Abstract
ObjectiveTo test the hypothesis that treatment decisions (treatment with a PCSK9-mAb versus no treatment) are both more effective and more cost-effective when based on estimated lifetime benefit than when based on estimated risk reduction over 10 years.MethodsA microsimulation model was constructed for 10,000 patients with stable cardiovascular disease (CVD). Costs and quality-adjusted life years (QALYs) due to recurrent cardiovascular events and (non)vascular death were estimated for lifetime benefit-based compared to 10-year risk-based treatment, with PCSK9 inhibition as an illustration example. Lifetime benefit in months gained and 10-year absolute risk reduction were estimated using the SMART-REACH model, including an individualized treatment effect of PCSK9 inhibitors based on baseline low-density lipoprotein cholesterol. For the different numbers of patients treated (i.e. the 5%, 10%, and 20% of patients with the highest estimated benefit of both strategies), cost-effectiveness was assessed using the incremental cost-effectiveness ratio (ICER), indicating additional costs per QALY gain.ResultsLifetime benefit-based treatment of 5%, 10%, and 20% of patients with the highest estimated benefit resulted in an ICER of €36,440/QALY, €39,650/QALY, or €41,426/QALY. Ten-year risk-based treatment decisions of 5%, 10%, and 20% of patients with the highest estimated risk reduction resulted in an ICER of €48,187/QALY, €53,368/QALY, or €52,390/QALY.ConclusionTreatment decisions (treatment with a PCSK9-mAb versus no treatment) are both more effective and more cost-effective when based on estimated lifetime benefit than when based on estimated risk reduction over 10 years
Highlights
Recent guidelines for cardiovascular prevention all recommend estimating an individual patient’s risk (10-year risk of cardiovascular disease) for decision-making on whether or not to start preventive interventions [1,2,3,4]
A recently developed prediction model for secondary prevention, the Second Manifestation of ARTerial disease (SMART)-REACH model, is able to estimate individual benefit of medication for prevention of cardiovascular disease (CVD) in patients with a history of stable CVD as 10-year risk reduction or as months gained from a lifetime perspective, the lifetime benefit [7]
Patients selected for treatment based on the highest lifetime benefit are, on average, more than 10 years younger compared to patients selected based on the highest absolute 10-year CVD-risk reduction
Summary
Recent guidelines for cardiovascular prevention all recommend estimating an individual patient’s risk (10-year risk of cardiovascular disease) for decision-making on whether or not to start preventive interventions [1,2,3,4]. The use of lifetime prediction models that adjust for competing risks provides a more intuitive approach which identifies younger patients who would benefit from treatment they would otherwise be denied and older patients who might not benefit from treatment they would otherwise be offered [6]. As older patients are at risk for non-CVD mortality, any reductions in CVD-mortality risk may be counterbalanced by a high risk for non-CVD mortality. This may result in 10-year risk estimations leading to an overestimation of the potential benefit of preventive treatment in older patients [9]
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