Quantifying hospital-associated costs, and accompanying travel costs and productivity losses, before and after withdrawing TNF-α inhibitors in juvenile idiopathic arthritis.

OBJECTIVE: To quantify differences in hospital-associated costs, and accompanying travel costs and productivity losses, before and after withdrawing TNF-α inhibitors (TNFi) in JIA patients. METHODS: This was a retrospective analysis of prospectively collected data from electronic medical records of paediatric JIA patients treated with TNFi, which were immediately discontinued, spaced (increased treatment interval) or tapered (reduced subsequent doses). Costs of hospital-associated resource use (consultations, medication, radiology procedures, laboratory testing, procedures under general anaesthesia, hospitalization) and associated travel costs and productivity losses were quantified during clinically inactive disease until TNFi withdrawal (pre-withdrawal period) and compared with costs during the first and second year after withdrawal initiation (first and second year post-withdrawal). RESULTS: Fifty-six patients were included of whom 26 immediately discontinued TNFi, 30 spaced and zero tapered. Mean annual costs were €9165/patient on active treatment (pre-withdrawal) and decreased significantly to €5063/patient (-44.8%) and €6569/patient (-28.3%) in the first and second year post-withdrawal, respectively (P < 0.05). Of these total annual costs, travel costs plus productivity losses were €834/patient, €1180/patient, and €1320/patient in the three periods respectively. Medication comprised 80.7%, 61.5% and 72.4% of total annual costs in the pre-withdrawal, first and second year post-withdrawal period, respectively. CONCLUSION: In the first two years after initiating withdrawal, the total annual costs were decreased compared with the pre-withdrawal period. However, cost reductions were lower in the second year compared with the first year post-withdrawal, primarily due to restarting or intensifying biologics. To support biologic withdrawal decisions, future research should assess the full long-term societal cost impacts, and include all biologics.

Experiences of Irritable Bowel Syndrome Patients in a Virtual Yoga Program: Qualitative Findings from a Clinical Trial.

BACKGROUND AND AIMS: In-person yoga interventions have shown feasibility and effectiveness in improving the outcomes of patients with irritable bowel syndrome (IBS), but experiences in virtual yoga interventions have not been examined. This study aimed to explore patients' experiences of a virtually delivered yoga intervention for IBS. METHODS: An embedded qualitative substudy was included in a randomized controlled trial examining the feasibility and effectiveness of a virtual yoga program among adult patients with IBS. Semi-structured interviews captured participants' past and current experiences, program satisfaction, perceived impact on IBS symptoms and overall physical and mental health, facilitators and barriers to participation, perceptions of social support and supervised learning, and input on improving future programming. Data were coded and analyzed in duplicate using NVivo 12. An analytic template based on the interview guide was developed and thematic analysis identified themes, as well as the relationship between themes and subthemes. RESULTS: Among the 14 participants (all female, mean age 47.7 years), three major themes were identified: (1) positive experience in the yoga program, (2) incorporating yoga into IBS management post-study, and (3) recommendations for program improvement. CONCLUSION: Patients with IBS experience in a virtual yoga program was positive with improvements in physical and mental health outcomes. Considering the barriers and facilitators to participating in an online yoga program along with participant recommendations may improve future intervention design and delivery to increase self-efficacy and confidence among patients with IBS.

Validation of the EQ-5D-Y-5L parent-proxy version among children with juvenile idiopathic arthritis.

