data analytic capacities, and fulfills national quality reporting requirements. Here we report the registry's architecture and initial data, and we demonstrate how RISE is being used to improve the quality of care. RISE is a certified Centers for Medicare and Medicaid Services Qualified Clinical Data Registry, allowing collection of data without individual patient informed consent. We analyzed data between October 1, 2014 and September 30, 2015 to characterize initial practices and patients captured in RISE. We also analyzed medication use among rheumatoid arthritis (RA) patients and performance on several quality measures. Across 55 sites, 312 clinicians contributed data to RISE; 72% were in group practice, 21% in solo practice, and 7% were part of a larger health system. Sites contributed data on 239,302 individuals. Among the subset with RA, 34.4% of patients were taking a biologic or targeted synthetic disease‐modifying antirheumatic drug (DMARD) at their last encounter, and 66.7% were receiving a nonbiologic DMARD. Examples of quality measures include that 55.2% had a disease activity score recorded, 53.6% a functional status score, and 91.0% were taking a DMARD in the last year. RISE provides critical infrastructure for improving the quality of care in rheumatology and is a unique data source to generate new knowledge. Data validation and mapping are ongoing and RISE is available to the research and clinical communities to advance rheumatology.

The last few decades have seen major therapeutic advancements in rheumatoid arthritis (RA) therapeutics. New disease-modifying antirheumatic drugs (DMARDs) have continued to emerge, creating more choices for people. However, no therapeutic works for all patients. Each has its own inherent benefits, risks, costs, dosing, and monitoring considerations. In parallel, there has been a focus on personalized medicine initiatives that tailor therapeutic decisions to patients based on their unique characteristics or biomarkers. Personalized effect estimates require an understanding of a patient's baseline probability of response to treatment and data on the comparative effectiveness of the available treatments. However, even if accurate risk prediction models are available, trade-offs often still need to be made between treatments. In this paper, we review the history of RA therapeutics and progress that has been made toward personalized risk predictive models for DMARDs, outlining where knowledge gaps still exist. We further review why patient preferences play a key role in a holistic view of personalized medicine and how this links with shared decision-making. We argue that a “preference misdiagnosis” may be equally important as a medical misdiagnosis but is often overlooked.

New antiretroviral therapy (ART) regimens for HIV could improve clinical outcomes for patients. To inform global guidelines, we aimed to assess the comparative effectiveness of recommended ART regimens for HIV in ART-naive patients.

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The objective of this study was to evaluate the effectiveness of an online patient decision aid with individualised potential outcomes of surgery, on the quality of decisions for knee replacement surgery in routine clinical care.

OSA is a common yet underdiagnosed respiratory disorder characterized by recurrent upper airway obstruction during sleep. OSA results in sleep fragmentation and repetitive hypoxemia and is associated with a variety of adverse consequences including excessive daytime sleepiness, reduced quality of life, cardiovascular disease, decreased learning skills, and neurocognitive impairment. Neurocognitive impairments that have been linked to poor sleep include memory deficits, decreased learning skills, inability to concentrate, and decreased alertness. Furthermore, the societal and economic costs of OSA are substantial; for example, patients with OSA have a significantly greater risk of motor vehicle crashes, consume more health-care resources, and have associated annual costs in the billions of dollars per year. It is increasingly recognized that OSA may also have substantial economic consequences. Specifically, there is accumulating evidence implicating OSA as an important contributor to work disability (including absenteeism, presenteeism) and work-related injuries. This review summarizes the current state of knowledge in these two areas.

The Affordable Care Act includes provisions to encourage patient-centered care through the use of shared decision making (SDM) and patient decision aids (PtDA). PtDAs are tools that can help encourage SDM by providing information about competing treatment options and elucidating patients׳ values and preferences. Implementing PtDAs into routine practice may incur additional costs through training or increases in physician time. Prominent commentaries have proposed that these costs might be offset if patients choose less expensive options than their providers. However, the cost-effectiveness of PtDAs to date is unclear. The aim of this study was to review the economic evidence from PtDA trials. Our search identified 5347 articles, with 29 included following full-text review. Only one economic evaluation of a PtDA has been completed, which found a PtDA to be cost-saving in women with menorrhagia. Other studies included in the review indicated that PtDAs will likely increase up-front costs, but in some contexts may reduce short-term costs by reducing the uptake of invasive treatments, such as elective surgery. Few studies comprehensively captured long-term costs or measured benefits in a manner conducive to economic evaluation (QALYs or general health utilities). Our review suggests that policy makers currently have insufficient economic evidence to appropriately consider their investments in PtDAs.

