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IntroductionUtility instruments are used to assess patients' health-related quality of life for cost-utility analysis (CUA). However, for cancer patients, the dimensions of generic utility instruments may not capture all the information relevant to the impact of cancer. Cancer-specific utilities provide a useful alternative. Under the auspices of the Multi-Attribute Utility in Cancer Consortium, a cancer-specific utility algorithm was derived from the FACT-G. The new FACT-8D contains eight dimensions: pain, fatigue, nausea, sleep, work, support from family/friends, sadness, and worry health will get worse. The aim of the study was to obtain a Canadian value set for the FACT-8D.MethodsA discrete choice experiment was administered to a Canadian general population online panel, quota sampled by age, sex, and province/territory of residence. Respondents provided responses to 16 choice sets. Each choice set consisted of two health states described by the FACT-8D dimensions plus an attribute representing survival duration. Sample weights were applied and the responses were analyzed using conditional logistic regression, parameterized to fit the quality-adjusted life year framework. The results were converted into utility weights by evaluating the marginal rate of substitution between each level of each FACT-8D dimension with respect to duration.Results2228 individuals were recruited. The analysis dataset included n = 1582 individuals, who completed at least one choice set; of which, n = 1501 completed all choice sets. After constraining to ensure monotonicity in the utility function, the largest decrements were for the highest levels of pain (- 0.38), nausea (- 0.30), and problems doing work (- 0.23). The decrements of the remaining dimensions ranged from - 0.08 to - 0.18 for their highest levels. The utility of the worst possible health state was defined as - 0.65, considerably worse than dead.ConclusionsThe largest impacts on utility included three generic dimensions (i.e., pain, support, and work) and nausea, a symptom caused by cancer (e.g., brain tumours, gastrointestinal tumours, malignant bowel obstruction) and by common treatments (e.g., chemotherapy, radiotherapy, opioid analgesics). This may make the FACT-8D more informative for CUA evaluating in many cancer contexts, an assertion that must now be tested empirically in head-to-head comparisons with generic utility measures.

BackgroundScientific advances have led to the discovery of novel treatments with high prices. The cost to publicly fund high-cost drugs may threaten the sustainability of drug budgets in different health care systems. In oncology, there are concerns that health-benefit gains are diminishing over time and that the economic evidence to support funding decisions is too limited.MethodsTo assess the additional costs and benefits gained from oncology drugs over time, we used treatment protocols and efficacy results from U.S. Food and Drug Administration records to calculate cost-effectiveness ratios for drugs approved to treat first- and second-line metastatic or advanced breast, colorectal, and non-small cell lung cancer during the years 1994-2013. We assessed reimbursement recommendations reached by health technology assessment agencies in the U.K., Australia, and Canada.ResultsCost-effectiveness ratios were calculated for 50 drugs approved by the U.S. regulator. The more recent approvals were often based on surrogate efficacy outcomes and had extremely high costs, often triple the costs of drugs approved in previous years. Over time, the effectiveness gains have increased for some cancer indications; however, for other indications (non-small cell lung and second-line colorectal cancer), the magnitude of gains in effectiveness decreased. Reimbursement recommendations for drugs with the highest cost-effectiveness ratios were the most inconsistent.ConclusionEvaluation of the clinical benefits that oncology drugs offer as a function of their cost has become highly complex, and for some clinical indications, health benefits are diminishing over time. There is an urgent need for better economic evidence from oncology drug trials and systematic processes to inform funding decisions.Implications for practiceHigh-cost oncology drugs may threaten the ability of health care systems to provide access to promising new drugs for patients. In order to make better drug-funding decisions and enable equitable access to breakthrough treatments, discussions in the oncology community should include economic evidence. This study summarizes the extra benefits and costs of newly approved drugs from pivotal trials during the postgenomic era of drug discovery. The reader will gain an appreciation of the need for economic evidence to make better drug-reimbursement decisions and the dynamics at play in today's oncology drug market.