OBJECTIVES: Juvenile idiopathic arthritis (JIA) is the most common type of arthritis among children. It can cause joint pain and permanent physical damage, which affects mobility and daily activities. The EQ-5D-Y-3L self-report version has been validated in JIA, but the validity of EQ-5D-Y-5L remains unknown. We examined the psychometric properties of the EQ-5D-Y-5L parent-proxy version among children with JIA. METHODS: We used data from the Understanding Childhood Arthritis Network Canadian-Dutch collaboration study cohort, including patients with new-onset JIA, and those starting or stopping biologics. Clinical data and the parent-proxy version of the childhood health assessment questionnaire (CHAQ) and EQ-5D-Y-5L were collected. We evaluated the ceiling and floor effect; convergent and divergent validity using Spearman's rank correlation; known-group validity using one-way ANOVA (Analysis of Variance) and effect size; and informativity using Shannon's evenness index. RESULTS: 467 patient visits representing 407 patients were analyzed. The EQ-5D-Y-5L had no ceiling/floor effect. The EQ-5D-Y-5L showed good convergent (e.g., EQ-5D-Y-5L pain/discomfort dimension vs. CHAQ pain index (Spearman's r = 0.74, 95% confidence interval (C.I.): 0.69-0.79)), divergent (e.g., EQ-5D-Y-5L pain/discomfort dimension vs. CHAQ eating dimension (Spearman's r = 0.19, 95% C.I.: 0.09-0.29)) and known-group validity (e.g., mean EQ-5D-Y-5L level summary score for patients with inactive versus active disease status, 6.34 vs. 10.52 (p < 0.001, effect size = 1.20 (95% C.I.: 0.95-1.45)). Shannon's evenness index ranged from 0.52 to 0.88, suggesting most dimensions had relatively even distributions. CONCLUSIONS: In this patient sample, EQ-5D-Y-5L parent-proxy version exhibited construct validity and informativity, suggesting the EQ-5D-Y-5L can be used to measure the quality of life of children with JIA.

Juvenile idiopathic arthritis is the most common type of arthritis affecting children. It can cause pain and permanent physical damage to joints and affects mobility and daily activities. While there is no cure yet, new therapies like biologics are effective. However, biologics are expensive and can have side effects. To decide when is the best time to use these biologics, we need to understand their cost and impact on patients. EQ-5D-Y-5L is a common tool to measure how the disease affects a patient's life. It is unclear whether EQ-5D-Y-5L works well for patients with juvenile idiopathic arthritis. In this study, we compared the EQ-5D-Y-5L to another tool that measures how the illness impacts functional ability. We looked to see if the EQ-5D-Y-5L could tell the difference between children who were more or less sick. We also assessed whether the EQ-5D-Y-5L has the ability to describe patients with different severity in health status. This study indicates that the EQ-5D-Y-5L is a good tool to measure the health of patients with juvenile idiopathic arthritis. Findings from this study support the use of the EQ-5D-Y-5L among this patient population in future clinical trials and research studies.

Exploring patient perspectives on EQ-5D-5L data visualization within an individualized decision aid for total knee arthroplasty (TKA) in Alberta, Canada.

BACKGROUND: Decision aids can help patients set realistic expectations. In this study, we explored alternative presentations to visualise patient-reported outcomes (EQ-5D-5L) data within an online, individualized patient decision aid for total knee arthroplasty (TKA) that, in part, generates individualized comparisons based on age, sex and body mass index, to enhance usability prior to implementation into routine clinical practice. METHODS: We used data visualization techniques to modify the presentation of EQ-5D-5L outcomes data within the decision aid. The EQ-5D-5L data was divided into two parts allowing patients to compare themselves to similar individuals (1) pre-surgery and (2) 1-year post-surgery. We created 2 versions for each part and sought patient feedback on comprehension, usefulness, and visual appeal. Patients from an urban orthopedic clinic were recruited and their ratings and comments were recorded using a researcher-administered checklist. Data were managed using Microsoft Excel, R version 3.6.1 and ATLAS.ti V8 and analyzed using descriptive statistics and directed content analysis. RESULTS: A total of 24 and 25 patients participated in Parts 1 and 2, respectively. Overall, there was a slight preference for Version 1 in Part 1 (58.3%) and Version 2 in Part 2 (64%). Most participants demonstrated adequate comprehension for all versions (range 50-72%) and commented that the instructions were clear. While 50-60% of participants rated the content as useful, including knowing the possible outcomes of surgery, some participants found the information interesting only, were unsure how to use the information, or did not find it useful because they had already decided on a treatment. Participants rated visual appeal for all versions favorably but suggested improvements for readability, mainly larger font and image sizes and enhanced contrast between elements. CONCLUSIONS: Based on the results, we will produce an enhanced presentation of EQ-5D-5L data within the decision aid. These improvements, along with further usability testing of the entire decision aid, will be made before implementation of the decision aid in routine clinical practice. Our results on patients' perspectives on the presentation of EQ-5D-5L data to support decision making for TKA treatments contributes to the knowledge on EQ-5D-5L applications within healthcare systems for clinical care.

