The use of EQ-5D-5L as a patient-reported outcome measure in evaluating community rehabilitation services in Alberta, Canada.

BACKGROUND: The purpose of this study was to describe the characteristics and health-related quality of life (HRQL) of patients accessing community rehabilitation services in Alberta, Canada, using routinely collected EQ-5D-5L data, and explore factors associated with the impact of these services. METHODS: A retrospective, longitudinal, observational design was used. Patients completed the EQ-5D-5L and demographic questions at intake and end of rehabilitation care. Change in EQ-5D-5L dimensions from intake until end of rehabilitation was examined using the Pareto Classification of Health Change. Change scores were calculated for the EQ-5D-5L index, VAS, and total sum scores. Change groups in the EQ-5D-5L index and VAS scores, were defined by minimally important differences of 0.04 and 7.0, respectively. One level change was considered important for the total sum score. Effect size of the change in index, VAS, and total sum scores was also examined. Chi-squared tests were conducted to examine whether change in EQ-5D-5L varied by age, gender, region, and having anxiety/depression at intake. RESULTS: Three service programs were examined; pulmonary rehabilitation (n = 542), group-based community exercise (n = 463), and physiotherapy for bone and joint care (n = 391). At intake, HRQL in all programs was lower than that of the general Alberta population norms and improved by end of rehabilitation. The mean (SD) change in index, VAS, and total sum scores were 0.02 (0.13), 6.0 (18.3), and - 0.5 (2.4) in pulmonary rehabilitation, 0.06 (0.13), 6.6 (18.7), - 1.2 (2.4) in community exercise, and 0.13 (0.16), 1.2 (0.9), and - 2.8 (2.8) in physiotherapy, respectively. Based on change of the index score, 24% deteriorated, 38% improved, and 38% had no change in pulmonary rehabilitation; 17% deteriorated, 51% improved, and 32% had no change in community exercise; 5% deteriorated, 72% improved, and 23% had no change in physiotherapy. Similar trends were seen in the VAS and total sum scores. Older age, urban region, and having anxiety/depression at intake were associated with positive change in EQ-5D-5L. CONCLUSIONS: The results of this study are intended to inform program/service level decisions by describing the characteristics and HRQL of patients accessing community rehabilitation, as well as the predictors of change in health status, which will help direct future program growth and service changes.

Mapping the Edmonton Symptom Assessment System-Revised: Renal to the EQ-5D-5L in patients with chronic kidney disease.

PURPOSE: The Edmonton Symptom Assessment System-Revised: Renal (ESAS-r: Renal) is a disease-specific patient-reported outcome measure (PROM) that assesses symptoms common in chronic kidney disease (CKD). There is no preference-based scoring system for the ESAS-r: Renal or a mapping algorithm to predict health utility values. We aimed to develop a mapping algorithm from the ESAS-r: Renal to the Canadian EQ-5D-5L index scores. METHODS: We used data from a multi-centre cluster randomized-controlled trial of the routine measurement and reporting of PROMs in hemodialysis units in Northern Alberta, Canada. In two arms of the trial, both the ESAS-r: Renal and the EQ-5D-5L were administered to CKD patients undergoing hemodialysis. We used data from one arm for model estimation, and data from the other for validation. We explored direct and indirect mapping models; model selection was based on statistical fit and predictive power. RESULTS: Complete data were available for 506 patient records in the estimation sample and 242 in the validation sample. All models tended to perform better in patients with good health, and worse in those with poor health. Generalized estimating equations (GEE) and generalized linear model (GLM) on selected ESAS-r: Renal items were selected as final models as they fitted the best in estimation and validation sample. CONCLUSION: When only ESAS-r: Renal data are available, one could use GEE and GLM to predict EQ-5D-5L index scores for use in economic evaluation. External validation on populations with different characteristics is warranted, especially where renal-specific symptoms are more prevalent.

The performance of the EQ-5D-3L in screening for anxiety and depressive symptoms in hospital and community settings.

