BACKGROUND:This study was conducted to evaluate the population-level healthcare resource utilization (HCRU) and costs for men diagnosed with low-risk prostate cancer (PCa) either on active surveillance (AS) or not on AS, in which AS was defined as receiving no treatment within 1 year of diagnosis and two biopsies. METHODS:AS men aged 40 to 105 years, diagnosed with stage I or II PCa, had a prostate-specific antigen (PSA) level < 20 ng/mL, a Gleason score between 5 and 7, and were matched (1:1) with men not receiving AS. The index date is defined as the date 1 year after PCa diagnosis. HCRU and costs were assessed using a macro-based costing methodology and costs standardized to 2023 CAD. Means (SD) and medians (interquartile ranges) per person-year values were reported annually. RESULTS:During the year leading up to the index date, the mean number of HCRU per patient-year (PPY) was significantly lower for the AS cases versus non-AS in terms of cancer clinic visits (1.7 vs. 26.7), hospital outpatient clinic visits (3.6 vs. 4.9), all physician visits (16.3 vs. 17.5), and specialist visits (10.7 vs. 11.6). The mean overall cost PPY was $6100 ± $12,400 for AS cases and $10,400 ± $17,800 for non-AS men (median overall cost PPY= $3500 [IQR: $2100-$5700] vs. $3700 [IQR: $2100-$7200] (p = 0.0001, respectively). CONCLUSIONS:HCRU and costs calculated for AS and non-AS low-risk PCa men indicate the cost savings potential for AS.

The article "Defining Early Health Technology Assessment: Building Consensus Using Delphi Technique" offers a useful definition of early HTA (eHTA) as an assessment to inform development, research, or investment decisions. From a lifecycle HTA (LC-HTA) perspective, we emphasize that eHTA should be viewed as part of a broader, continuous evidence and decision-making process rather than a stand-alone activity. Integrating eHTA within LC-HTA strengthens alignment across phases of technology development, supports anticipatory evidence planning, and promotes adaptive reassessment over time-enabling more coherent, learning-oriented HTA systems.

Objective: The objective of this scoping review is to develop a list of items for potential inclusion in the Preferred Reporting Items for Systematic reviews and Meta-Analyses (PRISMA) reporting guidelines for network meta-analysis (NMA), scoping reviews (ScRs), and rapid reviews (RRs). Introduction: The PRISMA extensions for NMA and ScRs were published in 2015 and 2018. However, since then, their methodologies and innovations, including automation, have evolved. There is no reporting guideline for RRs. In 2020, an updated PRISMA statement was published, reflecting advances in the conduct and reporting of systematic reviews. These advances are not yet incorporated into these PRISMA extensions. We will update our previous methods for scoping reviews to inform the update of PRISMA-NMA and PRISMA-ScR as well as the development of the PRISMA-RR reporting guidelines. Inclusion criteria: This review will include any study design evaluating the completeness of reporting, offering reporting guidance, or assessing methods relevant to NMA, ScRs, or RRs. Editorial guidelines and tutorials that describe items related to reporting completeness will also be eligible. Methods: We will follow the JBI guidance for scoping reviews. For each PRISMA extension, we will i) search multiple electronic databases from inception to present, ii) search for unpublished studies, and iii) scan the reference lists of included studies. There will be no language limitations. Screening and data extraction will be conducted by 2 researchers independently. A third researcher will resolve discrepancies. We will conduct frequency analyses of the identified items. The final list of items will be considered for potential inclusion in the relevant PRISMA reporting guidelines. Review registration: NMA protocol (OSF: osf.io/7bkwy); ScR protocol (OSF: osf.io/7bkwy); RR protocol (OSF: osf.io/3jcpe); EQUATOR registration link: https://www.equator-network.org/library/reporting-guidelines-under-development/reporting-guidelines-under-development-for-systematic-reviews/