Meditation and Yoga for Irritable Bowel Syndrome: A Randomized Clinical Trial.

INTRODUCTION: Delivered in person, yoga is effective in managing irritable bowel syndrome (IBS) symptoms. The evidence for efficacy, feasibility, and safety of virtually delivered yoga for patients with IBS is unknown. METHODS: Adults diagnosed with IBS were randomized to either Hatha yoga intervention of 8 weekly online classes delivered virtually or an advice-only control group and assessed at baseline and postintervention. We used an unadjusted ANOVA to determine differences between and within groups on the primary outcome (decrease of ≥50 points in IBS Symptom Severity Scale [IBS-SSS]) and secondary outcomes (quality of life, anxiety and depression, fatigue, somatic symptoms, perceived stress, COVID-19 stress, and self-compassion). We assessed feasibility through recruitment and attrition rates, adherence, participant satisfaction, and safety (i.e., adverse events). RESULTS: Seventy-nine people participated (mean age 45.4 years [SD = 14.0], 92% women, 20% attrition rate). IBS-SSS decreased significantly in the treatment group (Δ change = 54.7, P = 0.028), but not in the control group (Δ change = 22.6, P = 0.277). Fourteen patients (37%) in the yoga group reached a clinically relevant decrease of ≥50 points on the IBS-SSS postintervention compared with 8 patients (20%) in the control group ( P = 0.242). No significant difference was found between groups in IBS-SSS score postintervention ( P = 0.149), but significant differences in favor of the treatment group for quality of life ( P = 0.030), fatigue ( P = 0.035), and perceived stress ( P = 0.040) were identified. The yoga program demonstrated feasibility. Intention to practice yoga decreased significantly in both groups from baseline to postintervention ( P < 0.001). However, the decline in intention did not correlate with practice minutes. DISCUSSION: Virtually delivered yoga is safe and feasible, and effective in reducing IBS symptoms. Based on the primary end point, the intervention was not superior to an advice-only control group.

Pharmacological treatment patterns in patients with juvenile idiopathic arthritis in the Netherlands: a real-world data analysis.

OBJECTIVE: To investigate medication prescription patterns among children with JIA, including duration, sequence and reasons for medication discontinuation. METHODS: This study is a single-centre, retrospective analysis of prospective data from the electronic medical records of JIA patients receiving systemic therapy aged 0-18 years between 1 April 2011 and 31 March 2019. Patient characteristics (age, gender, JIA subtype) and medication prescriptions were extracted and analysed using descriptive statistics, Sankey diagrams and Kaplan-Meier survival methods. RESULTS: Over a median of 4.2 years follow-up, the 20 different medicines analysed were prescribed as monotherapy (n = 15) or combination therapy (n = 48 unique combinations) among 236 patients. In non-systemic JIA, synthetic DMARDs were prescribed to almost all patients (99.5%), and always included MTX. In contrast, 43.9% of non-systemic JIA patients received a biologic DMARD (mostly adalimumab or etanercept), ranging from 30.9% for oligoarticular persistent ANA-positive JIA, to 90.9% for polyarticular RF-positive JIA. Among systemic JIA, 91.7% received a biologic DMARD (always including anakinra). When analysing medication prescriptions according to their class, 32.6% involved combination therapy. In 56.8% of patients, subsequent treatment lines were initiated after unsuccessful first-line treatment, resulting in 68 unique sequences. Remission was the most common reason for DMARD discontinuation (44.7%), followed by adverse events (28.9%) and ineffectiveness (22.1%). CONCLUSION: This paper reveals the complexity of pharmacological treatment in JIA, as indicated by: the variety of mono- and combination therapies prescribed, substantial variation in medication prescriptions between subtypes, most patients receiving two or more treatment lines, and the large number of unique treatment sequences.

A Roadmap for Increasing the Usefulness and Impact of Patient-Preference Studies in Decision Making in Health: A Good Practices Report of an ISPOR Task Force.

