Development of a Patient-Reported Experience Measure Tool for Ambulatory Patients With Acute Unexpected Needs: The APEX Questionnaire.

Background: The aim of this study was to develop a patient-reported experience measure (PREM) for comparing the experience of care received by ambulatory patients with acute unexpected needs presenting in emergency departments (EDs), walk-in clinics, and primary care practices. Methods: The Ambulatory Patient EXperience (APEX) questionnaire was developed using a 5-phase mixed-methods approach. The questionnaire was pretested by asking potential users to rate its clarity, usefulness, redundancy, content and face validities, and discrimination on a 9-point scale (1 = strongly disagree to 9 = strongly agree). The pre-final version was then tested in a pilot study. Results: The final questionnaire is composed of 61 questions divided into 7 sections. In the pretest (n = 25), median responses were 8 and above for all dimensions assessed. In the pilot study, 63 participants were enrolled. Adjusted results show that access, cleanliness, and feeling treated with respect and dignity by nurses and physicians were significantly better in the clinics than in the ED. Conclusion: We developed a questionnaire to assess and compare experience of ambulatory care in different clinical settings.

Implementation of an audit and feedback module targeting low-value clinical practices in a provincial trauma quality assurance program: a cost-effectiveness study.

BACKGROUND: Audit and Feedback (A&F) interventions based on quality indicators have been shown to lead to significant improvements in compliance with evidence-based care including de-adoption of low-value practices (LVPs). Our primary aim was to evaluate the cost-effectiveness of adding a hypothetical A&F module targeting LVPs for trauma admissions to an existing quality assurance intervention targeting high-value care and risk-adjusted outcomes. A secondary aim was to assess how certain A&F characteristics might influence its cost-effectiveness. METHODS: We conducted a cost-effectiveness analysis using a probabilistic static decision analytic model in the Québec trauma care continuum. We considered the Québec Ministry of Health perspective. Our economic evaluation compared a hypothetical scenario in which the A&F module targeting LVPs is implemented in a Canadian provincial trauma quality assurance program to a status quo scenario in which the A&F module is not implemented. In scenarios analyses we assessed the impact of A&F characteristics on its cost-effectiveness. Results are presented in terms of incremental costs per LVP avoided. RESULTS: Results suggest that the implementation of A&F module (Cost = $1,480,850; Number of LVPs = 6,005) is associated with higher costs and higher effectiveness compared to status quo (Cost = $1,124,661; Number of LVPs = 8,228). The A&F module would cost $160 per LVP avoided compared to status quo. The A&F module becomes more cost-effective with the addition of facilitation visits; more frequent evaluation; and when only high-volume trauma centers are considered. CONCLUSION: A&F module targeting LVPs is associated with higher costs and higher effectiveness than status quo and has the potential to be cost-effective if the decision-makers' willingness-to-pay is at least $160 per LVP avoided. This likely represents an underestimate of true ICER due to underestimated costs or missed opportunity costs. Results suggest that virtual facilitation visits, frequent evaluation, and implementing the module in high-volume centers can improve cost-effectiveness.

Prevalence and incidence of Paget's disease of bone: Temporal trend over 20 years in the province of Quebec, Canada.

BACKGROUND: Paget's disease of bone (PDB) is a focal bone disorder characterized by an increased bone remodeling and an anarchic bone structure. A decline of prevalence and incidence of PDB has been observed in some countries. No epidemiological data are available on PDB in Canada. AIMS: We aimed at examining the evolution of the prevalence and incidence of PDB in Quebec (Canada) by analyzing health administrative databases. METHODS: PDB case definition relied on one or more hospitalizations, or one or more physician-billing claims with a diagnosis code of PDB. To identify incident cases, a 'run-in' period of four years (1996-1999) was used to exclude prevalent cases. For each fiscal year from 2000 to 2001 to 2019-2020 (population size 2,914,480), crude age and sex-specific prevalence and incidence rates of PDB among individuals aged ≥55 years were determined, and sex-specific rates were also standardized to the 2011 age structure of the Quebec population. Generalized linear regressions were used to test for linear changes in standardized prevalence and incidence rates. RESULTS: Over the study period, standardized prevalence of PDB has remained stable in Quebec, from 0.44 % in 2000/2001 to 0.43 % in 2019/2020 (mean change -0.002, p-value = 0.0935). For the 2019-2020 fiscal year, 13,165 men and women had been diagnosed with PDB and prevalence of PDB increased with age. Standardized incidence of PDB has decreased over time from 0.77/1000 in 2000/2001 to 0.28/1000 in 2019-2020 (mean change -0.228/year, p-value<0.0001), the incidence decreasing from 0.82/1000 to 0.37/1000 in men and from 0.76/1000 to 0.22/1000 in women, respectively. This decrease was observed in all age categories. CONCLUSION: With the exception of a slight increase in PDB prevalence up to 0.55 % in years 2005 to 2007, the prevalence of PDB has remained stable in Quebec over the past 20 years, 13,160 men and women being currently diagnosed with PDB. The incidence has decreased over time. Our results support the epidemiological changes of PDB reported in other countries.

