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Background: The EuroQol- 5 Dimension (EQ-5D) is a generic patient-reported outcome measure widely used to capture meaningful change in health-related quality of life between treatments to inform drug and health technology reimbursement decision making. The EQ-5D-5L (5 level) consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) and a visual analogue scale (VAS). Each dimension has 5 levels, ranging from 1 “no problems” to 5 “extreme problems”, generating 3125 distinct health states (HS). The VAS score ranges from 0 (worst health imaginable) to 100 (best health imaginable). We used Canadian Index values to calculate community-weighted utility scores for 5 pre-defined breast cancer (BC) disease states.Methods: This cross-sectional study included adult women diagnosed with stage I to IV BC, who completed EQ-5D-5L, during outpatient clinic visits at two academic cancer centres in Toronto. Participants were classified into 5 disease states, considered relevant both to clinical practice and economic modeling, ranging from primary breast cancer to metastatic disease. EQ-5D-5L scores were described for each disease state, and mean utility scores and VAS scores were calculated.Results: 549 women were included; the mean age was 57 (SD 12) years; 72% had a Charlson Comorbidity Index of 0. A majority reported “Slight” to “Moderate” problems for the “Pain/Discomfort” (60%) and “Anxiety/Depression” (55%) dimensions of EQ-5D-5L; in all other dimensions, most patients reported “No problems”. As expected, patients with metastatic BC had the highest proportion of reported problems. The mean EQ-5D-5L index score was 0.83 (SD 0.13; range 0.36 to 0.95), with a distribution skewed towards full health and a ceiling effect of 20% (n=110; Table 1). Only 126 different self-reported health states were observed in our patient population. Mean VAS was 75 (SD 17.5; range 10 to 100). For early BC, mean index scores were: 0.87 (n=49; SD 0.07) for patients on chemotherapy (+/-targeted therapy); 0.85 (n=212; SD 0.12) for patients on endocrine treatment (+/-targeted therapy); 0.79 (n=16; SD 16) for patients on targeted therapy; and 0.84 (n=129; SD 0.14) for patients not receiving systemic therapy. For metastatic BC, mean index scores were: 0.79 (n=36, SD 14) for patients on chemotherapy (+/-targeted therapy); 0.80 (n=79, SD 0.14) for patients on endocrine treatment (+/-targeted therapy); 0.69 (n=8; SD 0.19) for patients on targeted therapy; and 0.77 (n=20; SD 0.14) for patients not receiving systemic therapy.Conclusions: BC had an impact mainly in the “Pain/Discomfort” and “Anxiety/Depression” dimensions of EQ-5D-5L. A high ceiling effect was observed in this patient population, despite using the 5L version of EQ-5D. EQ-5D-5L health utilities were significantly lower for metastatic BC, but no significant differences were found among various early-stage BC states. Table 1.- Mean EQ-5D-5L Index and VAS scoresEQ-5D-5LIndex ScoresVASDisease StateN (%)Mean (SD)Min-MaxMean (SD)Min-Max1st year after primary BC146 (27)0.85 (0.12)*0.23-0.9576.5(16.6)10-1001st year after recurrence or primary BC13 (2)0.78 (0.21)0.13-0.9568.9(23.3)20-1002nd-5th year after a primary BC or recurrence treated with curative intent185 (34)0.84 (0.11)*0.21-0.9578.3(14.7)36-1006th and following years after a primary BC or recurrence treated with curative intent62 (11)0.86 (0.14)*0.36-0.9581.0(15.6)20-100Metastatic BC143 (26)0.78 (0.14)0.29-0.9567.3(19.2)21-100Total549 (100)0.83 (0.13)0.13-0.9575.0(17.5)10-100*significant difference was found between these disease states and metastatic diseases (P < 0.05). Citation Format: Sofia Torres, Ahmed Bayoumi, Maureen Trudeau, Ana Beatriz Kinupe Abrahao, Nicholas Mitsakakis, Murray Krahn, Geoffrey Liu. Eq-5d-5l utility scores for patients with breast cancer [abstract]. In: Proceedings of the 2021 San Antonio Breast Cancer Symposium; 2021 Dec 7-10; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2022;82(4 Suppl):Abstract nr P4-12-11.