Many qualitative and quantitative methods are readily available to study patient preferences in health. These methods are now being used to inform a wide variety of decisions, and there is a growing body of evidence showing studies of patient preferences can be used for decision making in a wide variety of contexts. This ISPOR Task Force report synthesizes current good practices for increasing the usefulness and impact of patient-preference studies in decision making. We provide the ISPOR Roadmap for Patient Preferences in Decision Making that invites patient-preference researchers to work with decision makers, patients and patient groups, and other stakeholders to ensure that studies are useful and impactful. The ISPOR Roadmap consists of 5 key elements: (1) context, (2) purpose, (3) population, (4) method, and (5) impact. In this report, we define these 5 elements and provide good practices on how patient-preference researchers and others can actively contribute to increasing the usefulness and impact of patient-preference studies in decision making. We also present a set of key questions that can support researchers and other stakeholders (eg, funders, reviewers, readers) to assess efforts that promote the ongoing impact (both intended and unintended) of a particular preference study and additional studies in the future.

Patient acceptable symptom state (PASS): thresholds for the EQ-5D-5L and Oxford hip and knee scores for patients with total hip and knee replacement.

PURPOSE: To define patient acceptable symptom state (PASS) cut-off values for the EQ-5D-5L and Oxford hip (OHS) and knee (OKS) scores 6 and 12 months after total hip (THR) or knee (TKR) replacement. To compare PASS cut-off values for the EQ-5D-5L scored using: (1) the Canadian value set, (2) the crosswalk value set, and (3) the equal weighted Level Sum Score (LSS). METHODS: We mailed questionnaires to consecutive patients following surgeon referral for primary THR or TKR and at 6 and 12 months post-surgery. Patient reported outcome measures (PROMs) were the EQ-5D-5L, the OHS, and OKS. We assessed PASS cut-off values for PROMs using percentile and ROC methods, with the Youden Index. RESULTS: Five hundred forty-two surgical patients (mean age, 64 years, 57% female, 49% THR) completed baseline and 12-month questionnaires. 89% of THR and 81% of TKR patients rated PASS as acceptable at 12 months. PASS cut-off values for THR for the EQ-5D-5L (Canadian) were 0.85 (percentile) and 0.84 (Youden) at 12 months. Cut-off values were similar for the LSS (0.85 and 0.85) and lower for the crosswalk value set (0.74 and 0.73), respectively. EQ-5D-5L cut-off values for TKR were Canadian, 0.77 (Percentile) and 0.78 (Youden), LSS, 0.75 and 0.80, and crosswalk, 0.67 and 0.74, respectively. Cut-off values 6 and 12 months post-surgery ranged from 38 to 39 for the OHS, and 28 to 36 for the OKS (range 0 worst to 48 best). CONCLUSION: PASS cut-off values for the EQ-5D-5L and Oxford scores varied, not only between methods and timing of assessment, but also by different EQ-5D-5L value sets, which vary between countries. Because of this variation, PASS cut-off values are not necessarily generalizable to other populations of TJR patients. We advise caution in interpreting PROMs when using EQ-5D-5L PASS cut-off values developed in different countries. A standardization of methods is needed before published cut-off values can be used with confidence in other populations.

Barriers experienced by families new to Alberta, Canada when accessing routine-childhood vaccinations.

BACKGROUND: As Canada and other high-income countries continue to welcome newcomers, we aimed to 1) understand newcomer parents' attitudes towards routine-childhood vaccinations (RCVs), and 2) identify barriers newcomer parents face when accessing RCVs in Alberta, Canada. METHODS: Between July 6th-August 31st, 2022, we recruited participants from Alberta, Canada to participate in moderated focus group discussions. Inclusion criteria included parents who had lived in Canada for < 5 years with children < 18 years old. Focus groups were transcribed verbatim and analyzed using content and deductive thematic analysis. The capability opportunity motivation behaviour model was used as our conceptual framework. RESULTS: Four virtual and three in-person focus groups were conducted with 47 participants. Overall, parents were motivated and willing to vaccinate their children but experienced several barriers related to their capability and opportunity to access RCVs. Five main themes emerged: 1) lack of reputable information about RCVs, 2) language barriers when looking for information and asking questions about RCVs, 3) lack of access to a primary care provider (PCP), 4) lack of affordable and convenient transportation options, and 5) due to the COVID-19 pandemic, lack of available vaccine appointments. Several minor themes were also identified and included barriers such as lack of 1) childcare, vaccine record sharing, PCP follow-up. CONCLUSIONS: Our findings highlight that several barriers faced by newcomer families ultimately stem from issues related to accessing information about RCVs and the challenges families face once at vaccination clinics, highlighting opportunities for health systems to better support newcomers in accessing RCVs.