Integration of primary contact physiotherapists in the emergency department for individuals presenting with minor musculoskeletal disorders: Protocol for an economic evaluation.

OBJECTIVES: 1) To compare the average cost of an emergency department (ED) visit for various minor musculoskeletal disorders between two models of care (physiotherapist and ED physician or ED physician alone); 2) To evaluate the incremental cost-effectiveness ratio (ICER) of these two models of care over a 3-month period post-initial visit; and 3) To estimate the ICER of three ED models of care (physiotherapist and ED physician, ED physician alone, physiotherapist alone) over a two-year period. METHODS: Obj.1: The costs incurred by participants in the two groups during their ED visit will be calculated using the Time-Driven Activity-Based Costing (TDABC) method. These costs will be compared using generalized linear models. Obj. 2: The ICER of the two models will be evaluated over three months via a cost-utility analysis that will combine costs and effectiveness data (quality-adjusted life years) using both Health system and Societal perspectives (patient + health system costs). Obj. 3: The 2-year ICER of the three above-mentioned models will be estimated using a mathematical model including a decision tree (0-3 months post-visit) and a Markov model (3-24 months post-visit), also using both Health system and Societal perspectives. Data to answer the three objectives will come from data collected during a randomized clinical trial (n = 78, CHU de Québec)which will be supplemented with data obtained via some of the CHU de Québec administrative databases (nominative data; SIURGE (ED management software), Cristal-Net (patient electronic record), and the ED's pharmacy transactions directory; administrative data: drug costs repository), the literature, and public cost repositories. CONCLUSION: This study will help to determine which model of care is most efficient for the management of individuals who come to the ED with minor musculoskeletal disorders. The increased involvement of various health professionals in the management of patients in the ED paves the way for the development of new avenues of practice and more efficient organization of services.

Integration of real-world evidence from different data sources in health technology assessment.

Real-world evidence (RWE) is being increasingly used by a wide range of stakeholders involved in the therapeutic product lifecycle but remains underutilized in the health technology assessment (HTA) process. RWE aims to fill the current evidence gaps, reduce the uncertainty around the benefits of medical technologies, and better understand the long-term impact of health technologies in real-world conditions. Despite the minimal use of RWE in some elements of HTA, there has been a larger push to further utilize RWE in the HTA processes. HTA bodies, as other stakeholders, work towards developing more robust means to leverage RWE from various data sources in the HTA processes. However, these agencies need to overcome important challenges before the broader incorporation of RWE into their routine practice. This paper aims to explore the extensive integration of RWE utilizing diverse sources of RWD. We discuss the utilization of RWE in HTA processes, considering aspects such as when, where, and how RWE can be effectively applied. Additionally, we seek the potential challenges and barriers associated with the utilization of different data sources.

Evaluating the effectiveness of a multifaceted intervention to reduce low-value care in adults hospitalized following trauma: a protocol for a pragmatic cluster randomized controlled trial.