BACKGROUND: To examine the performance of the EQ-5D-3L in screening for anxiety and depressive symptoms in hospital and community settings compared to other patient-reported screening tools. METHODS: Data from a prospective cohort of patients discharged from general internal medicine wards from two hospitals in Edmonton, Alberta were used in this study. Two waves of measurements (discharge and 90-days post-discharge) were analyzed. The performance of the EQ-5D-3L was compared to other self-report screening tools: Generalized Anxiety Disorder 2-item questionnaire was used to categorize anxiety symptoms into absent (< 3) and present (≥ 3), and the Patient Health Questionnaire 9-items was used to categorize depressive symptoms by two severity cut-points: no (< 10) vs. mild (≥ 10), and no (< 15) vs. moderate-severe (≥ 15). Performance of EQ-5D-3L in screening for anxiety and depressive symptoms was evaluated using receiver operating curve (ROC) analysis. RESULTS: Average age of participants (n = 493) was 62.9 years (SD 18.6); 51% were female. At discharge, 30.0% screened positive for mild and 12.8% for moderate-severe depressive symptoms, while 27.6% screened positive for anxiety symptoms. For co-morbid symptoms, 17.1% screened positive for anxiety and any depressive symptoms, while 10.8% for anxiety and moderate-severe depressive symptoms. While the EQ-5D-3L had limited screening ability in hospital, the anxiety/depression dimension performed well in the community setting (90-days post-discharge) in screening for anxiety (area under ROC 0.79), depressive symptoms (any: 0.78, moderate-severe: 0.84), and a combination of both (any: 0.86; moderate-severe: 0.91). CONCLUSIONS: The EQ-5D-3L anxiety/depression dimension could be a useful tool in screening for anxiety and depressive symptoms in community settings compared to other self-report screening tools. The usefulness of the EQ-5D-3L as a screening tool in other settings and populations is warranted.

A cluster randomized controlled trial for the Evaluation of routinely Measured PATient reported outcomes in HemodialYsis care (EMPATHY): a study protocol.

BACKGROUND: Kidney failure requiring dialysis is associated with poor health outcomes and health-related quality of life (HRQL). Patient-reported outcome measures (PROMs) capture symptom burden, level of functioning and other outcomes from a patient perspective, and can support clinicians to monitor disease progression, address symptoms, and facilitate patient-centered care. While evidence suggests the use of PROMs in clinical practice can lead to improved patient experience in some settings, the impact on patients' health outcomes and experiences is not fully understood, and their cost-effectiveness in clinical settings is unknown. This study aims to fill these gaps by evaluating the effectiveness and cost-effectiveness of routinely measuring PROMs on patient-reported experience, clinical outcomes, HRQL, and healthcare utilization. METHODS: The EMPATHY trial is a pragmatic multi-centre cluster randomized controlled trial that will implement and evaluate the use of disease-specific and generic PROMs in three kidney care programs in Canada. In-centre hemodialysis units will be randomized into four groups, whereby patients: 1) complete a disease-specific PROM; 2) complete a generic PROM; 3) complete both types of PROMs; 4) receive usual care and do not complete any PROMs. While clinical care pathways are available to all hemodialysis units in the study, for the three active intervention groups, the results of the PROMs will be linked to treatment aids for clinicians and patients. The primary outcome of this study is patient-provider communication, assessed by the Communication Assessment Tool (CAT). Secondary outcomes include patient management and symptoms, use of healthcare services, and the costs of implementing this intervention will also be estimated. The present protocol fulfilled the Standard Protocol Items: Recommendations for Intervention Trials (SPIRIT) checklist. DISCUSSION: While using PROMs in clinical practice is supported by theory and rationale, and may engage patients and enhance their role in decisions regarding their care and outcomes, the best approach of their use is still uncertain. It is important to rigorously evaluate such interventions and investments to ensure they provide value for patients and health systems. TRIAL REGISTRATION: Protocol version (1.0) and trial registration data are available on www.clinicaltrials.gov , identifier: NCT03535922 , registered May 24, 2018.

The relationship of neighbourhood-level material and social deprivation with health-related quality of life.

PURPOSE: To examine the relationship of neighbourhood-level material and social deprivation with health-related quality of life, measured by the EQ-5D-5L, in the general adult population. METHODS: A sample of 11,835 adults living in Alberta, Canada was drawn from three combined annual Health Quality Council of Alberta Satisfaction and Experiences with Health Care Services surveys from 2012 to 2016. Neighbourhood-level material and social deprivation indices were derived using the Pampalon index and the 2006 Canadian census. The EQ-5D-5L dimensions, index and VAS scores were compared across the deprivation indices quintiles in the overall sample and by participants' sub-groups. Differences were tested using ANOVA or Chi-square test as appropriate. Multivariate linear regression models were conducted to examine the independent association of material and social deprivation with the EQ-5D-5L index and VAS scores, and multinomial logistic regression models with each of the EQ-5D-5L dimensions. RESULTS: Respondents in higher material or social deprivation categories had lower EQ-5D-5L index and VAS scores than those in the least deprived categories. Additionally, respondents with higher material deprivation were more likely to report problems on mobility, usual activities, and pain/discomfort; those with higher social deprivation were more likely to report problems on mobility, self-care, usual activities, and anxiety/depression. CONCLUSION: Higher neighbourhood-level material and social deprivation is significantly associated with lower health-related quality of life in the general adult population. Examining the factors leading to this inequity in health between individuals living in the least and most deprived neighbourhoods is imperative to mitigating these inequities.