The complexity of diagnosing rare disease: An organizing framework for outcomes research and health economics based on real-world evidence.

PURPOSE: To facilitate robust economic analyses of clinical exome and genome sequencing, this study was taken up with the objective of establishing a framework for organizing diagnostic testing trajectories for patients with rare disease. METHODS: We collected diagnostic investigations-related data before exome sequencing from the medical records of 228 cases. Medical geneticist experts participated in a consensus building process to develop the SOLVE Framework for organizing the complex range of observed tests. Experts categorized tests as indicator or nonindicator tests on the basis of their specificity for diagnosing rare diseases. Face validity was assessed using case vignettes. RESULTS: Most cases had symptom onset at birth (42.5%) or during childhood (43.4%) and had intellectual disability (73.3%). On average, the time spent seeking a diagnosis before sequencing was 1989 days (SD = 2137) and included 16 tests (SD = 14). Agreement across experts on test categories ranged from 83% to 96%. The SOLVE Framework comprised observed tests, including 186 indicator and 39 nonindicator tests across cytogenetic/molecular, biochemical, imaging, electrical, and pathology test categories. CONCLUSION: Real-world diagnostic testing data can be ascertained and organized to reflect the complexity of the journey of the patients with rare diseases. SOLVE Framework will improve the accuracy and certainty associated with value-based assessments of genomic sequencing.

A Simulation Modelling Study of Referral Distribution Policies in a Centralized Intake System for Surgical Consultation.

Delays beyond recommended wait times, especially for specialist services, are associated with adverse health outcomes. The Alberta Surgical Initiative aims to improve the referral wait time-the time between a referral is received at the central intake to the time a specialist sees the patient. Using the discrete event simulation modelling approach, we evaluated and compared the impact of four referral distribution policies in a central intake system on three system performance measures (number of consultations, referral wait time and surgeon utilization). The model was co-designed with clinicians and clinic staff to represent the flow of patients through the system. We used data from the Facilitated Access to Surgical Treatment (FAST) centralized intake referral program for General Surgery to parameterize the model. Four distribution policies were evaluated - next-available-surgeon, sequential, "blackjack," and "kanban." A sequential distribution of referrals for surgical consultation among the surgeons resulted in the worst performance in terms of the number of consultations, referral wait time and surgeon utilization. The three other distribution policies are comparable in performance. The "next available surgeon" model provided the most efficient and robust model, with approximately 1,000 more consultations, 100 days shorter referral time and a 14% increase in surgeon utilization. Discrete event simulation (DES) modelling can be an effective tool to illustrate and communicate the impact of the referral distribution policy on system performance in terms of the number of consultations, referral wait time and surgeon utilization.

(Un)standardized testing: the diagnostic odyssey of children with rare genetic disorders in Alberta, Canada.

PURPOSE: We provide a description of the diagnostic odyssey for a cohort of children seeking diagnosis of a rare genetic disorder in terms of the time from initial consultation to most recent visit or receipt of diagnosis, the number of tests per patient, and the types of tests received. METHODS: Retrospective chart review of 299 children seen at the Alberta Children's Hospital (ACH) Genetics Clinic (GC) for whom the result of at least one single-gene test, gene panel, or chromosome microarray analysis (CMA) was recorded. RESULTS: Of 299 patients, 90 (30%) received a diagnosis in the period of the review. Patients had an average of 5.4 tests each; 236 (79%) patients received CMA; 172 (58%) patients received single-gene tests and 34 (11%) received gene panels; 167 (56%) underwent imaging/electrical activity studies. The mean observation period was 898 days (95% confidence interval [CI] 791, 1004). Among patients with visits recorded prior to visiting ACH GC, 43% of the total observation time occurred prior to the GC. CONCLUSION: As genomic technologies expand, the nature of the diagnostic odyssey will change. This study has outlined the current standard of care in the ACH GC, providing a baseline against which future changes can be assessed.