Over the last decade, a number of biologic response modifiers (BRMs) have emerged and transformed rheumatoid arthritis (RA) management. Due to their relatively high costs, economic evaluations have attempted to determine their place in the RA treatment armamentarium. This article reviews three key areas where changes to the treatment paradigm challenges findings of existing economic evaluations.

Many health systems are interested in the impact of disease and interventions on non-health outcomes. Over the last 10 years, work productivity has become one of the most important topics. This study was conducted to review guidelines for economic evaluations worldwide to identify how views on the types of productivity costs to be included differ across jurisdictions and to review recent trials that have measured productivity losses to identify trends and compare consistency with guidelines from different jurisdictions. The guidelines from 28 countries were evaluated and only 12 required productivity costs to be included in the main analysis or the base case analysis. Little specific guidance was provided around the types of productivity costs to be included. Correspondingly, we identified only 10 trials that explicitly measured productivity outcomes and all were conducted after the year 2001. While there was a growth in the proportion of trials evaluating biologics to measure this outcome, it showed that fewer than 50% of even recent studies failed to measure or report productivity. Furthermore, most trials did not use a standard and validated questionnaire to measure all productivity loss components. In conclusion, whether the rationale for the exclusion of productivity impacts is that healthcare budgets should only be concerned with health impacts and ignore general social welfare impacts or whether productivity impacts should be ignored to maintain generational equity or whether the methodology of productivity measurement leads to imprecise estimates, the reality is that productivity impacts are real and to ignore them is tantamount to not being fully accountable to our citizenry.

The quality-adjusted life-year (QALY) is a construct that integrates the value or preference for a health state over the period of time in that health state. The main use of QALY is in cost-utility analysis, to help make resource allocation decisions when faced with choices. Although the concept of the QALY is appealing, there is ongoing debate regarding their usefulness and approaches to deriving QALY. In 2008, OMERACT engaged in an effort to agree on QALY approaches that can be used in rheumatology. Based on a Web questionnaire and a subsequent meeting, rheumatologists questioned whether it was relevant for OMERACT (1) to investigate use of a QALY that represents the patients’ perspective, (2) to explore the validity of the visual analog scale (VAS) to value health, and (3) to understand the validity of mapping health-specific instruments on existing preference instruments. This article discusses the pros and cons of these points in light of current insight from the point of view of health economics and decision-making theory. It also considers the further research agenda toward a QALY approach in rheumatology.

Recent years have witnessed a shift in the therapeutic approach for patients with early rheumatoid arthritis (RA). The focus of interest has been the improved outcomes achieved through the use of early aggressive disease-modifying therapy, including the use of biologic agents. Such strategies have acquisition costs which typically exceed those of older anti-rheumatic strategies. However, improved outcomes might lead to fewer hospitalizations and physician visits and improved employability, leading to future cost savings. This is in addition to the health benefits which patients value as improvements in quality of life. With many services competing to spend often limited health-care budgets, information on the relative benefits and costs of new approaches for treating RA can be useful in deciding on efficient allocation and treatment decisions.

Background: To conduct a systematic review and thematic synthesis of qualitative studies on the pregnancy and early parenting experiences of patients with inflammatory arthritis (IA). Methods: We searched online databases for English-language, qualitative studies capturing the experiences of females with IA or their healthcare providers with pregnancy and/or early parenthood. We extracted findings from included studies and used thematic synthesis to develop descriptive and higher-order analytical themes. Results: Of 20 included studies, our analysis identified 5 analytical themes among patients and 3 among providers. Patients’ reproductive desires, the impact of IA on their ability to experience pregnancy, and the availability of information to guide preparedness informed their pregnancy decisions. Patients’ IA management, pregnancy expectations, and access to support influenced their reproductive experiences. Patients’ experiences seeking information and care revealed substantial gaps in reproductive care provision to patients with IA. Reproductive uncertainty related to IA placed a heavy burden on patients’ emotional and psychological wellbeing. Reproductive care provision was influenced by providers’ perceived professional responsibility to address patients’ reproductive goals, fears of negative outcomes, and capacity to harness patient trust, incorporate reproductive care into rheumatology practice and facilitate multi-disciplinary care coordination. Conclusions: Our review illuminated several barriers to experiencing pregnancy among patients with IA, particularly related to pregnancy planning support, availability of information, and care coordination among the patient’s healthcare team. To improve care, these barriers may be mitigated through the provision of relevant, practical, and consistent information as well as patient-centred multi-disciplinary approaches for managing pregnancy among patients with IA.