Background: To improve symptom management in cancer patients, the province of Ontario (Canada) implemented the collection of the Edmonton Symptom Assessment System (ESAS) and Patient-Reported Functional Status (PRFS) tool at cancer centre outpatient visits. ESAS measures 9 symptoms on a scale from 0 (no symptoms) to 10 (worst possible symptom); scores > 4 (moderate to severe) require intervention. The PRFS tool is a version of the Eastern Cooperative Oncology Group performance status tool designed to be completed by the patient; scores range from 0 (“normal with no limitation”) to 4 (“pretty much bedridden, rarely out of bed”). Our main objective was to describe the symptom burden and functional status of breast cancer (BC) patients.Methods: This cross-sectional study included women with stage I-IV BC who completed the ESAS and the PRFS tool during outpatient clinic visits at 2 Toronto academic centres. ESAS and PRFS scores were described for 3 participant groups: women diagnosed with early-stage BC (ESBC= stages I-III) in the year prior to enrolment; women with ESBC diagnosed > 1 year prior to enrolment; and women with metastatic BC. Multivariable logistic regression models were used to identify factors associated with ESAS scores > 4 for each ESAS symptom. Covariates included age, comorbidity, BC subtype, disease group, previous surgery, and current systemic therapy. Results: Of 381 women, 107 (28%) were diagnosed with ESBC ≤ 1 year prior to enrolment; 171 (45%) were diagnosed with ESBC > 1 year prior to enrolment; 103 (27%) had metastatic BC. Mean age was 57 (SD17) years. For ESBC, tiredness had the highest incidence of moderate to severe scores, followed by impaired well-being and anxiety (Table 1). For metastatic BC, impaired well-being had the highest incidence of moderate to severe symptoms, followed by tiredness and anxiety. More than 20% of metastatic BC patients had scores > 4 for pain, drowsiness, shortness of breath and depression. Most reported PRFS scores of 0 (“Normal with no limitations”) or 1 (“Not my normal self, but able to be up and about with fairly normal activities”), but 17% of metastatic BC patients reported a PRFS of 2 (“Not feeling up to most things, but in bed or chair less than half the day”). A comorbidity score ≥1 was associated with moderate to severe depression (OR 2.0; 95%CI 1.1-3.7), lack of appetite (OR 3.1; 95%CI 1.4-6.6), shortness of breath (OR 2.6; 95%CI 1.3-5.2), tiredness (OR 2.0; 95%CI 1.2-3.3) and impaired well-being (OR 2.0; 95%CI 1.2-3.4). Metastatic BC was associated with moderate to severe scores for anxiety (OR 2.3; 95%CI 1.2-4.5), pain (OR 3.0; 95%CI 1.4-6.4) and impaired well-being (OR 2.1; 95%CI 1.1-4.0). Conclusion: A high prevalence of symptom scores > 4 was found both in ESBC and metastatic BC, highlighting the need of symptom screening for all BC patients. Particular attention should be paid to patients with comorbidity scores ≥ 1, who have increased odds of presenting higher symptom scores. Table 1.- Prevalence of ESAS scores ≥ 4 and PRFS scores for each disease group.ESAS SymptomsPercent with ESAS scores ≥ 4 (moderate to severe)Early-Stage Breast Cancer – 1st year (N=107)Early-Stage Breast Cancer – > 1st year (N=171)Metastatic Breast Cancer (N=103)Total (N=381)Pain12.1%14.7%30.1%18.2%Tiredness25.2%30.1%39.8%31.3%Drowsiness1.8%12.9%25.2%16.6%Nausea9.4%3.5%7.8%4.2%Lack of Appetite10.3%7.1%13.6%9.5%Shortness of Breath14%7.6%22.3%12.4%Depression17.7%14.6%22.6%16.5%Anxiety24.3%17.2%35.3%21.3%Well-being1.8%25.7%44.1%30.3%PRFS scoresPercent with PRFS ScoreNormal with no limitations (0)30.8%49.7%28.2%38.6%Not my normal self, but able to be up and about with fairly normal activities (1)52.3%42.1%50.5%47.2%Not feeling up to most things, but in bed or chair less than half the day (2)12.2%4.7%16.5%10%Able to do little activity and spend most of the day in bed or chair (3)3.7%2.3%4.9%3.4%Pretty much bedridden, rarely out of bed (4)---- Citation Format: Sofia Torres, Ahmed Bayoumi, Geoffrey Liu, Ana Beatriz Kinupe Abrahao, Nicholas Mitsakakis, Murray Krahn, Maureen Trudeau. Patient reported symptoms and functional status in breast cancer [abstract]. In: Proceedings of the 2021 San Antonio Breast Cancer Symposium; 2021 Dec 7-10; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2022;82(4 Suppl):Abstract nr P4-10-22.