Incomplete information and irrelevant attributes in stated-preference values for health interventions.

Violations of the assumptions of complete information [CI] and independence of irrelevant alternatives (IIA) in discrete-choice experiment (DCE) data imply sensitivity of preference estimates to the decision context and the alternatives evaluated. There is a paucity of evidence on how these two assumptions affect health-preference results and whether the usual specifications of random-parameters logit models are sufficient to address these violations. We assessed the appropriateness of these assumptions in a DCE valuating interventions to prevent long-term health problems that could be identified through whole genome sequencing. A DCE survey was administered to members of a nationally representative consumer panel to elicit their preferences for options to reduce the risk of health problems. The treatment options presented (surgery, medication, and watchful waiting) and the context for the decisions elicited (severity and likelihood of the health problem) were varied experimentally to evaluate the sensitivity of preference results to such changes. We find evidence of IIA violations as the options presented to prevent health changed. Our results also are consistent with the expectation that additional substitutes decrease the monetized value of alternatives. We also find some evidence that the decision context can moderate such effects, which constitutes a new finding.

Attitudes, behaviours and barriers to public health measures for COVID-19: a survey to inform public health messaging.

BACKGROUND: Public support of public health measures including physical distancing, masking, staying home while sick, avoiding crowded indoor spaces and contact tracing/exposure notification applications remains critical for reducing spread of COVID-19. The aim of our work was to understand current behaviours and attitudes towards public health measures as well as barriers individuals face in following public health measures. We also sought to identify attitudes persons have regarding a COVID-19 vaccine and reasons why they may not accept a vaccine. METHODS: A cross-sectional online survey was conducted in August 2020, in Alberta, Canada in persons 18 years and older. This survey evaluated current behaviours, barriers and attitudes towards public health measures and a COVID-19 vaccine. Cluster analysis was used to identify key patterns that summarize data variations among observations. RESULTS: Of the 60 total respondents, the majority of persons were always or often physically distancing (73%), masking (65%) and staying home while sick (67%). Bars/pubs/lounges or nightclubs were visited rarely or never by 63% of respondents. Persons identified staying home while sick to provide the highest benefit (83%) in reducing spread of COVID-19. There were a large proportion of persons who had not downloaded or used a contact tracing/exposure notification app (77%) and who would not receive a COVID-19 vaccine when available (20%) or were unsure (12%). Reporting health authorities as most trusted sources of health information was associated with greater percentage of potential uptake of vaccine but not related to contact tracing app download and use. Individuals with lower concern of getting and spreading COVID-19 showed the least uptake of public health measures except for avoiding public places such as bars. Lower concern regarding COVID-19 was also associated with more negative responses to taking a potential COVID-19 vaccine. CONCLUSION: These results suggest informational frames and themes focusing on individual risks, highlighting concern for COVID-19 and targeting improving trust for health authorities may be most effective in increasing public health measures. With the ultimate goal of preventing spread of COVID-19, understanding persons' attitudes towards both public health measures and a COVID-19 vaccine remains critical to addressing barriers and implementing targeted interventions and messaging to improve uptake.

Addressing Challenges of Economic Evaluation in Precision Medicine Using Dynamic Simulation Modeling.

OBJECTIVES: The objective of this article is to describe the unique challenges and present potential solutions and approaches for economic evaluations of precision medicine (PM) interventions using simulation modeling methods. METHODS: Given the large and growing number of PM interventions and applications, methods are needed for economic evaluation of PM that can handle the complexity of cascading decisions and patient-specific heterogeneity reflected in the myriad testing and treatment pathways. Traditional approaches (eg, Markov models) have limitations, and other modeling techniques may be required to overcome these challenges. Dynamic simulation models, such as discrete event simulation and agent-based models, are used to design and develop mathematical representations of complex systems and intervention scenarios to evaluate the consequence of interventions over time from a systems perspective. RESULTS: Some of the methodological challenges of modeling PM can be addressed using dynamic simulation models. For example, issues regarding companion diagnostics, combining and sequencing of tests, and diagnostic performance of tests can be addressed by capturing patient-specific pathways in the context of care delivery. Issues regarding patient heterogeneity can be addressed by using patient-level simulation models. CONCLUSION: The economic evaluation of PM interventions poses unique methodological challenges that might require new solutions. Simulation models are well suited for economic evaluation in PM because they enable patient-level analyses and can capture the dynamics of interventions in complex systems specific to the context of healthcare service delivery.