BackgroundAn important challenge with the application of next-generation sequencing technology is the possibility of uncovering incidental genomic findings. A paucity of evidence on personal utility for incidental findings has hindered clinical guidelines. Our objective was to estimate personal utility for complex information derived from incidental genomic findings.MethodsWe used a discrete-choice experiment to evaluate participants' personal utility for the following attributes: disease penetrance, disease treatability, disease severity, carrier status and cost. Study participants were drawn from the Canadian public. We analyzed the data with a mixed logit model.ResultsIn total, 1200 participants completed our questionnaire (available in English and French). Participants valued receiving information about high-penetrance disorders but expressed disutility for receiving information on low-penetrance disorders. The average willingness to pay was $445 (95% confidence interval [CI] $322-$567) to receive incidental findings in a scenario where clinicians returned information about high-penetrance, medically treatable disorders, but only 66% of participants (95% CI 63%-71%) indicated that they would choose to receive information in that scenario. On average, participants placed an important value ($725, 95% CI $600-$850) on having a choice about what type of findings they would receive, including receipt of information about high-penetrance, treatable disorders or receipt of information about high-penetrance disorders with or without available treatment. The predicted uptake of that scenario was 76% (95% CI 72%-79%).InterpretationMost participants valued receiving incidental findings, but personal utility depended on the type of finding, and not all participants wanted to receive incidental results, regardless of the potential health implications. These results indicate that to maximize benefit, participant-level preferences should inform the decision about whether to return incidental findings.

Programme budgeting and marginal analysis (PBMA) is becoming an increasingly popular tool in setting health service priorities. This paper presents a novel multi-attribute utility (MAU) approach to setting health service priorities using PBMA. This approach includes identifying the attributes of the MAU function; describing and scaling attributes; quantifying trade-offs between attributes; and combining single conditional utility functions into the MAU function. We illustrate the MAU approach using a PBMA case study in mental health services from the Community Health Sector in metropolitan South Australia. Keywords: Priority setting; Programme budgeting; Marginal analysis; Decision analysis; Multi-attribute utility theory; Mental health; Australia

Background: The trial assigning individualized options for treatment (Rx) (TAILORx) confirmed the predictive value of the 21-gene recurrence score (RS) assay in hormone receptor (HR)-positive, HER2-negative, node-negative breast cancer and established thresholds for chemotherapy benefit in younger and older patients. Real-world chemotherapy use and RS-guided treatment costs in British Columbia post-TAILORx were examined.Methods: The authors assembled 3 cohorts of HR-positive, HER2-negative, node-negative patients with breast cancer defined by diagnosis: before RS funding (cohort 1 [C1]: January 2013-December 2013), after introduction of public RS funding (cohort 2 [C2]: July 2015-June 2016), and after TAILORx results (cohort 3 [C3]: July 2018-June 2019). Chemotherapy use was compared between cohorts by age and RS. Budgetary impacts of RS testing on chemotherapy costs were evaluated pre- and post-TAILORx.Results: Among the 2066 patients included, chemotherapy use declined by 19% after RS funding was introduced and by an additional 23% after TAILORx publication (P = .001). Reduction in chemotherapy use was significant for RS 11-20 tumors (C3 vs C2, P = .004). There was no significant change in chemotherapy use in patients >50 years old (C2:12% vs C3:10%, P = .22). RS testing was associated with higher cost savings post-TAILORx, except in patients 70 to 80 years old, where testing led to excess costs when adjusting for the low rate of RS-concordant chemotherapy prescribed.Conclusions: TAILORx has had population-based impacts on chemotherapy prescribing in intermediate RS tumors and patients ≤50 years old. The lower clinical use of RS and increased spending in patients 70-80 years old highlights the importance of careful selection of older candidates for high-cost genomic testing.Lay Summary: The 21-gene recurrence score (RS) test helps predict whether patients with hormone-positive, HER2-negative, lymph node-negative breast cancer are likely to benefit from chemotherapy. The recent trial assigning individualized options for treatment (Rx) (TAILORx) found that patients with intermediate RS tumors did not benefit from chemotherapy. The authors assessed whether TAILORx results translated to real-world changes in chemotherapy prescribing patterns. In this study, chemotherapy use decreased by 23% after TAILORx, with the greatest reductions seen among intermediate RS tumors and younger patients. In contrast, RS testing had lower clinical value and increased treatment costs in elderly patients, which requires further study to ensure optimal care for this age group. [ABSTRACT FROM AUTHOR]