Objectives Chronic hepatitis C (CHC) infection affects more than 70 million people worldwide and imposes considerable health and economic burdens on patients and society. This study estimated 2 understudied components of the economic burden, patient out-of-pocket (OOP) costs and time costs, in patients with CHC in a tertiary hospital clinic setting and a community clinic setting. Methods This was a multicenter, cross-sectional study with hospital-based (n = 174) and community-based (n = 101) cohorts. We used a standardized instrument to collect healthcare resource use, time, and OOP costs. OOP costs included patient-borne costs for medical services, nonprescription drugs, and nonmedical expenses related to healthcare visits. Patient and caregiver time costs were estimated using an hourly wage value derived from patient-reported employment income and, where missing, derived from the Canadian census. Sensitivity analysis explored alternative methods of valuing time. Costs were reported in 2020 Canadian dollars. Results The mean 3-month OOP cost was $55 (95% confidence interval [CI] $21-$89) and $299 (95% CI $170-$427) for the community and hospital cohorts, respectively. The mean 3-month patient time cost was $743 (95% CI $485-$1002) (community) and $465 (95% CI $248-$682) (hospital). The mean 3-month caregiver time cost was $31 (95% CI $0-$63) (community) and $277 (95% CI $174-$380) (hospital). Patients with decompensated cirrhosis bore the highest costs. Conclusions OOP costs and patient and caregiver time costs represent a considerable economic burden to patient with CHC, equivalent to 14% and 21% of the reported total 3-month income for the hospital-based and community-based cohorts, respectively.

Background Hepatitis C virus (HCV) infection causes substantial morbidity and mortality in Canada and is of concern among First Nations communities. In partnership with the Ontario First Nations HIV/AIDS Education Circle, we described trends in HCV testing and epidemiologic features among Status First Nations people in Ontario. Methods In this retrospective study, we used health administrative databases for 2006-2014 in Ontario with 3 cohorts of Status First Nations people: those tested for HCV for the first time, those who tested positive for HCV antibodies or RNA, and those with no HCV laboratory or testing records. We examined cohort characteristics, and the annual prevalence and incidence of testing and diagnosis of HCV infection. Outcomes were stratified by region, sex and residence within or outside of First Nations communities. Results During the study period, 2423 Status First Nations people were diagnosed with HCV infection, 20 481 received their first test, and 135 185 had no test record. The point prevalence of ever having been tested increased from 6.3 (95% confidence interval [CI] 6.2-6.5) per 100 people in 2006 to 16.2 (95% CI 16.0-16.4) per 100 people in 2014. The point prevalence of diagnosed HCV infection increased from 0.9 (95% CI 0.9-1.0) to 2.0 (95% CI 1.9-2.0) per 100 people. The incidence of first test and of diagnosis increased from 12.1 (95% CI 11.5-12.6) to 21.3 (95% CI 20.5-22.1) per 1000 person-years and from 1.3 (95% CI 1.1-1.5) to 2.3 (95% CI 2.1-2.6) per 1000 person-years, respectively. Testing, diagnosis and prevalence of HCV infection were consistently higher among people living outside of versus within First Nations communities, but larger increases over time were observed among those living within First Nations communities. Interpretation Testing and diagnosis of HCV infection increased from 2006 to 2014 among Status First Nations people in Ontario. Our findings indicate the need for population-level efforts to eliminate hepatitis C in First Nations communities.

Background: Colonization and marginalization have affected the risk for and experience of hepatitis C virus (HCV) infection for First Nations people in Canada. In partnership with the Ontario First Nations HIV/AIDS Education Circle, we estimated the publicly borne health care costs associated with HCV infection among Status First Nations people in Ontario. Methods: In this retrospective matched cohort study, we used linked health administrative databases to identify Status First Nations people in Ontario who tested positive for HCV antibodies or RNA between 2004 and 2014, and Status First Nations people who had no HCV testing records or only a negative test result (control group, matched 2:1 to case participants). We estimated total and net costs (difference between case and control participants) for 4 phases of care: prediagnosis (6 mo before HCV infection diagnosis), initial (after diagnosis), late (liver disease) and terminal (6 mo before death), until death or Dec. 31, 2017, whichever occurred first. We stratified costs by sex and residence within or outside of First Nations communities. All costs were measured in 2018 Canadian dollars. Results: From 2004 to 2014, 2197 people were diagnosed with HCV infection. The mean net total costs per 30 days of HCV infection were $348 (95% confidence interval [CI] $277 to $427) for the prediagnosis phase, $377 (95% CI $288 to $470) for the initial phase, $1768 (95% CI $1153 to $2427) for the late phase and $893 (95% CI −$1114 to $3149) for the terminal phase. After diagnosis of HCV infection, net costs varied considerably among those who resided within compared to outside of First Nations communities. Net costs were higher for females than for males except in the terminal phase. Interpretation: The costs per 30 days of HCV infection among Status First Nations people in Ontario increased substantially with progression to advanced liver disease and finally to death. These estimates will allow for planning and evaluation of provincial and territorial population-specific hepatitis C control efforts.