BACKGROUND: While simple Audit & Feedback (A&F) has shown modest effectiveness in reducing low-value care, there is a knowledge gap on the effectiveness of multifaceted interventions to support de-implementation efforts. Given the need to make rapid decisions in a context of multiple diagnostic and therapeutic options, trauma is a high-risk setting for low-value care. Furthermore, trauma systems are a favorable setting for de-implementation interventions as they have quality improvement teams with medical leadership, routinely collected clinical data, and performance-linked to accreditation. We aim to evaluate the effectiveness of a multifaceted intervention for reducing low-value clinical practices in acute adult trauma care. METHODS: We will conduct a pragmatic cluster randomized controlled trial (cRCT) embedded in a Canadian provincial quality assurance program. Level I-III trauma centers (n = 30) will be randomized (1:1) to receive simple A&F (control) or a multifaceted intervention (intervention). The intervention, developed using extensive background work and UK Medical Research Council guidelines, includes an A&F report, educational meetings, and facilitation visits. The primary outcome will be the use of low-value initial diagnostic imaging, assessed at the patient level using routinely collected trauma registry data. Secondary outcomes will be low-value specialist consultation, low-value repeat imaging after a patient transfer, unintended consequences, determinants for successful implementation, and incremental cost-effectiveness ratios. DISCUSSION: On completion of the cRCT, if the intervention is effective and cost-effective, the multifaceted intervention will be integrated into trauma systems across Canada. Medium and long-term benefits may include a reduction in adverse events for patients and an increase in resource availability. The proposed intervention targets a problem identified by stakeholders, is based on extensive background work, was developed using a partnership approach, is low-cost, and is linked to accreditation. There will be no attrition, identification, or recruitment bias as the intervention is mandatory in line with trauma center designation requirements, and all outcomes will be assessed with routinely collected data. However, investigators cannot be blinded to group allocation and there is a possibility of contamination bias that will be minimized by conducting intervention refinement only with participants in the intervention arm. TRIAL REGISTRATION: This protocol has been registered on ClinicalTrials.gov (February 24, 2023, # NCT05744154 ).

Potential Avoidable Costs of Low-Value Clinical Practices in Acute Injury Care in an Integrated Canadian Provincial Trauma System.

This economic evaluation estimated the direct health care costs associated with 11 low-value clinical practices in acute trauma care in the integrated health care system of Quebec, Canada.

A value-based comparison of the management of respiratory diseases in walk-in clinics and emergency departments.

OBJECTIVES: Our aim was to compare some of the health outcomes and costs associated with value of care in emergency departments (ED) and walk-in clinics for ambulatory patients presenting with an acute respiratory disease. METHODS: A health records review was conducted from April 2016 through March 2017 in one ED and one walk-in clinic. Inclusion criteria were: (i) ambulatory patients at least 18 years old, (ii) discharged home with a diagnosis of upper respiratory tract infection (URTI), pneumonia, acute asthma, or acute exacerbation of chronic obstructive pulmonary disease. Primary outcome was the proportion of patients returning to any ED or walk-in clinic within three and seven days of the index visit. Secondary outcomes were the mean cost of care and the incidence of antibiotic prescription for URTI patients. The cost of care was estimated from the Ministry of Health's perspectives using time-driven activity-based costing. RESULTS: The ED group included 170 patients and the walk-in clinic group 326 patients. The return visit incidences at three and seven days were, respectively, 25.9% and 38.2% in the ED vs. 4.9% and 14.7% in the walk-in clinic (adjusted relative risk (arr) of 4.7 (95% CI 2.6-8.6) and 2.7 (1.9-3.9)). The mean cost ($Cdn) of the index visit care was 116.0 (106.3-125.7) in the ED vs. 62.5 (57.7-67.3) in the walk-in clinic (mean difference of 56.4 (45.7-67.1)). Antibiotic prescription for URTI was 5.6% in the ED vs. 24.7% in the walk-in clinic (arr 0.2, 0.01-0.6). CONCLUSIONS: This study is the first in a larger research program to compare the value of care between walk-in clinics and the ED. The potential advantages of walk-in clinics over EDs (lower costs, lower incidence of return visits) for ambulatory patients with respiratory diseases should be considered in healthcare planning.