OBJECTIVE: To evaluate the shortterm effect of adalimumab on work productivity in patients with moderate to severe active rheumatoid arthritis (RA). METHODS: In a substudy of the Canadian Adalimumab Clinical Trial (CanAct), clinical, health status, and productivity outcomes were measured at baseline and 12 weeks. Patients were classified as responders and nonresponders by the 20% American College of Rheumatology (ACR20) improvement criterion and the minimum clinically important difference (MCID) of the Health Assessment Questionnaire (HAQ) score (0.22), respectively. The Health and Labour Questionnaire (HLQ) was used to measure productivity outcomes and costs. RESULTS: Included in the analysis were 389 patients completing both baseline and 12-week HLQ questionnaire. Absenteeism (a decrease of 0.5 workdays per 2 weeks) and unpaid work productivity (3.5 fewer hours unpaid help per 2 weeks) were improved significantly after 12 weeks. Improvements in productivity outcomes were associated with clinical response. Bootstrapping results suggest that responders achieved statistically significant improvement in presenteeism (ACR20) and unpaid work productivity (ACR20 and HAQ) versus nonresponders. The costs saved by responders were up to $155.04 per 2 weeks more than those by nonresponders. CONCLUSION: The costs of adalimumab were partially offset, even in the short term, by cost savings induced by clinical response among Canadian patients with moderate to severe RA. These findings complement results of other study analyses that demonstrate early and sustained benefits of adalimumab.

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OBJECTIVE: Cost-utility analysis is increasingly important as healthcare providers aim to invest scarce resources in interventions offering the greatest health benefit. The ability to attach utility values to health states is essential, and is increasingly performed using generic scales. However, the evidence regarding the validity of generic utility scales in rheumatoid arthritis (RA) is unclear. We summarize and review evidence on the validity and comparative performance of generic utility scales in RA. METHODS: We searched the English-language medical literature for studies using utilities in RA between 1980 and mid-2006. Reports describing primary evidence of the validity or performance of a generic utility scale in RA were selected, summarized, and reviewed using the OMERACT filter. RESULTS: In total 923 articles were identified, of which 228 reported the use of utility scales in RA; 26 studies related to the validation or evidence of generic utility scales in RA, the EQ-5D, Health Utility Index-2 (HUI2) and HUI3, SF-6D, and Quality of Well-Being Scale. The EQ-5D, HUI2 and HUI3, and SF-6D all have consistent evidence of construct validity and responsiveness in RA, but each has limitations. CONCLUSION:The EQ-5D and HUI3 have been the most extensively studied instruments and show validity and responsiveness for use in RA, but both instruments have limitations. The SF-6D is relatively new and appears to have potential for use in milder RA, but needs further evaluation. More longitudinal head-to-head evaluation of measures is needed across the spectrum of RA disease severity to further investigate their comparative properties, and to seek consensus on the best utility measure for use in economic evaluation.

To estimate the relationship between preference‐based measures, EuroQol (EQ‐5D) and SF‐6D, and the Health Assessment Questionnaire (HAQ) disability index (DI) in patients with rheumatoid arthritis (RA), and to characterize components that are predictors of health utility.Patients with RA participating in 2 studies in the UK (n = 151) and Canada (n = 319) completed the HAQ, EQ‐5D, and Short Form 36 (SF‐36). The SF‐36, a generic measure of quality of life, was converted into the preference‐based SF‐6D. From these results we developed models of the relationship between the HAQ and SF‐6D and EQ‐5D using various regression analyses.The optimal model developed for the EQ‐5D entered levels for each item as independent variables (model 5). A root mean square error (RMSE) of 0.18 suggested relatively good predictive ability. For the SF‐6D, RMSEs were lower (0.09), suggesting better predictions than for the EQ‐5D, but models with more explanatory variables did not improve results (model 2 or 4 optimal). The models were able to predict actual SF‐6D and EQ‐5D across the range of the HAQ DI.Our approach enabled calculations of quality‐adjusted life years from existing trials where only the HAQ was measured. All aspects of the HAQ may not be reflected in the preference‐based measures, and this method is suboptimal to direct measurement of health state utility in clinical trials. Given this limitation, our approach provides an alternative for researchers who need health‐state utility values, but had not included a preference‐based measure in their clinical study because of resource constraints or a desire to limit patient burden.