INTRODUCTION:Clinical guidelines recommend active surveillance (AS) as the preferred strategy for men with localized grade group (GG) 1 prostate cancer (PCa). We determined if the percentage of GG1 PCa patients in Ontario, Canada, managed by AS changed after the introduction of AS clinical guidelines and assessed adherence to the recommended followup protocol. METHODS:Using Ontario administrative databases, we conducted a time series analysis (autoregressive integrated moving average [ARIMA] models) in a population-based cohort of men diagnosed with GG1 PCa (2010-2018). Men were classified as managed by AS if they had repeat (confirmatory) biopsy within two years. Sensitivity analyses (treatment classification variation) and secondary analyses (low-risk GG1 and GG2 PCa) were conducted. RESULTS:We identified 12 236 eligible GG1 PCa patients, of which 7749 (63.3%) were initially managed by AS. Percentage AS increased from 44% in 2010 to 81% in 2018. Interrupted time series analysis estimated an immediate step change of 6.2 percentage points (95% confidence interval [CI] 3.0, 9.4) and a difference in slope of -2.3 percentage points (95% CI -6.9, 2.3) per year. Findings were robust to sensitivity analyses and similar for low-risk PCa. Adherence to monitoring and AS uptake in GG2 patients were not associated with guideline publication. Limitations include lack of treatment intent information in administrative data. CONCLUSIONS:The use of AS for low-grade PCa patients in Ontario increased from almost one in two patients in 2010 to four in five patients in 2017/2018. Adoption appeared to reflect the growing acceptance of AS prior to the guidelines, as well as an increase in response to the guideline introduction.

Background This study assessed health care system costs and resource utilization for adult women with breast cancer in Ontario, Canada. The goal was to update costs by stage, age, and phase of care from a health care system perspective. Data and methods A retrospective analysis was conducted using linked population-based administrative data. The study included women diagnosed with breast cancer from 2017 to 2021, with follow-up data until 2022. Cases were matched with controls in a 1:5 ratio using birth year, local health integrative network, income quintile, and resource utilization band at baseline. Incremental costs were estimated using linear regression. The modified income quintile was the neighbourhood- or area-level income quintile. Results Among the 37,133 cases matched with 185,665 controls, the average age at diagnosis was 62 years. For the entire study duration, cases incurred an additional cost of $27,485 per year, compared with controls. Costs rose with disease severity, ranging from $15,588 for stage Ito $137,319 for stage IV. The highest incremental costs occurred during the first 12 months after diagnosis (initial: $43,408), followed by the last 12 months before death (terminal: $25,940), and then interim years (continuous: $9,533 per year). Additionally, the incremental cost of breast cancer was higher when diagnosis was before age 70 ($28,415), compared with diagnosis at age 70 and older ($25,254). Interpretation The findings align with previous studies on breast cancer costs for the health care system. Additionally, variations in costs based on disease severity, care phase, and age were emphasized, highlighting higher costs for metastatic breast cancer cases, women younger than 70 years, and the initial 12 months following diagnosis.

Health Canada’s Notice of Compliance with conditions (NOC/c) policy provides expedited access to promising new therapies for serious conditions. This retrospective cohort analysis sought to examine the use of the NOC/c policy for oncology drugs, to determine whether confirmatory studies are aligned with the approved indication, and to assess the impact of the NOC/c policy by quantifying the number of oncology drugs approved under this policy that become the standard of care treatment for their respective indications. The findings suggest that Health Canada’s NOC/c policy has been successful in providing expedited access to promising new therapies. However, ensuring that confirmatory trials are under way at the time of NOC/c issuance may provide assurance that studies addressing the uncertainty associated with NOC/c will not be unnecessarily prolonged. The inclusion of real-world evidence (RWE) as part of confirmatory evidence may help fill gaps.