RéSUMé: OBJECTIFS: Notre objectif était de comparer certains des résultats sanitaires et des coûts associés à la valeur des soins dans les services d'urgence et les cliniques sans rendez-vous pour les patients ambulatoires souffrant d'une maladie respiratoire aiguë. MéTHODES: Une revue des dossiers médicaux a été réalisée d'avril 2016 à mars 2017 dans un service d'urgence et une clinique sans rendez-vous. Les critères d'inclusion étaient les suivants : (i) patients ambulatoires âgés d'au moins 18 ans, (ii) renvoyés chez eux avec un diagnostic d'infection des voies respiratoires supérieures (IVRS), de pneumonie, d'asthme aigu ou d'exacerbation aiguë de la maladie pulmonaire obstructive chronique. Le résultat primaire était la proportion de patients retournant à un service d'urgence ou à une clinique sans rendez-vous dans les trois et sept jours suivant la visite de référence. Les résultats secondaires étaient le coût moyen des soins et l'incidence de la prescription d'antibiotiques pour les patients atteints d'IVRS. Le coût des soins a été estimé à partir des perspectives du ministère de la santé, en utilisant la méthode de calcul des coûts par activité en fonction du temps. RéSULTATS: Le groupe des urgences comprenait 170 patients et le groupe des cliniques sans rendez-vous 326 patients. Les incidences des visites de retour à trois et sept jours étaient respectivement de 25,9 % et 38,2 % dans le service des urgences contre 4,9 % et 14,7 % à la clinique sans rendez-vous (risque relatif ajusté (arr) de 4,7 (IC à 95 % 2,6 à 8,6) et 2,7 (1,9-3,9)). Le coût moyen ($CAN) de la visite de référence était de 116,0 (106,3-125,7) aux urgences contre 62,5 (57,7-67,3) dans la clinique sans rendez-vous (différence moyenne de 56,4 (45,7-67,1)). La prescription d'antibiotiques pour l'IVRS était de 5,6 % aux urgences contre 24,7 % dans la clinique sans rendez-vous (arr 0,2, 0,01-0,6). CONCLUSIONS: Cette étude est la première d'un programme de recherche plus vaste visant à comparer la valeur des soins entre les cliniques sans rendez-vous et les urgences. Les avantages potentiels des cliniques sans rendez-vous par rapport aux services d'urgence (coûts moindres, incidence plus faible des visites de retour) pour les patients ambulatoires souffrant de maladies respiratoires devraient être pris en compte dans la planification des soins de santé.

Molecular test of Paget's disease of bone in families not linked to SQSTM1 gene mutations.

Purpose: Paget's disease of bone (PDB) is a focal metabolic bone disorder characterized by an increased bone remodeling. Fifteen to 40 % of PDB patients have a familial form with an autosomal dominant inheritance. Disease-causing mutations of the SQSTM1 gene have been linked to PDB in about 40 % of families whereas genes linked to the remaining families are unknown. Several single nucleotide polymorphisms (SNPs) have been associated with PDB in unrelated patient non-carriers of a SQSTM1 mutation. The current clinical practice guidelines still recommend the measure of serum total alkaline phosphatase (sALP) for PDB screening. In unrelated individual non-carriers of SQSTM1 mutations, we previously developed a genetic test combining male sex with five genetic markers (rs499345, rs5742915, rs2458413, rs3018362, rs2234968), giving rise to an area under the curve (AUC) for PDB phenotype of 0.73 (0.69; 0.77). A combination of male sex with total calcium corrected for albumin and Procollagen type I N-terminal propeptide (P1NP), had an AUC of 0.82 (0.73; 0.92). Combining both genetic and biochemical tests increased the AUC to 0.89 (0.83; 0.95). Objective: This study aimed at estimating the performance of our previous test of PDB, in families not linked to SQSTM1 mutations with disease-causing genes yet unknown, and at developing a new algorithm if the performance is not satisfactory. Methods: We genotyped the five SNPs cited above, and measured calcium corrected for albumin and P1NP in 181 relatives, with PDB or not, from 19 PDB families not linked to SQSTM1 mutations. Bivariate and multivariate logistic regression models including male sex were fitted to search for a molecular test that could best detect PDB in these families. A receiving operating characteristics analysis was done to establish a cut-off point for continuous variables. Results: Logistic regression estimates of our previous molecular test gave rise to a high sensitivity of 78 %, 97 % and 88 % for the genetic, biochemical, and combined test but the specificity was very low, 35 %, 11 % and 21 %, respectively. This poor specificity persisted even when the cut-off point was changed. We then generated in these families, new logistic regression estimates but on the same parameters as mentioned above, giving rise to an AUC of 0.65 (0.55; 0.75) for the genetic test, of 0.84 (0.74; 0.94) for the biochemical test, and 0.89 (0.82; 0.96) for the combination test, the latter having a sensitivity of 96 % and specificity of 57 %. By comparison serum P1NP alone gave rise to an AUC of 0.84 (0.73; 0.94), with a sensitivity of 71 % and a specificity of 79 %. Conclusion: In PDB families not linked to SQSTM1 mutations, the estimates of our previous molecular test gave rise to a poor specificity. Using new estimates, the biochemical and combined tests have similar predictive abilities than our former test. Serum P1NP is a bone marker of interest for the screening for PDB in families not linked to SQSTM1 mutations.

Health Utilities Index Mark 3 scores for children and youth: Population norms for Canada based on cycles 5 (2016 and 2017) and 6 (2018 and 2019) of the Canadian Health Measures Survey.