OBJECTIVE: To construct a prognostic algorithm to predict 5-year functional outcome in rheumatoid arthritis (RA), based on the Health Assessment Questionnaire (HAQ). METHODS: Data from all patients with 5-year followup (n = 985) were used from an inception cohort, the Early Rheumatoid Arthritis Study (ERAS). Possibly relevant prognostic factors considered in the initial stage of the model-building process were standard clinical, radiological, and laboratory features measured at baseline and at 1 year. Multivariate analysis was performed using logistic regression, and the predictive performance of the model was tested using measures of discrimination and calibration. RESULTS: Bootstrap resampling identified 6 variables that consistently predicted severe functional outcome. Functional grade III/IV (odds ratio 6.7) and HAQ at 1 year (odds ratio 2.4) were the most important. Other variables included socioeconomic status, hemoglobin, and radiographic and disease activity scores. Estimates of the regression coefficients and performance were corrected for over-fitting. Reasonably large values for the c-index (0.82) and the Nagelkerke R2 (0.39) indicate that the set of prognostic factors explains the variation in outcome to a degree that implies good prediction for individual patients. CONCLUSION: The algorithm identifies patients in the first year of RA who are likely to have poor function by 5 years and who could potentially benefit from aggressive drug therapy. A nomogram is produced for simple application of the model in clinical practice. While further external validation is necessary, this model could allow clinicians to target aggressive therapy earlier in a patient's disease course.

The past 10 years has witnessed a major transformation in the treatment of rheumatoid arthritis, a chronic condition that leads to significant morbidity, impairment in quality of life and mortality. Adalimumab joins a class of biologic response modifiers that prevent joint destruction and maintain functional status. For expensive interventions such as biologic response modifiers to be a valuable use of healthcare resources, they must lower healthcare costs by reducing the prevalence of hospitalizations, assist people with rheumatoid arthiritis in maintaining employment and improve patient quality of life. The rheumatoid artiritis market is competitive and growing quickly. Policy makers are faced with decisions surrounding the value of biologic response modifiers over conventional therapies, and whether one biologic response modifier has an advantage over another.

Although on balance, human milk confers more health benefits in comparison to formula, breastfeeding may not be the optimal choice for a given family at a given time. Shared decision-making (SDM) can improve patient-centred care for infant feeding choices. We aimed to identify the decision-making needs, challenges, and opportunities among health care professionals supporting infant feeding choices.

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OBJECTIVE: To estimate the lost investment of domestically educated doctors migrating from sub-Saharan African countries to Australia, Canada, the United Kingdom, and the United States. Design Human capital cost analysis using publicly accessible data. Settings Sub-Saharan African countries. Participants Nine sub-Saharan African countries with an HIV prevalence of 5% or greater or with more than one million people with HIV/AIDS and with at least one medical school (Ethiopia, Kenya, Malawi, Nigeria, South Africa, Tanzania, Uganda, Zambia, and Zimbabwe), and data available on the number of doctors practising in destination countries. MAIN OUTCOME MEASURES: The financial cost of educating a doctor (through primary, secondary, and medical school), assuming that migration occurred after graduation, using current country specific interest rates for savings converted to US dollars; cost according to the number of source country doctors currently working in the destination countries; and savings to destination countries of receiving trained doctors. RESULTS: In the nine source countries the estimated government subsidised cost of a doctor’s education ranged from $21 000 (£13 000; €15 000) in Uganda to $58 700 in South Africa. The overall estimated loss of returns from investment for all doctors currently working in the destination countries was $2.17bn (95% confidence interval 2.13bn to 2.21bn), with costs for each country ranging from $2.16m (1.55m to 2.78m) for Malawi to $1.41bn (1.38bn to 1.44bn) for South Africa. The ratio of the estimated compounded lost investment over gross domestic product showed that Zimbabwe and South Africa had the largest losses. The benefit to destination countries of recruiting trained doctors was largest for the United Kingdom ($2.7bn) and United States ($846m). CONCLUSIONS: Among sub-Saharan African countries most affected by HIV/AIDS, lost investment from the emigration of doctors is considerable. Destination countries should consider investing in measurable training for source countries and strengthening of their health systems.