Background and ObjectivesTo determine the association between material deprivation and direct health care costs and clinical outcomes following stroke in the context of a publicly funded universal health care system.MethodsIn this population-based cohort study of patients with ischemic and hemorrhagic stroke admitted to the hospital between 2008 and 2017 in Ontario, Canada, we used linked administrative data to identify the cohort, predictor variables, and outcomes. The exposure was a 5-level neighborhood material deprivation index. The primary outcome was direct health care costs incurred by the public payer in the first year. Secondary outcomes were death and admission to long-term care.ResultsAmong 90,289 patients with stroke, the mean (SD) per-person costs increased with increasing material deprivation, from $50,602 ($55,582) in the least deprived quintile to $56,292 ($59,721) in the most deprived quintile (unadjusted relative cost ratio and 95% confidence interval 1.11 [1.08, 1.13] and adjusted relative cost ratio 1.07 [1.05, 1.10] for least compared to most deprived quintile). People in the most deprived quintile had higher mortality within 1 year compared to the least deprived quintile (adjusted hazard ratio [HR] 1.07 [1.03, 1.12]) as well as within 3 years (adjusted HR 1.09 [1.05, 1.13]). Admission to long-term care increased incrementally with material deprivation and those in the most deprived quintile had an adjusted HR of 1.33 (1.24, 1.43) compared to those in the least deprived quintile.DiscussionMaterial deprivation is a risk factor for increased costs and poor outcomes after stroke. Interventions targeting health inequities due to social determinants of health are needed.Classification of EvidenceThis study provides Class II evidence that the neighborhood-level material deprivation predicts direct health care costs.

Evaluating the relevance of published health utilities to the context of a cost-utility analysis (CUA) remains an essential, yet often overlooked, task. The objective of this study was to provide guidance on this process through the development of the Health utility Application Tool (HAT). We conducted semi-structured telephone interviews with Canadian stakeholders from reimbursement bodies, academia, and the pharmaceutical industry to identify current practices and perspectives of the application of the health utility literature to CUAs. An online survey with international members of the general health economics and outcomes research community was also conducted to gather opinions on key concepts. Based on the themes emerging from the interviews and online questionnaire, the HAT includes questions prompting investigators to consider the following constructs: similarity of the clinical condition in the health utility study and the CUA; similarity of health utility study participant demographics and the demographics of the CUA’s target population; similarity of the health state descriptions in the health utility study and the CUA; and the method of assigning utility weights. Considerations of transparency prompted additional items, including: means by which the health utility study was identified; type of respondents; study design; and measure used to collect health utility estimates. The HAT is intended to guide the evaluation of the applicability of published health utilities for a CUA, thus promoting transparency and accountability in the selection of model inputs.

Background Exercise maintenance interventions are needed for cardiac rehabilitation (CR) graduates to maintain moderate and vigorous-intensity physical activity (MVPA). We tested an exercise facilitator intervention (EFI) to promote exercise maintenance compared with usual care (UC) separately in men and women. Methods This was a 3-site, randomized (1:1), parallel-group, superiority trial (ECO-PCR). CR graduates were stratified by site and sex and randomly allocated (concealed). EFI participants received a face-to-face introductory session, 5 small-group counseling teleconferences, and 3 personal calls from a trained facilitator over 50 weeks. In-person assessments were undertaken at baseline and 26 and 52 weeks after randomization. The primary outcome was weekly minutes of MVPA, measured by accelerometer. Secondary outcomes were exercise capacity, risk factors, quality of life, and enrollment in community-based exercise programs. Effects were tested with the use of linear mixed models. Results A total of 449 CR graduates (135 women, 314 men) were randomised (n = 226 EFI, n = 223 UC). In the intention-to-treat analysis for men and for women, there were no significant effects for treatment or time on MVPA. In a planned secondary analysis that considered only those adherent to EFI (completed ≥ 66% of sessions; per-protocol), bouted MVPA (ie, in sustained bouts of ≥ 10 min) was higher in women in the EFI group (mean = 132.6 ± 135.2 min/wk at 52 weeks) compared with UC (111.8 ± 113.1; P = 0.013). Regarding secondary outcomes, in women, a treatment group main effect was observed for blood pressure (P = 0.011) and exercise capacity (P = 0.019; both per-protocol) favouring EFI; no other differences were observed. Conclusions In this trial of CR completers, an EFI showed promise for women, but was ineffective in men.