Background: Utility scores are an important tool for evaluating health-related quality of life. Utility score norms have been published for Canadian adults, but no nationally representative utility score norms are available for children and youth. Data and methods: Health Utilities Index Mark 3 (HUI3) data from two recent cycles of the Canadian Health Measures Survey (i.e., 2016 and 2017, and 2018 and 2019) were used to provide utility score norms for children aged 6 to 11 years and adolescents aged 12 to 17 years. Children younger than 14 years answered the HUI3 under the supervision of an adult, while older children answered without supervision. Utility scores were reported as a weighted average (95% confidence intervals [CIs]) and median values (interquartile range). Utility scores were stratified by sociodemographic and medical characteristics of the child or adolescent. Regression analyses were used to identify predictors of utility scores. All results were weighted using sampling weights provided by Statistics Canada. Results: Among the 2,297,136 children aged 6 to 11 years and the 2,329,185 adolescents aged 12 to 17 years in the weighted sample, the average utility scores were 0.95 (95% CI: 0.94 to 0.95) and 0.89 (95% CI: 0.87 to 0.90), respectively. Approximately 60% of the children and 34% of the adolescents had a utility score of 1.00. Analyses identified several factors associated with utility scores (e.g., age, chronic condition and income levels), although differences were observed between children and adolescents. Interpretation: This study provides utility score estimates based on a nationally representative sample of Canadian children and youth. Further research examining the determinants of utility scores of children and adolescents is warranted.

Oral Anticoagulants and the Risk of Dementia in Patients With Nonvalvular Atrial Fibrillation: A Population-Based Cohort Study.

BACKGROUND AND OBJECTIVES: Nonvalvular atrial fibrillation (NVAF) is associated with an increased risk of dementia. Oral anticoagulants (OACs) are essential for stroke prevention in NVAF, and studies have shown a possible protective effect on dementia. However, findings have been inconsistent and hampered by methodological limitations. Thus, we assessed whether the use of OACs is associated with a decreased incidence of dementia in patients with NVAF. In addition, we explored the impact of the cumulative duration of OAC use on the incidence of dementia. METHODS: Using the UK Clinical Practice Research Datalink, we formed a cohort of all patients aged 50 years or older with an incident diagnosis of NVAF between 1988 and 2017 and no prior OAC use, with a follow-up until 2019. Patients were considered unexposed until 6 months after their first OAC prescription for latency considerations and exposed thereafter until the end of follow-up. We used time-dependent Cox regression models to estimate hazard ratios (HRs), adjusted for 54 covariates, with 95% CIs for dementia associated with OAC use, compared with nonuse. We also assessed whether the risk varied with the cumulative duration of OAC use, compared with nonuse, by comparing prespecified exposure categories defined in a time-varying manner and by modeling the HR using a restricted cubic spline. RESULTS: The cohort included 142,227 patients with NVAF, with 8,023 cases of dementia over 662,667 person-years of follow-up (incidence rate 12.1, 95% CI 11.9-12.4 per 1,000 person-years). OAC use was associated with a decreased risk of dementia (HR 0.88, 95% CI 0.84-0.92) compared with nonuse. A restricted cubic spline also indicated a decreased risk of dementia, reaching a low at approximately 1.5 years of cumulative OAC use and stabilizing thereafter. Moreover, OAC use decreased the risk in patients aged 75 years and older (HR 0.84, 95% CI 0.80-0.89), but not in younger patients (HR 0.99, 95% CI 0.90-1.10). DISCUSSION: In patients with incident NVAF, OACs were associated with a decreased risk of dementia, particularly in elderly individuals. This warrants consideration when weighing the risks and benefits of anticoagulation in this population. CLASSIFICATION OF EVIDENCE: This study provides Class II evidence that in patients with NVAF, OAC use (vs nonuse) is associated with a decreased risk of dementia.

The use of early neuromuscular blockage in acute respiratory distress syndrome: A systematic review and meta-analyses of randomized clinical trials.