PURPOSE: This study aimed to investigate the impact of early versus not-early palliative care among cancer decedents on end-of-life health care costs. METHODS: Using linked administrative databases, we created a retrospective cohort of cancer decedents between 2004 and 2014 in Ontario, Canada. We identified those who received early palliative care (palliative care service used in the hospital or community 12 to 6 months before death [exposure]). We used propensity score matching to identify a control group of not-early palliative care, hard matched on age, sex, cancer type, and stage at diagnosis. We examined differences in average health system costs (including hospital, emergency department, physician, and home care costs) between groups in the last month of life. RESULTS: We identified 144,306 cancer decedents, of which 37% received early palliative care. After matching, we created 36,238 pairs of decedents who received early and not-early (control) palliative care; there were balanced distributions of age, sex, cancer type (24% lung cancer), and stage (25% stage III and IV). Overall, 56.3% of early group versus 66.7% of control group used inpatient care in the last month (P < .001). Considering inpatient hospital costs in the last month of life, the early group used an average (±standard deviation) of $7,105 (±$10,710) versus the control group of $9,370 (±$13,685; P < .001). Overall average costs (±standard deviation) in the last month of life for patients in the early versus control group was $12,753 (±$10,868) versus $14,147 (±$14,288; P < .001). CONCLUSION: Receiving early palliative care reduced average health system costs in the last month of life, especially via avoided hospitalizations [ABSTRACT FROM AUTHOR]

Abstract Background Multi attribute utility (MAU) instruments are used to include the health related quality of life (HRQoL) in economic evaluations of health programs. Comparative studies suggest different MAU instruments measure related but different constructs. The objective of this paper is to describe the methods employed to achieve content validity in the descriptive system of the Assessment of Quality of Life (AQoL)-6D, MAU instrument. Methods The AQoL program introduced the use of psychometric methods in the construction of health related MAU instruments. To develop the AQoL-6D we selected 112 items from previous research, focus groups and expert judgment and administered them to 316 members of the public and 302 hospital patients. The search for content validity across a broad spectrum of health states required both formative and reflective modelling. We employed Exploratory Factor Analysis and Structural Equation Modelling (SEM) to meet these dual requirements. Results and Discussion The resulting instrument employs 20 items in a multi-tier descriptive system. Latent dimension variables achieve sensitive descriptions of 6 dimensions which, in turn, combine to form a single latent QoL variable. Diagnostic statistics from the SEM analysis are exceptionally good and confirm the hypothesised structure of the model. Conclusions The AQoL-6D descriptive system has good psychometric properties. They imply that the instrument has achieved construct validity and provides a sensitive description of HRQoL. This means that it may be used with confidence for measuring health related quality of life and that it is a suitable basis for modelling utilities for inclusion in the economic evaluation of health programs.