Published health utility studies are increasingly cited in cost–utility analyses to inform reimbursement decision-making. However, there is limited guidance for investigators looking to systematically evaluate the methodological quality of health utility studies or their applicability to decision contexts. To describe how health utility concepts are reflected in tools intended for use with the health economic literature, particularly with respect to the evaluation of methodological quality and context applicability. We reviewed the critical appraisal and reporting tools described in a 2012 report published by the Agency for Healthcare Research and Quality (AHRQ), supplemented with a keyword search of MEDLINE and EMBASE, to identify existing tools which include health utility constructs. From these tools, a list of relevant items was compiled and grouped into domain categories based on the methodological or applicability aspect they were directed toward. Of the 24 tools we identified, 12 contained items relevant to the evaluation of health utilities. Sixty-five items were considered relevant to the evaluation of quality, while 44 were relevant to the evaluation of applicability. Items were arranged into four domains: health state descriptions; selection and description of respondents; elicitation and measurement methods; and other considerations. As key inputs to cost–utility analyses, health utilities have the potential to significantly impact estimates of cost-effectiveness. Existing tools contain only general items related to the conduct or use of health utility studies. There is a need to develop tools that systematically evaluate the methodological quality and applicability of health utility studies.

Background High-quality estimates of health care costs are required to understand the burden of illness and to inform economic models. We estimated the costs associated with hepatitis C virus (HCV) infection from the public payer perspective in Ontario, Canada. Methods In this population-based retrospective cohort study, we identified patients aged 18-105 years diagnosed with chronic HCV infection in Ontario from 2003 to 2014 using linked administrative data. We allocated the time from diagnosis until death or the end of follow-up (Dec. 31, 2016) to 9 mutually exclusive health states using validated algorithms: no cirrhosis, no cirrhosis (RNA negative) (i.e., cured HCV infection), compensated cirrhosis, decompensated cirrhosis, hepatocellular carcinoma, both decompensated cirrhosis and hepatocellular carcinoma, liver transplantation, terminal (liver-related) and terminal (non-liver-related). We estimated direct medical costs (in 2018 Canadian dollars) per 30 days per health state and used regression models to identify predictors of the costs. Results We identified 48 239 patients with chronic hepatitis C, of whom 30 763 (63.8%) were men and 35 891 (74.4%) were aged 30-59 years at diagnosis. The mean 30-day costs were $798 (95% confidence interval [CI] $780-$816) (n = 43 568) for no cirrhosis, $661 (95% CI $630-$692) (n = 6422) for no cirrhosis (RNA negative), $1487 (95% CI $1375-$1599) (n = 4970) for compensated cirrhosis, $3659 (95% CI $3279-$4039) (n = 3151) for decompensated cirrhosis, $4238 (95% CI $3480-$4996) (n = 550) for hepatocellular carcinoma, $8753 (95% CI $7130-$10 377) (n = 485) for both decompensated cirrhosis and hepatocellular carcinoma, $4539 (95% CI $3746-$5333) (n = 372) for liver transplantation, $11 202 (95% CI $10 645-$11 760) (n = 3201) for terminal (liver-related) and $8801 (95% CI $8331-$9271) (n = 5278) for terminal (non-liver-related) health states. Comorbidity was the most significant predictor of total costs for all health states. Interpretation Our findings suggest that the financial burden of HCV infection is substantially higher than previously estimated in Canada. Our comprehensive, up-to-date cost estimates for clinically defined health states of HCV infection should be useful for future economic evaluations related to this disorder.

Objectives To examine long-term mortality, resource utilization, and healthcare costs in sepsis patients compared to hospitalized nonsepsis controls. Design Propensity-matched population-based cohort study using administrative data. Setting Ontario, Canada. Patients We identified a cohort of adults (≥ 18) admitted to hospitals in Ontario between April 1, 2012, and March 31, 2016, with follow-up to March 31, 2017. Sepsis patients were flagged using a validated International Classification of Diseases, 10th Revision-coded algorithm (Sepsis-2 definition), including cases with organ dysfunction (severe sepsis) and without (nonsevere). Remaining hospitalized patients were potential controls. Cases and controls were matched 1:1 on propensity score, age, sex, admission type, and admission date. Interventions None. Measurements and main results Differences in mortality, rehospitalization, hospital length of stay, and healthcare costs were estimated, adjusting for remaining confounders using Cox regression and generalized estimating equations. Of 270,669 sepsis cases, 196,922 (73%) were successfully matched: 64,204 had severe and 132,718 nonsevere sepsis (infection without organ dysfunction). Over follow-up (median 2.0 yr), severe sepsis patients had higher mortality rates than controls (hazard ratio, 1.66; 95% CI, 1.63-1.68). Both severe and nonsevere sepsis patients had higher rehospitalization rates than controls (hazard ratio, 1.53; 95% CI, 1.50-1.55 and hazard ratio, 1.41; 95% CI, 1.40-1.43, respectively). Incremental costs (Canadian dollar 2018) in sepsis cases versus controls at 1-year were: $29,238 (95% CI, $28,568-$29,913) for severe and $9,475 (95% CI, $9,150-$9,727) for nonsevere sepsis. Conclusions Severe sepsis was associated with substantially higher long-term risk of death, rehospitalization, and healthcare costs, highlighting the need for effective postdischarge care for sepsis survivors.