BACKGROUND: Acute Respiratory Distress Syndrome (ARDS) as defined by the Berlin definition has an approximate mortality rate of 40% and no curative treatment. Mutliple therapies have been studied to reduce mortality but only neuromuscular blocking agents show potential benefits on mortality and other complications of ARDS. OBJECTIVE: This review aimed to investigate the efficacy of neuromuscular blockers in ARDS METHODS: Medline, Embase, Cochrane Central and Web of Science were queried on October 1st, 2021. Randomized clinical trials comparing neuromuscular blockers to any comparator in treating ARDS were included. Primary outcome was mortality. Secondary outcomes were ventilator-free days, intensive care (ICU) length of stay (LOS) and complications. Results between sedation levels were examined with a Bayesian Network for Meta-analysis method. RESULTS: We included 6 trials compiling a total of 1557 patients. Neuromuscular blockers compared to any comparator in treating ARDS showed a reduction in mortality (RR 0.79 [95% CI, 0.62 to 0.99]). No difference in ventilator-free days (MD 0.68 [95% CI, -0.50 to 1.85]) or ICU LOS (MD 0.77 [95% CI, -2.99 to 4.54]) were found. A Bayesian Network Meta-analysis yielded no difference in mortality when using light sedation compared to heavy sedation in ARDS. (OR 0.58 [95% CrI, 0.07 to 4.46].) CONCLUSION: Neuromuscular blockers safely reduce mortality. Light sedation potentially has a similar impact on mortality as heavy sedation that carries some burden. A non-inferiority trial comparing both sedation levels may be warranted considering the added value of light sedation.

Adaptation of time-driven activity-based costing to the evaluation of the efficiency of ambulatory care provided in the emergency department.

Objectives: The aim of this study was: (1) to adapt the time-driven activity-based costing (TDABC) method to emergency department (ED) ambulatory care; (2) to estimate the cost of care associated with frequently encountered ambulatory conditions; and (3) to compare costs calculated using estimated time and objectively measured time. Methods: TDABC was applied to a retrospective cohort of patients with upper respiratory tract infections, urinary tract infections, unspecified abdominal pain, lower back pain and limb lacerations who visited an ED in Québec City (Canada) during fiscal year 2015-2016. The calculated cost of care was the product of the time required to complete each care procedure and the cost per minute of each human resource or equipment involved. Costing based on durations estimated by care professionals were compared to those based on objective measurements in the field. Results: Overall, 220 care episodes were included and 3080 time measurements of 75 different processes were collected. Differences between costs calculated using estimated and measured times were statistically significant for all conditions except lower back pain and ranged from $4.30 to $55.20 (US) per episode. Differences were larger for conditions requiring more advanced procedures, such as imaging or the attention of ED professionals. Conclusions: The greater the use of advanced procedures or the involvement of ED professionals in the care, the greater is the discrepancy between estimated-time-based and measured-time-based costing. TDABC should be applied using objective measurement of the time per procedure.

Economic Evaluation of In-Hospital Clinical Practices in Acute Injury Care: A Systematic Review.

OBJECTIVES: Underuse of high-value clinical practices and overuse of low-value practices are major sources of inefficiencies in modern healthcare systems. To achieve value-based care, guidelines and recommendations should target both underuse and overuse and be supported by evidence from economic evaluations. We aimed to conduct a systematic review of the economic value of in-hospital clinical practices in acute injury care to advance knowledge on value-based care in this patient population. METHODS: Pairs of independent reviewers systematically searched MEDLINE, Embase, Web of Science, and Cochrane Central Register for full economic evaluations of in-hospital clinical practices in acute trauma care published from 2009 to 2019 (last updated on June 17, 2020). Results were converted into incremental net monetary benefit and were summarized with forest plots. The protocol was registered with PROSPERO (CRD42020164494). RESULTS: Of 33 910 unique citations, 75 studies met our inclusion criteria. We identified 62 cost-utility, 8 cost-effectiveness, and 5 cost-minimization studies. Values of incremental net monetary benefit ranged from international dollars -467 000 to international dollars 194 000. Of 114 clinical interventions evaluated (vs comparators), 56 were cost-effective. We identified 15 cost-effective interventions in emergency medicine, 6 in critical care medicine, and 35 in orthopedic medicine. A total of 58 studies were classified as high quality and 17 as moderate quality. From studies with a high level of evidence (randomized controlled trials), 4 interventions were clearly dominant and 8 were dominated. CONCLUSIONS: This research advances knowledge on value-based care for injury admissions. Results suggest that almost half of clinical interventions in acute injury care that have been studied may not be cost-effective.

Trends in Canadian prescription drug purchasing: 2001-2020.