Background: A prospective cohort study was undertaken within the PERSPECTIVE I&I project to evaluate healthcare resource utilization and costs associated with breast cancer risk assessment and screening and overall costs stratified by risk level, in Ontario, Canada. Methods: From July 2019 to December 2022, 1997 females aged 50 to 70 years consented to risk assessment and received their breast cancer risk level and personalized screening action plan in Ontario. The mean costs for risk-stratified screening-related activities included risk assessment, screening and diagnostic costs. The GETCOST macro from the Institute of Clinical Evaluative Sciences (ICES) assessed the mean overall healthcare system costs. Results: For the 1997 participants, 83.3%, 14.4% and 2.3% were estimated to be average, higher than average, and high risk, respectively (median age (IQR): 60 [56–64] years). Stratification into the three risk levels was determined using the validated multifactorial CanRisk prediction tool that includes family history information, a polygenic risk score (PRS), breast density and established lifestyle/hormonal risk factors. The mean number of genetic counseling visits, mammograms and MRIs per individual increased with risk level. High-risk participants incurred the highest overall mean risk-stratified screening-related costs in 2022 CAD (±SD) at CAD 905 (±269) followed by CAD 580 (±192) and CAD 521 (±163) for higher-than-average and average-risk participants, respectively. Among the breast screening-related costs, the greatest cost burden across all risk groups was the risk assessment cost, followed by total diagnostic and screening costs. The mean overall healthcare cost per participant (±SD) was the highest for the average risk participants with CAD 6311 (±19,641), followed by higher than average risk with CAD 5391 (±8325) and high risk with CAD 5169 (±7676). Conclusion: Although high-risk participants incurred the highest risk-stratified screening-related costs, their costs for overall healthcare utilization costs were similar to other risk levels. Our study underscored the importance of integrating risk stratification as part of the screening pathway to support breast cancer detection at an earlier and more treatable stage, thereby reducing costs and the overall burden on the healthcare system.

Patients with lung cancer can experience significant psychological morbidities including depression. We characterize patterns and factors associated with interventions for symptoms of depression in stage IV non-small cell lung cancer (NSCLC). We conducted a population-based cohort study using health services administrative data in Ontario, Canada of stage IV NSCLC diagnosed from January 2007 to September 2018. A positive symptom of depression score was defined by reporting at least one ESAS (Edmonton Symptom Assessment System) depression score ≥ 2 following diagnosis until the end of follow-up (September 2019). Patient factors included age, sex, comorbidity burden, rurality of residence, and neighbourhood income quintile. Interventions included psychiatry assessment, psychology referral, social work referral and anti-depressant medical therapy (for patients ≥ 65 years with universal drug coverage). Multivariable modified Poisson regression models were used to examine the association between patient factors and intervention use for patients who reported symptoms of depression. In the cohort of 13,159 patients with stage IV NSCLC lung cancer, symptoms of depression were prevalent (71.4

BACKGROUND AND OBJECTIVE:Real-world evidence (RWE) can complement and fill knowledge gaps from randomized controlled trials to assist in health-technology assessment (HTA) for regulatory decision-making. However, the generation of RWE is an intricate process with many sequential decision points, and different methods and approaches may impact the quality and reliability of evidence. Standardization and transparency in reporting these decisions is imperative to appraise RWE and incorporate it into HTA decision-making. A partnership between Canadian health system stakeholders, namely, Health Canada and Canada's Drug Agency (formerly the Canadian Agency for Drugs and Technologies in Health), was established to develop guidance for the standardization of reporting of RWE for regulatory and HTA decision-making in Canada. STUDY DESIGN AND SETTING:A collaborative initiative to create structured guidance for RWE reporting in the context of regulatory and HTA decision-making. RESULTS:The developed guidance aims to standardize and ensure transparent reporting of RWE to improve its reliability and usefulness in regulatory and HTA processes. CONCLUSION:This guidance can be adapted for other jurisdictions and will have future extensions to incorporate emerging issues with RWE and HTA decision-making.