Abstract Background In the HPV FOCAL trial, we will establish the efficacy of hr-HPV DNA testing as a stand-alone screening test followed by liquid based cytology (LBC) triage of hr-HPV-positive women compared to LBC followed by hr-HPV triage with ≥ CIN3 as the outcome. Methods/Design HPV-FOCAL is a randomized, controlled, three-armed study over a four year period conducted in British Columbia. It will recruit 33,000 women aged 25-65 through the province's population based cervical cancer screening program. Control arm: LBC at entry and two years, and combined LBC and hr-HPV at four years among those with initial negative results and hr-HPV triage of ASCUS cases; Two Year Safety Check arm: hr-HPV at entry and LBC at two years in those with initial negative results with LBC triage of hr-HPV positives; Four Year Intervention Arm: hr-HPV at entry and combined hr-HPV and LBC at four years among those with initial negative results with LBC triage of hr-HPV positive cases Discussion To date, 6150 participants have a completed sample and epidemiologic questionnaire. Of the 2019 women enrolled in the control arm, 1908 (94.5%) were cytology negative. Women aged 25-29 had the highest rates of HSIL (1.4%). In the safety arm 92.2% of women were hr-HPV negative, with the highest rate of hr-HPV positivity found in 25-29 year old women (23.5%). Similar results were obtained in the intervention arm HPV FOCAL is the first randomized trial in North America to examine hr-HPV testing as the primary screen for cervical cancer within a population-based cervical cancer screening program. Trial Registration International Standard Randomised Controlled Trial Number Register, ISRCTN79347302

Purpose: Understanding the end-of-life psychosocial needs of cancer patients at home is a knowledge gap. This study describes the trajectory of psychosocial symptoms in the last 6 months of life among cancer decedents who were receiving home care.Methods: Observational population-based cohort study of cancer decedents who were receiving home care services between 2007 and 2014. Decedents had to have at least one home care assessment in the last 6 months of life for inclusion. Outcomes were the presence of psychosocial symptoms (i.e., anxiety, loneliness, depression, social decline, caregiver distress, and cognitive decline) at each week before death.Results: Our cohort included 27,295 unique cancer decedents (30,368 assessments), of which 58% died in hospital. Fifty-six percent were older than 74, and 47% were female. The prevalence of all symptoms increased approaching death, except loneliness. Social decline (48%-78%) was the most prevalent psychosocial symptom, though loneliness was reported in less than 10% of the cohort. Caregiver distress rose over time from 15%-27%. A third of the cohort reported issues with cognitive impairment. Multivariate regression showed that physical symptoms such as uncontrolled pain, impairment in independent activities of daily living, and a high level of health instability all significantly worsened the odds of having a psychosocial symptom in the last 3 months of life.Conclusion: In this large home care cancer cohort, trajectories of psychosocial symptoms worsened close to death. Physical symptoms, such as uncontrolled pain, were associated with having worse psychosocial symptoms at end of life. [ABSTRACT FROM AUTHOR]

Background: In Canada, the Canadian Agency for Drugs and Technologies in Health (CADTH) evaluates and makes recommendations for the reimbursement of cancer drugs. One component of its recommendation is based on an economic evaluation, which typically takes the form of a cost-utility analysis. A cost-utility analysis measures the effects of competing therapies with quality-adjusted life-years (QALYs). The data for this calculation typically come from generic, preference-based measures of health-related quality of life (HRQOL). The objective of this review is to determine the frequency at which HRQOL data are collected alongside cancer drug trials and used in the cost-utility analysis submitted to the CADTH pan-Canadian Oncology Drug Review (pCODR).Methods: Submissions between 2015 and 2018 to pCODR, the group charged with evaluating cancer drug submissions at CADTH, were reviewed. All pCODR submissions, either in progress or completed, were publicly available online. The search was restricted to completed evaluations.Results: Forty-three submissions met the inclusion criteria. The incremental gain in QALYs in most submissions from the new technology was small (median incremental gain, 0.86; interquartile range, 0.6-1.39). More than half of the submissions (56%) did not include original data on HRQOL, with most relying on previous studies of variable relevance and quality. Re-analyses by pCODR based on concerns over HRQOL data used in the submitted model were common (52%).Conclusions: Drug manufacturers do not consistently collect data on HRQOL alongside clinical trials and instead rely on evidence generated in previous studies to inform cost-utility analyses. These findings should induce manufacturers to collect original HRQOL data that are simultaneously relevant to patients and decision makers. [ABSTRACT FROM AUTHOR]