Patients identified as having chronic hepatitis C (CHC) infection can be effectively and rapidly treated using direct-acting antiviral agents. However, there remains a substantial burden of subclinical undetected infection. This study estimates the prevalence and undiagnosed proportion of CHC in British Columbia (BC) and Ontario, Canada using a model-based approach, informed by provincial population-level health administrative data. A two-step approach was used: Step 1) Two population-based retrospective analyses of administrative health data for a cohort of British Columbians and a cohort of Ontarians with CHC were conducted to generate population-level statistics of CHC-related health events; Step 2) using a validated natural history model of hepatitis C virus (HCV) infection and back-calculated the historical prevalence of CHC from the data collected in Step 1. Our retrospective study found that, in BC and Ontario, the number of newly diagnosed CHC cases is declining yearly while the complications of the disease are increasing yearly. BC had a 2014 CHC prevalence of 1.04% (95% CI: 0.84%-1.44%), with 33.3% (95% CI: 25.5%-42.0%) of CHC cases undiagnosed. Ontario had a 2014 CHC prevalence of 0.91% (95% CI: 0.83%-1.02%) with 36.0% (95% CI: 31.2%-38.9%) of CHC cases undiagnosed. Our study offers robust estimates based on the integration of a validated natural history model with population-level health administrative data on HCV-related events, which can provide vital evidence for policy-makers to develop appropriate policies to achieve the elimination targets. Our approach can also be applied and used to produce robust region-specific estimates in other countries.

Background Androgen deprivation therapy (ADT) for prostate cancer (PCa) is associated with metabolic perturbations and declines in bone mineral density (BMD). Exercise interventions provide multiple health benefits to older men on ADT; however, their effect on metabolic biomarkers and BMD remains unclear. Methods A secondary analysis of a phase II randomized controlled trial was conducted to assess the effect of a six-month moderate-intensity aerobic and resistance exercise program on metabolic biomarkers and BMD in men on ADT. Participants were randomized to three different exercise delivery models: personal training; supervised group exercise; or home-based exercise. Analysis of metabolic biomarkers (lipid profile and glucose) was conducted at baseline, six and twelve months. BMD of the lumbar spine, femoral neck and hip were assessed at baseline and twelve months. Both within- and between-group analyses of change scores adjusted for baseline values were performed. Results Forty-eight men (mean age 69.8y) were enrolled. Baseline values of metabolic biomarkers and BMD were comparable between groups and the three groups were combined for the primary analysis. At six months, no changes in metabolic biomarkers were found; however, at twelve months low-density lipoprotein (+0.28 mmol/L; 95%CI, 0.04 to 0.51) and total cholesterol (+0.31 mmol/L; 95%CI, 0.00 to 0.61) were significantly increased from baseline. No changes were found in BMD. In a secondary between-group analysis, no improvements were observed for any metabolic biomarker or BMD measurement. Conclusions Different exercise prescription parameters (modality and intensity) or combined diet/exercise interventions may be needed to foster favorable metabolic and skeletal adaptations during ADT.

Objectives There is a growing interest in health technology assessment and economic evaluations in developing countries such as Ethiopia. The objective of this study was to derive an EQ-5D-5L value set from the Ethiopian general population to facilitate cost utility analysis. Methods A nationally representative sample (N = 1050) was recruited using a stratified multistage quota sampling technique. Face-to-face, computer-assisted interviews using the EuroQol Portable Valuation Technology (EQ-PVT) protocol of composite time trade-off (c-TTO) and discrete choice experiments (DCEs) were undertaken to elicit preference scores. The feasibility of the EQ-PVT protocol was pilot tested in a sample of the population (n = 110). A hybrid regression model combining c-TTO and DCE data was used to estimate the final value set. Results In the pilot study, the acceptability of the tasks was good, and there were no special concerns with undertaking the c-TTO and DCE tasks. The coefficients generated from a hybrid model were logically consistent. The predicted values for the EQ-5D-5L ranged from −0.718 to 1. Level 5 anxiety/depression had the largest impact on utility decrement (−0.458), whereas level 5 self-care had the least impact (−0.222). The maximum predicted value beyond full health was 0.974 for the 11112 health state. Conclusions This is the first EQ-5D-5L valuation study in Africa using international valuation methods (c-TTO and DCE) and also the first using the EQ-PVT protocol to derive a value set. We expect that the availability of this value set will facilitate health technology assessment and health-related quality-of-life research and inform policy decision making in Ethiopia.