6650 Background: To date, economic analyses of tissue-based next generation sequencing genomic profiling (NGS) have required models with multiple assumptions, with little real-world evidence on overall survival (OS), clinical trial enrollment or end-of-life (EOL) quality of care. The OCTANE clinical trial (NCT02906943) is a prospective study evaluating the role of NGS for advanced solid tumors in Ontario, Canada. We performed a cost consequence analysis of OCTANE. Methods: We undertook a longitudinal, propensity score-matched retrospective cohort study using linked administrative data. OCTANE patients (pts) at Princess Margaret Cancer Centre from August 2016 until March 2019 undergoing NGS panel testing (555 or 163-gene panels) were matched with contemporary controls from across Ontario not enrolled in OCTANE. Patients were matched according to 19 variables including age, sex, place of residence, tumor site, symptom burden, income quintile, comorbidities and prior lines of systemic therapy. Primary outcomes were mean per capita health care costs (2019 Canadian dollars [CAD]) from the public payer’s perspective, OS, clinical trial enrollment and EOL quality metrics. Full 2-year follow-up data was available. Sensitivity analyses considered alternative matched cohort specifications. Results: There were 782 OCTANE pts with 782 matched controls. Variables were balanced after matching (standardized difference [std. diff.]<0.10). Most common tumor sites were: Ovary (30.4%), endometrium (15.0%) breast (12.3%) and colon (8.6%). OCTANE pts had higher mean healthcare costs than controls ($79,702 vs. $59,550), mainly due to costs of oncology visits ($33,165 vs. $26,197), outpatient clinic visits ($8,696 vs. $5,114) and emergency visits ($1,723 vs. $1,373) (all p<0.05). Publicly funded drug costs were less for OCTANE pts ($20,015 vs. $24,465). Overall, OCTANE enrollment was not associated with improved OS (restricted mean survival time (RMST) [standard error]: 1.50 (±0.03) vs. 1.44 (±0.03) years, log-rank p=0.153), but OCTANE was associated with longer OS in ovarian cancer (RMST: 1.69 (±0.05) vs. 1.45 (±0.06) years, p=0.011) and biliary tract tumors (RMST: 1.16 (±0.13) vs. 0.80 (±0.11) years, p=0.02). Importantly, OCTANE correlated with increased clinical trial enrollment (25.5% vs. 9.5%, p<0.001) and better EOL quality due to fewer deaths in hospital (10.2% vs 16.4%, p=0.003). Results were robust in sensitivity analysis. Conclusions: There was an increase in healthcare costs associated with NGS testing for advanced cancers. The impact on OS was not significant in the overall population, but varied across tumor types. OCTANE was associated with greater trial enrollment, lower publicly funded drug costs and fewer in hospital deaths suggesting important considerations in determining the value of NGS for advanced cancers. Clinical trial information: NCT02906943 .

INTRODUCTION AND OBJECTIVE: The Precise study, a prospective randomized trial, demonstrated that MRI with only targeted biopsy was non-inferior to systematic TRUS guided biopsies in the detection of GG 2 PCa. An unanswered question is the outcome in those patients who avoided a biopsy due to a negative MRI.We addressed the outcome of patients on this trial who were unbiopsied, or had a negative or GG1 targeted biopsy, compared to those whose systematic biopsy was negative or GG1, with respect to the rate of subsequent prostate cancer diagnosis based on a 2 year MRI performed in all patients. In addition, we used administrative provincial data to compare the net costs of systematic biopsy vs MRI and targeted biopsy. METHODS: The study was carried out at 5 Canadian centres. The Precise trial accrued 453 biopsy-naïve men with a clinical suspicion of PCa advised to have a prostate biopsy. All those not initially diagnosed with GG 2 PCa (unbiopsied due to a negative MRI, or a negative biopsy, or GG1 disease if biopsied) were entered in the follow up study. All Ontario patients were entered in the economic analysis. The intervention was an MRI at year 2 in all men in both MRI and systematic biopsy groups. Biopsy was performed for PiRads 3 or clinical suspicion. RESULTS: 73 patients in the MRI arm and 69 patients in the systematic biopsy arm had a 2 year evaluable MRI. In the MRI and syst bx arm, 70% and 72% had a negative 2 year MRI. Of the 16 patients in the systematic biopsy arm who had a 2 year biopsy, 8 were negative, 2 were GG1, and 6 were GG 2. In the MRI arm, 8 were biopsied; 4 were negative, and 4 were GG 2. At 2 years, in the Syst Bx and MRI groups, 27% and 30% either were diagnosed with GG 2. disease; treated, died, or progressed. 15% from the TRUS biopsy group had a hospital visit after biopsy compared to less 6% from the MRIþ group. The mean per person per year (PPPY) costs for the TRUS and All MRI groups (MRIand MRIþ) were $7,828 and $8,525, respectively. CONCLUSIONS: After 2 years of follow up including an MRI in all patients, there was no difference in the rate of CS Pca diagnosis between the MRI with only targeted biopsy and systematic biopsy groups, despite 40% of men in the MRI group avoiding biopsy. These patients will be followed for an additional 6 years to confirm this finding. Economic analysis using a linked provincial databased demonstrated no significant difference in net cost between the 2 strategies. This data reinforces that an imaging first diagnostic strategy is safe and costeffective. ClinicalTrials.gov Identifier: NCT02936258