BACKGROUND: In 2019, more than $34.5 billion was spent on prescription drugs in Canada. However, little is known about the distribution of this spending across medications and settings (outpatient and inpatient) over time. The objective of this paper is to describe the largest expenditures by medication class over time in inpatient and outpatient settings. This information can help to guide policies to control prescription medication expenditures. METHODS: IQVIA's Canadian Drugstore and Hospital Purchases Audit data from January 1, 2001, to December 31, 2020, were used. In this dataset, purchasing was stratified by outpatient drugstore and inpatient hospital. Spending trajectories in both settings were compared to total expenditure over time. Total expenditure of the 25 medications with the largest expenditure were compared over time, stratified by setting. Nominal costs were used for all analysis. RESULTS: In 2001, spending in the outpatient and inpatient settings was greatest on atorvastatin ($467.0 million) and erythropoietin alpha ($91.2 million), respectively. In 2020, spending was greatest on infliximab at $1.2 billion (outpatient) and pembrolizumab at $361.6 million (inpatient). Annual outpatient spending, although increasing, has been growing at a slower rate (5.3%) than inpatient spending (7.0%). In both settings, spending for the top 25 medications has become increasingly concentrated on biologic agents, with a reduction in the diversity of therapeutic classes of agents over time. DISCUSSION: Identification of the concentration on spending on biologic agents is a key step in managing costs of prescription medications in Canada. Given the increases in spending on biologic agents over the last 20 years, current cost-control mechanisms may be insufficient. Future research efforts should focus on examining the effectiveness of current cost-control mechanisms and identifying new approaches to cost control for biologic agents.

Health-Related Quality of Life of Patients Presenting to the Emergency Department with a Musculoskeletal Disorder.

BACKGROUND: Musculoskeletal disorders (MSKDs) are among the most disabling and costly non-fatal health conditions. They may lead to long-term consequences such as chronic pain, physical limitations, and poorer quality of life. They also account for a significant proportion of emergency department visits, representing between 18% and 25% of all visits, depending on country. PURPOSE: To assess the health-related quality of life of patients presenting to the emergency department with a MSKD, to convert their answers to utility scores and to explore the association between diverse socio-demographic and clinical variables and patients' health-related quality of life. PATIENTS AND METHODS: This is an analysis of cross-sectional data obtained during the baseline assessment performed as part of a 6-month pragmatic randomized controlled trial conducted in an academic emergency department. We included patients aged 18-80 years with a minor MSKD. The main outcome measures were health-related quality of life (five dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) and utility scores (-0.148 - worse than death, 0 - dead, 0.949 - perfect health) measured with the EQ-5D-5L. Possible associations were explored by comparing scores across subgroups based on certain socio-demographic (eg, age, gender, triage score) and clinical factors (eg, pain interference on function, pain intensity) and with reference values using descriptive statistics (mean, median), rankFD ANOVAs, and χ 2 tests. RESULTS: Sixty-nine participants completed the EQ-5D-5L. Mean and median utility scores were, respectively, 0.536 (95% CI: 0.479-0.594) and 0.531 (IQR: 0.356-0.760). Participants with higher levels of pain (<4/10: 0.741, 95% CI: 0.501-0.980; 4-7/10: 0.572, 0.500-0.644; >7/10: 0.433, 0.347-0.518) or pain interference on function (<4/10: 0.685, 95% CI: 0.605-0.764; 4-7/10: 0.463, 0.394-0.533; >7/10: 0.294, 0.126-0.463) presented significantly lower utility scores. No significant differences were found for other socio-demographic characteristics. CONCLUSION: In patients with MSKDs who present to the emergency department, higher levels of pain or pain interference are associated with decreased health-related quality of life. These findings need to be confirmed on a larger scale.

A comparison of confounder selection and adjustment methods for estimating causal effects using large healthcare databases.

PURPOSE: Confounding adjustment is required to estimate the effect of an exposure on an outcome in observational studies. However, variable selection and unmeasured confounding are particularly challenging when analyzing large healthcare data. Machine learning methods may help address these challenges. The objective was to evaluate the capacity of such methods to select confounders and reduce unmeasured confounding bias. METHODS: A simulation study with known true effects was conducted. Completely synthetic and partially synthetic data incorporating real large healthcare data were generated. We compared Bayesian adjustment for confounding (BAC), generalized Bayesian causal effect estimation (GBCEE), Group Lasso and Doubly robust estimation, high-dimensional propensity score (hdPS), and scalable collaborative targeted maximum likelihood algorithms. For the hdPS, two adjustment approaches targeting the effect in the whole population were considered: Full matching and inverse probability weighting. RESULTS: In scenarios without hidden confounders, most methods were essentially unbiased. The bias and variance of the hdPS varied considerably according to the number of variables selected by the algorithm. In scenarios with hidden confounders, substantial bias reduction was achieved by using machine-learning methods to identify proxies as compared to adjusting only by observed confounders. hdPS and Group Lasso performed poorly in the partially synthetic simulation. BAC, GBCEE, and scalable collaborative-targeted maximum likelihood algorithms performed particularly well. CONCLUSIONS: Machine learning can help to identify measured confounders in large healthcare databases. They can also capitalize on proxies of unmeasured confounders to substantially reduce residual confounding bias.