How the Routine Use of Patient-Reported Outcome Measures for Hemodialysis Care Influences Patient-Clinician Communication: A Mixed-Methods Study.

BACKGROUND AND OBJECTIVES: Patient-reported outcome measures invite patients to self-report aspects of their quality of life and have been reported to enhance communication with clinicians. We aimed to examine how routine use of patient-reported outcome measures in in-center hemodialysis units influenced patient-clinician communication. DESIGN, SETTING, PARTICIPANTS, & MEASUREMENTS: A concurrent, longitudinal, mixed-methods approach was used. We used data from a cluster randomized controlled trial of 17 hemodialysis units in northern Alberta that introduced a patient-reported outcome measures intervention. Patient-clinician communication was assessed using a modified Communication Assessment Tool. Using interpretive description, we explored patients' and nurses' perceptions of communication pertaining to routine patient-reported outcome measure use. Through purposeful sampling, we interviewed ten patients and eight nurses and conducted six observations in the dialysis units, which were documented in field notes. We reviewed 779 patient responses to open-ended survey questions from randomized controlled trial data. Qualitative data were thematically analyzed. RESULTS: Overall, patient-reported outcome measure use did not substantively improve patient-clinician communication. There was a small positive change in mean total Communication Assessment Tool scores (range, 1-5) from baseline to 12 months in patient-reported outcome measure use units (0.25) but little difference from control group units that did not use patient-reported outcome measures (0.21). The qualitative findings provide in-depth insights into why patient-reported outcome measure use did not improve patient-clinician communication. The purpose of patient-reported outcome measure use was not always understood by patients and clinicians; patient-reported outcome measures were not implemented as originally intended in the trial, despite clinician training; there were challenges using patient-reported outcome measures as a means to communicate; and patient-reported outcome measure use was perceived to have limited value. CONCLUSIONS: While patient-reported outcome measures use did not improve patient-clinician communication, qualitative data suggest implementation challenges, including limited clarity of purpose and perceived limited value, that may have limited the effectiveness of the intervention.

Patient-reported outcome measures in the care of in-centre hemodialysis patients.

Kidney failure requiring dialysis is associated with high symptom burden and low health-related quality of life (HRQL). Patient-reported outcome measures (PROMs) are standardized instruments that capture patients' symptom burden, level of functioning, and HRQL. The routine use of PROMs can be used to monitor aspects of patients' health that may otherwise be overlooked, inform care planning, and facilitate the introduction of treatments. Incorporating PROMs into clinical practice is an appropriate strategy to engage patients and enhance their role in decisions regarding their care and outcomes. However, the implementation of PROMs measurement and associated interventions can be challenging given the nature of clinical practice in busy hemodialysis units, the variations in organization and clinical workflow across units, as well as regional programs. Implementing PROMs and linking these with actionable treatment aids to alleviate bothersome symptoms and improve patients' wellbeing is key to improving patients' health. Other considerations in implementing PROMs within a hemodialysis setting include integration into electronic medical records, purchase and configuration of electronic tools (i.e., tablets), storage and disinfection of such tools, and ongoing IT resources. It is important to train clinicians on the practical elements of using PROMs, however there is also a need to engage clinicians to use PROMs on an ongoing basis. This article describes how PROMs have been implemented at in-centre hemodialysis units in Alberta, Canada, addressing each of these elements.

Burden of mental health symptoms and perceptions of their management in in-centre hemodialysis care: a mixed methods study.

BACKGROUND: We aimed to describe (1) depressive and anxiety symptom burdens reported by adults on in-centre hemodialysis in Northern Alberta, Canada and (2) patients' and nurses' perceptions of managing such symptoms using routine patient-reported outcome measures (PROMs). METHODS: A longitudinal mixed methods approach was employed. Cluster randomized controlled trial data exposed the prevalence of positive screens (scores ≥ 3) for depressive (PHQ-2) and anxiety (GAD-2) symptoms. A descriptive qualitative approach was used to understand patients' and nurses' perceptions of managing these symptoms using the ESAS-r: Renal and EQ-5D-5L. Using purposeful sampling, patients and nurses were invited for interviews. Field notes were documented from 6 dialysis unit observations. Patients' responses to open-ended survey questions and nurses' electronic chart notes related to mental health were compiled. Thematic and content analyses were used. RESULTS: Average age of patients (n = 408) was 64.0 years (SD 15.4), 57% were male, and 87% were not working; 29% screened positive for depressive symptoms, 21% for anxiety symptoms, and 16% for both. From patient (n = 10) and nurse (n = 8) interviews, unit observations, patient survey responses (n = 779) and nurses' chart notes (n = 84), we discerned that PROMs (ESAS-r: Renal/EQ-5D-5L) had the potential to identify and prompt management of mental health concerns. However, opinions differed about whether mental health was within kidney care scope. Nonetheless, participants agreed there was a lack of mental health resources. CONCLUSIONS: Prevalence of depressive and anxiety symptoms aligned with existing literature. Tensions regarding mental health management highlight the need for systemic decisions about how routine PROM use, including mental health assessment, may be optimized to meet patients' needs.