BACKGROUND:Despite the fact that direct oral anticoagulants (DOACs) are favoured over warfarin for stroke prevention in patients with non-valvular atrial fibrillation (NVAF), physicians need to maintain competence in using and monitoring warfarin since many patients have contraindications or other barriers to using DOACs. Unlike DOACs, warfarin therapy requires regular blood testing to ensure that it is within a target range to ensure efficacy and safety. There is limited real-world data on the adequacy of warfarin control and the cost and burden of monitoring warfarin therapy in Canadian NVAF patients.OBJECTIVES:In a large cohort of Canadian patients with NVAF on warfarin we assessed time in therapeutic range (TTR), determinants of TTR, process of care, direct costs, health related quality of life and loss of work time and productivity related to warfarin therapy.METHODS:Five hundred and fifty one patients with NVAF, either newly initiated or stable on warfarin were prospectively enrolled across 9 Canadian provinces from primary care practices and anticoagulant clinics. Participating physicians provided baseline demographic and medical information. Patients completed diaries for 48 weeks, capturing information about International Normalized Ratio (INR) test results, test locations, process of INR monitoring, direct costs of travel, health-related quality of life and work productivity measures. TTR was estimated using linear interpolation of INR results and linear regression used to investigate associations between TTR and factors (defined a priori).RESULTS:Four hundred and eighty (87.1%) patients had complete follow-up with an overall TTR of 74.4% based on 7,175 physician-reported INR values from 501 patients. 88% of this cohort were monitored through routine medical care (RMC). The average number of INRs per patient during the 48-week period was 14.1 (standard deviation (SD) = 8.3) tests with a mean duration of 23.8 (SD = 11.1) days between tests. We did not find a relationship between TTR and age, sex, presence of major comorbidities, patient's province of residence or rural vs. urban residence. 12% of patients monitored through anticoagulant clinics had significantly better TTR than patients monitored through RMC (82% vs. 74%; 95% confidence interval: -13.8, -1.2; p = 0.02). Health related quality of life utility values were high and remained consistent throughout the study. The majority of patients reported no impact on either work productivity or impairment of regular activities due to being on long-term warfarin treatment.CONCLUSIONS:We showed excellent overall TTR in an observed Canadian cohort, with monitoring through a dedicated anticoagulant clinic being associated with a statistically and clinically significant improvement in TTR. The burden of warfarin therapy on patients' health related quality of life or daily work and activities was low.

Pain is a common symptom in stage IV non-small cell lung cancer (NSCLC). The objective of the study was to examine the use of interventions and factors associated with interventions for pain. A population-based cohort study in Ontario, Canada was conducted with patients diagnosed with stage IV NSCLC from January 2007 to September 2018. An Edmonton Symptom Assessment System (ESAS) score of ≥4 defined moderate-to-severe pain following diagnosis. The study cohort included 13,159 patients, of which 68.5% reported at least one moderate-to-severe pain score. Most patients were assessed by a palliative care team (85.4%), and the majority received radiation therapy (73.2%). The use of nerve block was rare (0.8%). For patients ≥65 years of age who had drug coverage, 59.6% received an opiate prescription. Patients with moderate-to-severe pain were more likely to receive palliative assessment or radiation therapy compared to patients with none or mild pain. Patients aged ≥70 years and with a greater comorbidity burden were associated with less likelihood to receive radiation therapy. Patients from rural/non-major urban residence and with a greater comorbidity burden were also less likely to receive palliative care assessment. Factors associated with interventions for pain are described to inform future symptom management in this population.

Concurrent cohorts of 644,932 women aged 50–74 screened annually due to family history, dense breasts or biennially in the Ontario Breast Screening Program (OBSP) from 2011–2014 were linked to provincial administrative datasets to determine health system resource utilization and costs. Age-adjusted mean and median total healthcare costs (2018 CAD) and incremental cost differences were calculated by screening outcome and compared by recommendation using regression models. Healthcare costs were compared overall and 1 year after a false positive (n = 46,081) screening mammogram and 2 years after a breast cancer diagnosis (n = 6011). Mean overall healthcare costs by age were highest for those 60–74, particularly with annual screening for family/personal history (CAD 5425; 95% CI: 5308 to 5557) compared to biennial. Although the mean incremental cost difference was higher (23.4%) by CAD 10,235 (95% CI: 6141 to 14,329) per breast cancer for women screened annually for density ≥ 75% compared to biennially, the cost difference was 12.0% lower (−CAD 461; 95% CI: −777 to −114) per false positive result. In contrast, for women screened annually for family/personal history, the mean cost difference per false positive was 19.7% higher than for biennially (CAD 758; 95% CI: 404 to 1118); however, the cost difference per breast cancer was only slightly higher (2.5%) by CAD 1093 (95% CI: −1337 to CAD 3760). Understanding that associated costs of annual compared to biennial screening may balance out by age and outcome can assist decision-making regarding the use of limited healthcare resources.

