Toth Peter P, Danese Mark, Villa Guillermo, Qian Yi, Beaubrun Anne, Lira Armando, Jansen Jeroen P
a CGH Medical Center , Sterling , IL , USA.
b Ciccarone Center for the Prevention of Heart Disease , Johns Hopkins University School of Medicine , Lutherville , MD , USA.
J Med Econ. 2017 Jun;20(6):555-564. doi: 10.1080/13696998.2017.1284078. Epub 2017 Jan 25.
To estimate real-world cardiovascular disease (CVD) burden and value-based price range of evolocumab for a US-context, high-risk, secondary-prevention population.
Burden of CVD was assessed using the UK-based Clinical Practice Research Datalink (CPRD) in order to capture complete CV burden including CV mortality. Patients on standard of care (SOC; high-intensity statins) in CPRD were selected based on eligibility criteria of FOURIER, a phase 3 CV outcomes trial of evolocumab, and categorized into four cohorts: high-risk prevalent atherosclerotic CVD (ASCVD) cohort (n = 1448), acute coronary syndrome (ACS) (n = 602), ischemic stroke (IS) (n = 151), and heart failure (HF) (n = 291) incident cohorts. The value-based price range for evolocumab was assessed using a previously published economic model. The model incorporated CPRD CV event rates and considered CV event reduction rate ratios per 1 mmol/L reduction in low-density lipoprotein-cholesterol (LDL-C) from a meta-analysis of statin trials by the Cholesterol Treatment Trialists Collaboration (CTTC), i.e. CTTC relationship.
Multiple-event rates of composite CV events (ACS, IS, or coronary revascularization) per 100 patient-years were 12.3 for the high-risk prevalent ASCVD cohort, and 25.7, 13.3, and 23.3, respectively, for incident ACS, IS, and HF cohorts. Approximately one-half (42%) of the high-risk ASCVD patients with a new CV event during follow-up had a subsequent CV event. Combining these real-world event rates and the CTTC relationship in the economic model, the value-based price range (credible interval) under a willingness-to-pay threshold of $150,000/quality-adjusted life-year gained for evolocumab was $11,990 ($9,341-$14,833) to $16,856 ($12,903-$20,678) in ASCVD patients with baseline LDL-C levels ≥70 mg/dL and ≥100 mg/dL, respectively.
Real-world CVD burden is substantial. Using the observed CVD burden in CPRD and the CTTC relationship, the cost-effectiveness analysis showed that, accounting for uncertainties, the expected value-based price for evolocumab is higher than its current annual cost, as long as the payer discount off list price is greater than 20%.
评估美国高危二级预防人群中依洛尤单抗的真实世界心血管疾病（CVD）负担及基于价值的价格范围。
使用英国临床实践研究数据链（CPRD）评估CVD负担，以获取包括心血管死亡率在内的完整心血管负担。根据依洛尤单抗3期心血管结局试验FOURIER的纳入标准，从CPRD中选择接受标准治疗（SOC；高强度他汀类药物）的患者，并分为四个队列：高危动脉粥样硬化性心血管疾病（ASCVD）患病率队列（n = 1448）、急性冠状动脉综合征（ACS）（n = 602）、缺血性卒中（IS）（n = 151）和心力衰竭（HF）（n = 291）发病队列。使用先前发表的经济模型评估依洛尤单抗基于价值的价格范围。该模型纳入了CPRD心血管事件发生率，并考虑了胆固醇治疗试验协作组（CTTC）他汀类试验荟萃分析中每降低1 mmol/L低密度脂蛋白胆固醇（LDL-C）的心血管事件降低率比值，即CTTC关系。
每100患者年的复合心血管事件（ACS、IS或冠状动脉血运重建）多事件发生率，高危ASCVD患病率队列中为12.3，ACS、IS和HF发病队列中分别为25.7、13.3和23.3。随访期间发生新心血管事件的高危ASCVD患者中，约一半（42%）随后发生了心血管事件。将这些真实世界事件发生率与经济模型中的CTTC关系相结合，对于基线LDL-C水平分别≥70 mg/dL和≥100 mg/dL的ASCVD患者，在支付意愿阈值为每获得一个质量调整生命年150,000美元的情况下，依洛尤单抗基于价值的价格范围（可信区间）为11,990美元（9,341 - 14,833美元）至16,856美元（12,903 - 20,678美元）。
真实世界的CVD负担很重。使用CPRD中观察到的CVD负担和CTTC关系，成本效益分析表明，考虑到不确定性，只要支付方相对于标价的折扣大于20%，依洛尤单抗基于价值的预期价格就高于其当前的年度成本。