Health utility is a quantitative global measure of patients’ health status. This retrospective cohort study aimed to compare health utilities of patients with mild to moderate versus severe carpal tunnel syndrome and determine inter-instrumental agreement. Health utilities of 29 patients with varying severity of carpal tunnel syndrome were measured indirectly by Short-Form Sixth Dimension and EuroQol 5D questionnaire and directly by Chained Standard Gamble and a visual analogue scale. Health utility was 0.69 for Short-Form Sixth Dimension, 0.78 for EuroQol 5D Questionnaire, 0.98 for Chained Standard Gamble, and 0.76 for the visual analogue scale. There was a significant inter-instrumental agreement between three of the instruments, but not the Chained Standard Gamble. The difference in health utilities between patients with mild or moderate versus severe carpal tunnel syndrome was significant only for the EuroQol 5D questionnaire. We conclude based on our results that there are no clear indications on how health utilities can be integrated into decision analysis models and economic evaluation regarding carpal tunnel syndrome of various severities . Level of evidence: IV

Background We sought to develop mapping functions that use EORTC responses to approximate health utility (HU) scores for patients with head and neck cancer (HNC). Methods In total, 209 outpatients with HNC completed the EORTC QLQ-C30 & QLQ-H&N35 (EORTC), EQ-5D-5L and the HUI-3. Results of the EORTC were mapped onto both EQ-5D-5L and HUI-3 scores using ordinary least squares regression and two-part models. Results The OLS model mapping EORTC onto the EQ-5D-5L performed best (adjusted R2 = .75, 10-fold cross-validation RMSE = 0.064, MAE 0.050). The HUI-3 model mapping onto EORTC through OLS was more limited (adjusted R2 = .5746, 10-fold cross cross-validation RMSE = 0.168, MAE 0.080). The EQ-5D-5L model was able to discriminate between certain clinical indices of disease severity on subgroup analysis. Conclusion The EORTC to EQ-5D-5L mapping algorithm has good predictive validity and may enable researchers to translate EORTC scores into HU scores for head and neck patients with cancer.

Objectives: To conduct a cost-utility analysis comparing drug strategies involving octreotide, lanreotide, pasireotide, and pegvisomant for the treatment of patients with acromegaly who have failed surgery, from a Brazilian public payer perspective. Methods: A probabilistic cohort Markov model was developed. One-year cycles were employed. The patients started at 45 years of age and were followed lifelong. Costs, efficacy, and quality of life parameters were retrieved from the literature. A discount rate (5%) was applied to both costs and efficacy. The results were reported as costs per quality-adjusted life year (QALY), and incremental cost-effectiveness ratios (ICERs) were calculated when applicable. Scenario analyses considered alternative dosages, discount rate, tax exemption, and continued use of treatment despite lack of response. Value of information (VOI) analysis was conducted to explore uncertainty and to estimate the costs to be spent in future research. Results: Only lanreotide showed an ICER reasonable for having its use considered in clinical practice (R$ 112,138/US$ 28,389 per QALY compared to no treatment). Scenario analyses corroborated the base-case result. VOI analysis showed that much uncertainty surrounds the parameters, and future clinical research should cost less than R$ 43,230,000/US$ 10,944,304 per year. VOI also showed that almost all uncertainty that precludes an optimal strategy choice involves quality of life. Conclusions: With current information, the only strategy that can be considered cost-effective in Brazil is lanreotide treatment. No second-line treatment is recommended. Significant uncertainty of parameters impairs optimal decision-making, and this conclusion can be generalized to other countries. Future research should focus on acquiring utility data.

Background: Cancer in children presents unique issues for diagnosis, treatment and survivorship care. Phase-specific comparative cost estimates are important for informing healthcare planning. Objectives: The aim of this paper is to compare direct medical costs of childhood cancer by phase of care in British Columbia (BC) and Ontario (ON). Methods: For cancer patients diagnosed at 50% higher in ON. Phase-specific in-patient hospital costs (the major cost category) represented 63%–82% of ON costs, versus 43%–73% of BC costs. Phase-specific diagnostic tests and procedures accounted for 1.0%–3.4% of ON costs and 2.8%–13.0% of BC costs. Conclusions: There are substantial cost differences between these two Canadian provinces, BC and ON, possibly identifying opportunities for healthcare planning improvement.