Importance:Melanoma treatment has evolved during the past decade with the adoption of adjuvant and palliative immunotherapy and targeted therapies, with an unclear impact on health care costs and outcomes in routine practice. Objective:To examine changes in health care costs, overall survival (OS), and time toxicity associated with primary treatment of melanoma. Design, Setting, and Participants:This cohort study assessed a longitudinal, propensity score (PS)-matched, retrospective cohort of residents of Ontario, Canada, aged 20 years or older with stages II to IV cutaneous melanoma identified from the Ontario Cancer Registry from January 1, 2018, to March 31, 2019. A historical comparison cohort was identified from a population-based sample of invasive melanoma cases diagnosed from the Ontario Cancer Registry from January 1, 2007, to December 31, 2012. Data analysis was performed from October 17, 2022, to March 13, 2023. Exposures:Era of melanoma diagnosis (2007-2012 vs 2018-2019). Main Outcomes and Measures:The primary outcomes were mean per-capita health care and systemic therapy costs (Canadian dollars) during the first year after melanoma diagnosis, time toxicity (days with physical health care contact) within 1 year of initial treatment, and OS. Standardized differences were used to compare costs and time toxicity. Kaplan-Meier methods and Cox proportional hazards regression were used to compare OS among PS-matched cohorts. Results:A PS-matched cohort of 731 patients (mean [SD] age, 67.9 [14.8] years; 437 [59.8%] male) with melanoma from 2018 to 2019 and 731 patients (mean [SD] age, 67.9 [14.4] years; 440 [60.2%] male) from 2007 to 2012 were evaluated. The 2018 to 2019 patients had greater mean (SD) health care (including systemic therapy) costs compared with the 2007 to 2012 patients ($47 886 [$55 176] vs $33 347 [$31 576]), specifically for stage III ($67 108 [$57 226] vs $46 511 [$30 622]) and stage IV disease ($117 450 [$79 272] vs $47 739 [$37 652]). Mean (SD) systemic therapy costs were greater among 2018 to 2019 patients: stage II ($40 823 [$40 621] vs $10 309 [$12 176]), III ($55 699 [$41 181] vs $9764 [$12 771]), and IV disease ($79 358 [$50 442] vs $9318 [$14 986]). Overall survival was greater for the 2018 to 2019 cohort compared with the 2007 to 2012 cohort (3-year OS: 74.2% [95% CI, 70.8%-77.2%] vs 65.8% [95% CI, 62.2%-69.1%], hazard ratio, 0.72 [95% CI, 0.61-0.85]; P < .001). Time toxicity was similar between eras. Patients with stage IV disease spent more than 1 day per week (>52 days) with physical contact with the health care system by 2018 to 2019 (mean [SD], 58.7 [43.8] vs 44.2 [26.5] days; standardized difference, 0.40; P = .20). Conclusions and Relevance:This cohort study found greater health care costs in the treatment of stages II to IV melanoma and substantial time toxicity for patients with stage IV disease, with improvements in OS associated with the adoption of immunotherapy and targeted therapies. These health system-wide data highlight the trade-off with adoption of new therapies, for which there is a greater economic burden to the health care system and time burden to patients but an associated improvement in survival.