Criteria and Process for Initiating and Developing an ISPOR Good Practices Task Force Report.

The International Society for Pharmacoeconomics and Outcomes Research (ISPOR)'s "Good Practices Task Force" reports are highly cited, multistakeholder perspective expert guidance reports that reflect international standards for health economics and outcomes research (HEOR) and their use in healthcare decision making. In this report, we discuss the criteria, development, and evaluation/consensus review and approval process for initiating a task force. The rationale for a task force must include a justification, including why this good practice guidance is important and its potential impact on the scientific community. The criteria include: (1) necessity (why is this task force required?); (2) a methodology-oriented focus (focus on research methods, approaches, analysis, interpretation, and dissemination); (3) relevance (to ISPOR's mission and its members); (4) durability over time; (5) broad applicability; and 6) an evidence-based approach. In addition, the proposal must be a priority specifically for ISPOR. These reports are valuable to researchers, academics, students, health technology assessors, medical technology developers and service providers, those working in other commercial entities, regulators, and payers. These stakeholder perspectives are represented in task force membership to ensure the report's overall usefulness and relevance to the global ISPOR membership. We hope that this discussion will bring transparency to the process of initiating, approving, and producing these task force reports and encourage participation from a diverse range of experts within and outside ISPOR.

Patient expectations and satisfaction 6 and 12 months following total hip and knee replacement.

PURPOSE: To assess (1) patient expectations before total hip (THR) and knee (TKR) replacement; (2) which expectations are met and unmet 6 and 12 months post-surgery; (3) the role of unmet expectations in satisfaction. METHODS: Questionnaires were mailed to consecutive patients following surgeon referral for primary THR or TKR. Patients listed their own expectations and also completed the Hospital for Special Surgery (HSS) Expectation Survey. We used content analysis to group expectations into themes. At 6 and 12 months post-surgery, patients were given a copy of their own list of individual expectations and reassessed each one as met or unmet. We also assessed fulfilled HSS expectations and satisfaction with surgery. RESULTS: The sample of 556 patients (49% THR, 57% female) had a mean age of 64 years (SD10). The five most frequent expectation themes were pain relief, mobility, walking, physical activities, and daily activities. Of these, physical activities had the lowest percentage met 12 months post-surgery. 95% (THR) and 87% (TKR) were satisfied/very satisfied with their surgery 12 months post-surgery. Very satisfied patients had a significantly greater percentage of met expectations (96% THR; 92% TKR) than dissatisfied patients (42% THR; 12% TKR). Although most expectations listed by patients were included in the HSS surveys, some were not, particularly for TKR. From 6 to 12 months, there was a significant increase in patient satisfaction for self-care, daily activities, and met expectations for THR and pain relief, self-care, daily activities, and recreational activities for TKR. CONCLUSIONS: Expectations should be explicitly addressed before surgery, including a discussion of realistic expectations, particularly for physical activities.

Geospatial patterns of comorbidity prevalence among people with osteoarthritis in Alberta Canada.