A National Approach to Reimbursement Decision-Making on Drugs for Rare Diseases in Canada? Insights from Across the Ponds.

INTRODUCTION: Regardless of the type of health system or payer, coverage decisions on drugs for rare diseases (DRDs) are challenging. While these drugs typically represent the only active treatment option for a progressive and/or life-threatening condition, evidence of clinical benefit is often limited because of small patient populations and the costs are high. Thus, decisions come with considerable uncertainty and risk. In Canada, interest in developing a pan-Canadian decision-making approach informed by international experiences exists. OBJECTIVE: To develop an inventory of existing policies and processes for making coverage decisions on DRDs around the world. METHODS: A systematic review of published and unpublished documents describing current policies and processes in the top 20 gross domestic product countries was conducted. Bibliographic databases, the Internet and government/health technology assessment organization websites in each country were searched. Two researchers independently extracted information and tabulated it to facilitate qualitative comparative analyses. Policy experts from each country were contacted and asked to review the information collected for accuracy and completeness. RESULTS: Almost all countries have multiple mechanisms through which coverage for a DRD may be sought. However, they typically begin with a review that follows the same process as drugs for more common conditions (i.e., the centralized review process), although specific submission requirements could differ (e.g., no need to submit a cost-effectiveness analysis). When drugs fail to receive a positive recommendation/decision, they are reconsidered by "safety net"-type programs. Eligibility criteria vary across countries, as do the decision options, which may be applied to individual patients or patient groups. CONCLUSIONS: With few exceptions, countries have not created separate centralized review processes for DRDs. Instead, they have modified components of existing mechanisms and added safety nets.

Involving patients in reducing decision uncertainties around orphan and ultra-orphan drugs: a rare opportunity?

INTRODUCTION: Uncertainty influences the amount of risk in decision making, and is typically related to clinical benefit, value for money, affordability, and/or adoption/diffusion of the technology (e.g., drug, device, procedure, etc.). Although evidence-based review processes within each stage of the technology lifecycle have been implemented to minimize uncertainty, high-quality information addressing that related to orphan and ultra-orphan drugs is often unavailable. The role that patients, as experts in their disease, may play in providing such information has yet to be fully explored. OBJECTIVE: The objective of this systematic review was to identify existing and proposed opportunities for patients with rare diseases and their families to provide input aimed at reducing decision uncertainties throughout the lifecycle of an orphan or ultra-orphan drug. METHODS: A comprehensive review of published and gray literature describing roles for patients and families in activities related to orphan and ultra-orphan drugs was conducted. In addition, the websites of regulatory and centralized reimbursement decision-making bodies in the top 22 OECD (Organisation for Economic Cooperation and Development) countries by gross domestic product (GDP) were scanned to identify current opportunities for patients with rare diseases in both stages. The websites of umbrella patient organizations for rare diseases in these countries were also scanned. These roles were then mapped onto a matrix to determine the stage in the technology lifecycle and types of uncertainties they directly or indirectly addressed. RESULTS: Across the 22 countries, nine roles for patients within regulatory related processes were identified, with at least one in each country. These roles were not specific to patients with rare diseases. Similarly, six different opportunities for patient input in centralized drug review processes were identified, all of which applied to patients, in general, rather than just those with rare diseases. 'Real-world' examples of patient involvement explicitly related to rare diseases centered around 11 different themes. Seven fell within the research and development or clinical trial stages of a drug's lifecycle. Of the remaining four, three were associated with education and advocacy. All of the proposed roles identified focused on greater involvement in (1) the design and conduct of clinical trials, or (2) the 'valuation' of evidence during reimbursement decision making. When mapped onto the matrix of decision uncertainties, almost all of the existing and proposed roles addressed 'clinical benefit'. Roles for patients in reducing 'value for money', affordability, or adoption/diffusion uncertainties were mainly indirect, and a result of patient involvement in activities aimed at generating information on clinical benefit, which is then used to inform discussions around these uncertainties. CONCLUSIONS: While patient involvement in activities that directly address uncertainties in clinical benefit may not be 'rare', opportunities for reducing those related to 'value for money', affordability, and adoption/diffusion remain scarce.