ImportanceExcessive bleeding requiring fibrinogen replacement is a serious complication of cardiac surgery. However, the relative cost-effectiveness of the 2 available therapies—fibrinogen concentrate and cryoprecipitate—is unknown.ObjectiveTo determine cost-effectiveness of fibrinogen concentrate vs cryoprecipitate for managing active bleeding in adult patients who underwent cardiac surgery.Design, Setting, and ParticipantsA within-trial economic evaluation of the Fibrinogen Replenishment in Surgery (FIBERS) randomized clinical trial (February 2017 to November 2018) that took place at 4 hospitals based in Ontario, Canada, hospitals examined all in-hospital resource utilization costs and allogeneic blood product (ABP) transfusion costs incurred within 28 days of surgery. Participants included a subset of 495 adult patients from the FIBERS trial who underwent cardiac surgery and developed active bleeding and acquired hypofibrinogenemia requiring fibrinogen replacement.InterventionsFibrinogen concentrate (4 g per dose) or cryoprecipitate (10 units per dose) randomized (1:1) up to 24 hours postcardiopulmonary bypass.Main Outcomes and MeasuresEffectiveness outcomes included number of ABPs administered within 24 hours and 7 days of cardiopulmonary bypass. ABP transfusion (7-day) and in-hospital resource utilization (28-day) costs were evaluated and a multivariable net benefit regression model built for the full sample and predefined subgroups.ResultsPatient level costs for 495 patients were evaluated (mean [SD] age 59.2 [15.4] years and 69.3% male.) Consistent with FIBERS, ABP transfusions and adverse events were similar in both treatment groups. Median (IQR) total 7-day ABP cost was CAD $2280 (US dollars [USD] $1697) (CAD $930 [USD $692]-CAD $4970 [USD $3701]) in the fibrinogen concentrate group and CAD $2770 (USD $1690) (IQR, CAD $1140 [USD $849]-CAD $5000 [USD $3723]) in the cryoprecipitate group. Median (interquartile range) total 28-day cost was CAD $38 180 (USD $28 431) $(IQR, CAD $26 350 [USD $19 622]-CAD $65 080 [USD $48 463]) in the fibrinogen concentrate group and CAD $38 790 (USD $28 886) (IQR, CAD $26 180 [USD $19 495]-CAD $70 380 [USD $52 409]) in the cryoprecipitate group. After exclusion of patients who were critically ill before surgery (11%) due to substantial variability in costs, the incremental net benefit of fibrinogen concentrate vs cryoprecipitate was positive (probability of being cost-effective 86% and 97% at $0 and CAD $2000 (USD $1489) willingness-to-pay, respectively). Net benefit was highly uncertain for nonelective and patients with critical illness.Conclusions and RelevanceFibrinogen concentrate is cost-effective when compared with cryoprecipitate in most bleeding adult patients who underwent cardiac surgery with acquired hypofibrinogenemia requiring fibrinogen replacement. The generalizability of these findings outside the Canadian health system needs to be verified.

The use of economic evaluations of end-of-life interventions may be limited by an incomplete appreciation of how patients and society perceive value at end of life. The objective of this study was to evaluate how patients, caregivers, and society value gains in quantity of life and quality of life (QOL) at the end of life. The validity of the assumptions underlying the use of the quality-adjusted life-years (QALY) as a measure of preferences at end of life was also examined.MEDLINE, Embase, CINAHL, PsycINFO, and PubMed were searched from inception to February 22, 2021. Original research studies reporting empirical data on healthcare priority setting at end of life were included. There was no restriction on the use of either quantitative or qualitative methods. Two reviewers independently screened, selected, and extracted data from studies. Narrative synthesis was conducted for all included studies. The primary outcomes were the value of gains in quantity of life and the value of gains in QOL at end of life.A total of 51 studies involving 53 981 participants reported that gains in QOL were generally preferred over quantity of life at the end of life across stakeholder groups. Several violations of the underlying assumptions of the QALY to measure preferences at the end of life were observed.Most patients, caregivers, and members of the general public prioritize gains in QOL over marginal gains in life prolongation at the end of life. These findings suggest that policy evaluations of end-of-life interventions should favor those that improve QOL. QALYs may be an inadequate measure of preferences for end-of-life care thereby limiting their use in formal economic evaluations of end-of-life interventions.