Development and Validation of an Instrument to Measure Health-Related Out-of-Pocket Costs: The Cost for Patients Questionnaire.

OBJECTIVE: The growth of healthcare spending is a major concern for insurers and governments but also for patients whose health problems may result in costs going beyond direct medical costs. To develop a comprehensive tool to measure direct and indirect costs of a health condition for patients and their families to various outpatient contexts. METHODS: We conducted a content and face validation including results of a systematic review to identify the items related to direct and indirect costs for patients or their families and an online Delphi to determine the cost items to retain. We conducted a pilot test-retest with 18 naive participants and analyzed data calculating intraclass correlation and kappa coefficients. RESULTS: An initial list of 34 items was established from the systematic review. Each round of the Delphi panel incorporated feedback from the previous round until a strong consensus was achieved. After 4 rounds of the Delphi to reach consensus on items to be included and wording, the questionnaire had a total of 32 cost items. For the test-retest, kappa coefficients ranged from -0.11 to 1.00 (median = 0.86), and intraclass correlation ranged from -0.02 to 0.99 (median = 0.62). CONCLUSIONS: A rigorous process of content and face development was implemented for the Cost for Patients Questionnaire, and this study allowed to set a list of cost elements to be considered from the patient's perspective. Additional research including a test-retest with a larger sample will be part of a subsequent validation strategy.

Evidence of the Different Associations of Prognostic Factors With Censoring Across Treatment Groups and Impact on Censoring Weight Model Specification: The Example of Anticoagulation in Atrial Fibrillation.

Inverse probability of censoring weights (IPCWs) may reduce selection bias due to informative censoring in longitudinal studies. However, in studies with an active comparator, the associations between predictors and censoring may differ across treatment groups. We used the clinical example of anticoagulation treatment with warfarin or a direct oral anticoagulant (DOAC) in atrial fibrillation to illustrate this. The cohort of individuals initiating an oral anticoagulant during 2010-2016 was identified from the Régie de l'assurance maladie du Québec (RAMQ) databases. The parameter of interest was the hazard ratio (HR) of the composite of stroke, major bleeding, myocardial infarction, or death associated with continuous use of warfarin versus DOACs. Two strategies for the specification of the model for estimation of censoring weights were explored: exposure-unstratified and exposure-stratified. The HR associated with continuous treatment with warfarin versus DOACs adjusted with exposure-stratified IPCWs was 1.26 (95% confidence interval: 1.20, 1.33). Using exposure-unstratified IPCWs, the HR differed by 15% in favor of DOACs (1.41, 95% confidence interval: 1.34, 1.48). Not accounting for the different associations between the predictors and informative censoring across exposure groups may lead to misspecification of censoring weights and biased estimate on comparative effectiveness and safety.

Canadian French translation and linguistic validation of the health-related quality of life utility measure for pre-school children.

BACKGROUND: There is a need to perform a Canadian French translation and linguistic validation of the health-related quality of life utility measure for pre-school children (HuPS) conceptually equivalent to the original Canadian English version. RESEARCH DESIGN AND METHODS: The translation process consisted of forward and back translations. The linguistic validation was performed with the parents of preschool children during face-to-face cognitive debriefing interviews. The whole process was done in accordance with academic standards and the guidance of the Food and Drug Administration (FDA) for patient-reported outcome instruments. RESULTS: The results of back translations indicated that 89% of the sentences were identical or almost identical to the original English-language wording. The review of the back translations led to a change in 13 sentences out of 91 from the reconciled forward translation, while the linguistic validation process with 13 parents led to 14 additional changes. Preliminary reliability validation results indicate a Cronbach's alpha of 0.73. CONCLUSION: The translation and linguistic testing processes were successful in creating a valid HuPS in Canadian French (HuPS-CF). This translation should be the subject of reliability and validity studies in a wide variety of clinical and general populations before to use in research projects.