AbstractBackgroundThe efficacy‐effectiveness gap between randomized trial and real‐world evidence regarding the clinical benefit of ipilimumab for metastatic melanoma (MM) has been well characterized by previous literature, consistent with initial concerns raised by health technology assessment agencies (HTAs). As these differences can significantly impact cost‐effectiveness, it is critical to assess the real‐world cost‐effectiveness of second‐line ipilimumab versus non‐ipilimumab treatments for MM.MethodsThis was a population‐based retrospective cohort study of patients who received second‐line non‐ipilimumab therapies between 2008 and 2012 versus ipilimumab treatment between 2012 and 2015 (after public reimbursement) for MM in Ontario. Using a 5‐year time horizon, censor‐adjusted and discounted (1.5%) costs (from the public payer's perspective in Canadian dollars) and effectiveness were used to calculate incremental cost‐effectiveness ratios (ICERs) in life‐years gained (LYGs) and quality‐adjusted life years (QALYs), with bootstrapping to capture uncertainty. Varying the discount rate and reducing the price of ipilimumab were done as sensitivity analyses.ResultsIn total, 329 MM were identified (Treated: 189; Controls: 140). Ipilimumab was associated with an incremental effectiveness of 0.59 LYG, incremental cost of $91,233, and ICER of $153,778/LYG. ICERs were not sensitive to discounting rate. Adjusting for quality of life using utility weights resulted in an ICER of $225,885/QALY, confirming the original HTA estimate prior to public reimbursement. Reducing the price of ipilimumab by 100% resulted in an ICER of $111,728/QALY.ConclusionDespite its clinical benefit, ipilimumab as second‐line monotherapy for MM patients is not cost‐effective in the real world as projected by HTA under conventional willingness‐to‐pay thresholds.

PURPOSE: The role of frailty in affecting survival in myelodysplastic syndromes (MDS) is increasingly recognized. Despite this, a paucity of data exists on the association between frailty and other clinically meaningful outcomes including health care resource utilization and costs of care. METHODS: We linked the Ontario subset of the prospective Canadian MDS registry (including baseline patient/disease characteristics) to population-based health system administrative databases. Baseline frailty was calculated from the 15-item MDS-specific frailty scale (FS-15). Primary outcomes were public health care utilization and 30-day standardized costs of care (2019 Canadian dollars) determined for each phase of disease (initial, continuation, and terminal phases). Negative binomial regression was used to assess the association between frailty and health care costs with Poisson regression to explore predictors of hospitalization. RESULTS: Among 461 patients with complete FS-15 scores, 374 (81.1%) had a hospitalization with a mean length of stay of 10.6 days. Controlling for age, comorbidities, Revised International Prognostic Scoring System, and transfusion dependence, the FS-15 was independently associated with hospitalization during the initial (P = .02) and continuation (P = .01) phases but not the terminal disease phase (P = .09). The mean 30-day standardized cost per patient was $8,499 (median, $6,295; interquartile range, $2,798-$11,996), largely driven by cancer clinic visits and hospitalization. On multivariable analysis, the FS-15 was independently associated with costs of care during the initial disease phase (P = .02). CONCLUSION: We demonstrate an association between frailty and clinically meaningful outcomes including hospitalization and costs of care in patients with MDS. Our results suggest that baseline frailty may help to inform patients and physicians of expected outcomes.

Introduction: Systematic transrectal ultrasonography (TRUS) biopsy has been the standard diagnostic tool for prostate cancer (PCa) but is subject to limitations, such as a high false-negative rate of cancer detection. Multiparametric magnetic resonance imaging (mpMRI) prior to biopsy is emerging as an alternative diagnostic procedure for PCa. The PRECISE study found that MRI followed by a targeted biopsy was more accurately able to identify clinically significant cancer than TRUS biopsy. Methods: PRECISE study patients recruited in Ontario between January 2017 and November 2019 were linked to various Ontario provincial administrative databases available at the Institute for Clinical and Evaluative Sciences (ICES) to determine health resources used, associated costs, and hospitalizations in the 14 days after biopsy. Costs are presented in 2021 CAD. Results: A total of 281 males were included in this study, with 48.4% of the patients in the TRUS biopsy group, 28.1% in the MRI+, and 23.5% in the MRI- group. Twenty-one patients (15%) from the TRUS biopsy group were seen at a hospital in the 14 days after their biopsy compared to fewer than five patients (6%) from the MRI+ group. The mean per person per year (PPPY) costs for the TRUS and all MRI groups (MRI- and MRI+) were $7828 and $8525, respectively. Conclusions: Patients in the TRUS biopsy group experienced more hospital encounters compared to patients who received an MRI prior to their biopsy. This economic analysis suggests that MRI imaging prior to biopsy is not associated with a significant increase in costs.