Kamstra Rhiannon, Durkin Mike, Cai Jennifer, Bookhart Brahim, Lafeuille Marie-Hélène, Pilon Dominic, Tiggelaar Sean, Manceur Ameur M, Lefebvre Patrick
a Analysis Group Inc. , Montréal , Québec , Canada.
b Janssen Scientific Affairs, LLC , Titusville , NJ , USA.
J Med Econ. 2019 Mar;22(3):280-287. doi: 10.1080/13696998.2018.1562817. Epub 2019 Jan 11.
To model direct medical costs associated with reductions in cardiovascular disease (CVD) events in T2DM patients reported in the CANVAS and EMPA-REG trials, which assessed the cardiovascular safety of canagliflozin and empagliflozin, respectively.
Costs were modeled from a US managed care organization (MCO) perspective for the CVD outcomes included in both trials: three-point major adverse cardiovascular event (MACE) and its components (cardiovascular-related death, nonfatal myocardial infarction, nonfatal stroke), as well as heart failure requiring hospitalization. The rate of CVD events averted (difference between study drug and placebo) was projected to the portion of an MCO T2DM population matching the respective trial's inclusion criteria. A targeted literature search for paid amounts directly associated with each CVD event provided the unit costs, which were applied to the projected number of events averted, to calculate costs avoided per member per year (PMPY). One-way sensitivity analyses were performed on events averted, unit costs, and percentages of trial-applicable patients.
Based on three-point MACE events averted, costs avoided PMPY of $6.17 (range: $1.27-$10.94) for CANVAS and $2.75 ($0.19-$4.83) for EMPA-REG were estimated. Costs avoided for individual components of MACE ranged from $0.77 to $3.84 PMPY for CANVAS and from -$0.97 (additional costs) to $1.54 for EMPA-REG. PMPY costs avoided for heart failure were $2.72 for CANVAS and $1.32 for EMPA-REG.
Models assumed independent, non-recurrent outcomes and were restricted to medical costs directly associated with the trial-reported events. The reductions in CVD events in T2DM patients reported for both CANVAS and EMPA-REG project to a positive cost avoidance for these events in an MCO population. The analysis did not include an assessment of the impact on total cost, as the costs associated with adverse events, drug utilization or other clinical outcomes were not examined.
对CANVAS和EMPA-REG试验中报告的2型糖尿病（T2DM）患者心血管疾病（CVD）事件减少所带来的直接医疗成本进行建模，这两项试验分别评估了卡格列净和恩格列净的心血管安全性。
从美国管理式医疗组织（MCO）的角度对两项试验中包含的CVD结局进行成本建模：三点主要不良心血管事件（MACE）及其组成部分（心血管相关死亡、非致命性心肌梗死、非致命性中风），以及需要住院治疗的心力衰竭。将避免的CVD事件发生率（研究药物与安慰剂之间的差异）推算至符合各自试验纳入标准的MCO T2DM人群部分。针对与每个CVD事件直接相关的支付金额进行有针对性的文献检索，以提供单位成本，并将其应用于推算出的避免事件数量，以计算每年每位成员避免的成本（PMPY）。对避免的事件、单位成本和适用试验患者的百分比进行单向敏感性分析。
基于避免的三点MACE事件，估计CANVAS的PMPY避免成本为6.17美元（范围：1.27 - 10.94美元），EMPA-REG为2.75美元（0.19 - 4.83美元）。CANVAS中MACE各个组成部分的PMPY避免成本范围为0.77至3.84美元，EMPA-REG为 - 0.97美元（额外成本）至1.54美元。CANVAS中因心力衰竭避免的PMPY成本为2.72美元，EMPA-REG为1.32美元。
模型假设结局相互独立且不复发，并且仅限于与试验报告事件直接相关的医疗成本。CANVAS和EMPA-REG报告的T2DM患者CVD事件减少预计会使MCO人群中这些事件的成本得到正向避免。该分析未包括对总成本影响的评估，因为未检查与不良事件、药物使用或其他临床结局相关的成本。
