Cost-Effectiveness Analysis of Evolocumab in Adult Patients with Atherosclerotic Cardiovascular Disease in Canada.

INTRODUCTION: To evaluate the cost-effectiveness of evolocumab when added to standard of care lipid-lowering treatment (LLT) for patients with atherosclerotic cardiovascular disease (ASCVD) who cannot adequately control their low-density lipoprotein cholesterol (LDL-C) despite optimized LLT in Canada. METHODS: An incremental cost-utility analysis was conducted using a Markov cohort state transition model adapted to the Canadian setting. Analyses were conducted from a public health and societal perspective using a lifetime time horizon for Canada. Scenario analyses were conducted on the basis of recommendations from the 2021 Canadian Cardiovascular Society (CCS) dyslipidemia guidelines. RESULTS: In ASCVD patients with prior myocardial infarction (MI) and baseline LDL-C ≥ 1.8 mmol/L, adding evolocumab to optimized statin therapy with or without ezetimibe is associated with an incremental cost per quality-adjusted life year (QALY) gained of $66,453 CAD. Furthermore, for every 100 patients treated with evolocumab for lifetime, adding evolocumab to optimized LLT will prevent approximately 52 cardiovascular (CV) events, of which seven would be fatal. The results are generally robust using univariate and simultaneous variation in model input parameters. Scenario analyses for patient populations as per the CCS guidelines suggest that evolocumab added to optimized LLT may be considered cost-effective, given an incremental cost-effectiveness ratio (ICER) threshold of CAD$100,000 per QALY gained. Limitations associated with this analysis should be interpreted in the context of data and modeling assumptions used. CONCLUSION: Overall, this analysis supports reimbursement of evolocumab by payers in patients with ASCVD who cannot reach LDL-C thresholds despite optimized LLT to reduce unnecessary fatal and non-fatal CV events.

Cost-effectiveness of romosozumab for the treatment of postmenopausal women at very high risk of fracture in Canada.

This study evaluated the cost-effectiveness of 1 year of romosozumab followed by alendronate versus oral bisphosphonates alone in women with postmenopausal osteoporosis at very high risk for fracture in Canada. Results showed that romosozumab sequenced to alendronate is a cost-effective treatment option, dominating both alendronate and risedronate alone. PURPOSE: To demonstrate the value of romosozumab sequenced to alendronate compared to alendronate or risedronate alone, for the treatment of osteoporosis in postmenopausal women with a history of osteoporotic fracture and who are at very high risk for future fracture in Canada. METHODS: A Markov model followed a hypothetical cohort of postmenopausal osteoporotic women at very high risk for future fractures, to estimate the cost-effectiveness of romosozumab and alendronate compared to oral bisphosphonates alone. A total treatment period of 5 years was assumed. Quality-adjusted life years and costs were estimated for each comparator across health states defined by different types of fragility fractures. RESULTS: Romosozumab/alendronate was associated with a lifetime gain of 0.103 and 0.127 QALYs and a cost reduction of $343 and $3805, relative to alendronate and risedronate, respectively. These results were driven by a reduction of the number of fractures (2561 per 1000 patients, versus 2700 for alendronate and 2724 for risedronate over lifetime). Romosozumab/alendronate had the highest probability of being cost-effective, relative to alendronate and risedronate, at any willingness to pay threshold value. CONCLUSION: Romosozumab/alendronate was associated with reduced costs and greater benefit relative to other comparators. Probabilistic, deterministic, and scenario analyses indicate that romosozumab/alendronate represents the best value for money; the uncertainty analyses are robust, and therefore romosozumab should be considered for reimbursement by public drug plans in Canada .

An international study of the quality of life of adult patients treated with home parenteral nutrition.

BACKGROUND & AIMS: Home parenteral nutrition-quality of life (HPN-QOL©) is a self-assessment tool for the measurement of QOL in patients on HPN. The aims of this study were: to re-assess the basic psychometric properties of the HPN-QOL© in a multinational sample of adult patients; to provide a description of QOL dimensions by short and long HPN treatment duration; to explore clinical factors potentially associated to QOL scores. METHODS: Patients (n = 699) from 14 countries completed the HPN-QOL©. The questionnaires were analysed to evaluate data completeness, convergent/discriminant validity and internal-consistency reliability. The association of overall QOL and HPN treatment duration as well as other clinical factors were investigated using multivariable linear regression models. RESULTS: The analysis of the multitrait-scaling and internal consistency indicates a good fit with the questionnaire structure for most items. Item discriminant validity correlation was satisfactory and psychometric evaluation of the HPN-QOL© in the different English, French and Italian language patient sub-groups confirmed psychometric equivalence of the three questionnaire versions. The results of the multivariable linear regression showed that QOL scores were significantly associated with HPN duration (better in long-term), underlying disease (better in Crohn's disease and mesenteric ischaemia) and living status (worse in living alone) and, after adjusting for the other factors, with the number of days of HPN infusion per week. CONCLUSIONS: The HPN-QOL©, is a valid tool for measurement of QOL in patients on HPN, to be used in the clinical practice as well as in research.