Abstract
In 2016, everolimus was approved by Health Canada for the treatment of unresectable, locally advanced or metastatic, well-differentiated, non-functional, neuroendocrine tumours (NET) of gastrointestinal (GI) or lung origin in adult patients with progressive disease. This analysis evaluated the cost-effectiveness of everolimus in this setting from a Canadian societal perspective. A partitioned survival model was developed to compare the cost per life-year (LY) gained and cost per quality-adjusted life-year (QALY) gained of everolimus plus best supportive care (BSC) versus BSC alone in patients with advanced or metastatic NET of GI or lung origin. Model health states included stable disease, disease progression, and death. Efficacy inputs were based on the RADIANT-4 trial and utilities were mapped from quality-of-life data retrieved from RADIANT-4. Resource utilization inputs were derived from a Canadian physician survey, while cost inputs were obtained from official reimbursement lists from Ontario and other published sources. Costs and efficacy outcomes were discounted 5% annually over a 10-year time horizon, and sensitivity analyses were conducted to test the robustness of the base case results. Everolimus had an incremental gain of 0.616 QALYs (0.823 LYs) and CA$89,795 resulting in an incremental cost-effectiveness ratio of CA$145,670 per QALY gained (CA$109,166 per LY gained). The probability of cost-effectiveness was 52.1% at a willingness to pay (WTP) threshold of CA$150,000 per QALY. Results of the probabilistic sensitivity analysis indicate that everolimus has a 52.1% probability of being cost-effective at a WTP threshold of CA$150,000 per QALY gained in Canada.
Highlights
Neuroendocrine tumours (NETs) are a relatively rare group of related but diverse malignancies originating from neuroendocrine cells in a variety of anatomical locations throughout the body[1]
Everolimus had an incremental gain of 0.616 quality-adjusted life-year (QALY) (0.823 LYs) and Canadian dollars (CA$)89,795 resulting in an incremental cost-effectiveness ratio of CA$145,670 per QALY gained (CA$109,166 per LY gained)
Results of the probabilistic sensitivity analysis indicate that everolimus has a 52.1% probability of being cost-effective at a willingness to pay (WTP) threshold of CA$150,000 per QALY gained in Canada
Summary
Neuroendocrine tumours (NETs) are a relatively rare group of related but diverse malignancies originating from neuroendocrine cells in a variety of anatomical locations throughout the body (e.g., endocrine glands, endocrine islets within glandular tissue, and cells dispersed between exocrine cells)[1]. Advanced NET is a rare, progressive, and fatal malignancy. Based on the National Comprehensive Cancer Network Outcomes Database, it is estimated that 72% of GI/lung NETs are non-functional[2]. Non-functioning NETs are asymptomatic and often go undiagnosed until the disease has advanced[1]. In 2016, everolimus was approved by Health Canada for the treatment of unresectable, locally advanced or metastatic, well-differentiated, non-functional, neuroendocrine tumours (NET) of gastrointestinal (GI) or lung origin in adult patients with progressive disease. This analysis evaluated the cost-effectiveness of everolimus in this setting from a Canadian societal perspective
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