BACKGROUND: Knowledge of geospatial pattern in comorbidities prevalence is critical to an understanding of the local health needs among people with osteoarthritis (OA). It provides valuable information for targeting optimal OA treatment and management at the local level. However, there is, at present, limited evidence about the geospatial pattern of comorbidity prevalence in Alberta, Canada. METHODS: Five administrative health datasets were linked to identify OA cases and comorbidities using validated case definitions. We explored the geospatial pattern in comorbidity prevalence at two standard geographic areas levels defined by the Alberta Health Services: descriptive analysis at rural-urban continuum level; spatial analysis (global Moran's I, hot spot analysis, cluster and outlier analysis) at the local geographic area (LGA) level. We compared area-level indicators in comorbidities hotspots to those in the rest of Alberta (non-hotspots). RESULTS: Among 359,638 OA cases in 2013, approximately 60% of people resided in Metro and Urban areas, compared to 2% in Rural Remote areas. All comorbidity groups exhibited statistically significant spatial autocorrelation (hypertension: Moran's I index 0.24, z score 4.61). Comorbidity hotspots, except depression, were located primarily in Rural and Rural Remote areas. Depression was more prevalent in Metro (Edmonton-Abbottsfield: 194 cases per 1000 population, 95%CI 192-195) and Urban LGAs (Lethbridge-North: 169, 95%CI 168-171) compared to Rural areas (Fox Creek: 65, 95%CI 63-68). Comorbidities hotspots included a higher percentage of First Nations or Inuit people. People with OA living in hotspots had lower socioeconomic status and less access to care compared to non-hotspots. CONCLUSIONS: The findings highlight notable rural-urban disparities in comorbidities prevalence among people with OA in Alberta, Canada. Our study provides valuable evidence for policy and decision makers to design programs that ensure patients with OA receive optimal health management tailored to their local needs and a reduction in current OA health disparities.

Adherence to human epidermal growth factor receptor-2 testing and adjuvant trastuzumab treatment guidelines in Ontario.

OBJECTIVES: We evaluated adherence of human epidermal growth factor receptor-2 testing using immunohistochemistry and fluorescence in situ hybridization, as well as adjuvant trastuzumab treatment according to Canadian guidelines, and predictors of trastuzumab use in early-stage breast cancer in Ontario. METHODS: Retrospective cohort of early-stage breast cancer patients identified in the Ontario Cancer Registry. Human epidermal growth factor receptor-2 test type, sequence, result(s), tumor grade, and hormone receptor status were abstracted from Ontario Cancer Registry pathology reports. Trastuzumab treatment was determined from provincial cancer agency records. Other variables were determined from administrative data sources. Logistic regression models were used to estimate adjusted odds ratios for factors associated with guideline adherence. RESULTS: The first human epidermal growth factor receptor-2 test result was the strongest predictor of confirmatory testing (p < 0.05). Human epidermal growth factor receptor-2 testing by immunohistochemistry accounted for the majority of documented first tests (94%; n = 8249). Overall, 27% (n = 2360) of tested patients received a second test by fluorescence in situ hybridization (46%) or immunohistochemistry (49%) assay. Most human epidermal growth factor receptor-2 equivocal patients (89%; n = 784) received a confirmatory test. Among human epidermal growth factor receptor-2-positive patients, only 57% (n = 385) received trastuzumab treatment within the study period. Human epidermal growth factor receptor-2 status was the strongest predictor of trastuzumab use. Younger patients (<70 years at diagnosis) and negative hormone receptor status had higher odds of trastuzumab treatment (p < 0.05) compared to older and positive hormone receptor status patients. CONCLUSIONS: Immunohistochemistry use as a first test was largely consistent with Canadian guidelines; however, immunohistochemistry was frequently used as a confirmatory test, which is not guideline-concordant. Monitoring these testing and treating patterns is necessary to optimize health outcomes associated with trastuzumab.

The value of diagnostic testing for parents of children with rare genetic diseases.

PURPOSE: Exome sequencing (ES) can rapidly identify disease-causing variants responsible for rare, single-gene diseases, and potentially reduce the duration of the diagnostic odyssey. Our study examines how parents and families value ES. METHODS: We developed a discrete choice experiment (DCE) survey that was administered to parents of children with rare diseases. The DCE included 14 choice tasks with 6 attributes and 3 alternatives. A valuation-space model was used to estimate willingness to pay, willingness to wait for test results, and minimum acceptable chance of a diagnosis for changes in each attribute. RESULTS: There were n = 319 respondents of whom 89% reported their child had genetic testing, and 66% reported their child had a diagnosis. Twenty-six percent reported that their child had been offered ES. Parents were willing to pay CAD$6590 (US$4943), wait 5.2 years to obtain diagnostic test results, and accept a reduction of 3.1% in the chance of a diagnosis for ES compared with operative procedures. CONCLUSION: Timely access to ES could reduce the diagnostic odyssey and associated costs. Before ES is incorporated routinely into care for patients with rare diseases in Canada and more broadly, there must be a clear understanding of its value to patients and families.