Jansen Jeroen P, Incerti Devin, Mutebi Alex, Peneva Desi, MacEwan Joanna P, Stolshek Bradley, Kaur Primal, Gharaibeh Mahdi, Strand Vibeke
a Precision Health Economics , Oakland , CA , USA.
b Amgen Inc. , Thousand Oaks , CA , USA.
J Med Econ. 2017 Jul;20(7):703-714. doi: 10.1080/13696998.2017.1307205. Epub 2017 Apr 5.
To determine the cost-effectiveness of treatment sequences of biologic disease-modifying anti-rheumatic drugs or Janus kinase/STAT pathway inhibitors (collectively referred to as bDMARDs) vs conventional DMARDs (cDMARDs) from the US societal perspective for treatment of patients with moderately to severely active rheumatoid arthritis (RA) with inadequate responses to cDMARDs.
An individual patient simulation model was developed that assesses the impact of treatments on disease based on clinical trial data and real-world evidence. Treatment strategies included sequences starting with etanercept, adalimumab, certolizumab, or abatacept. Each of these treatment strategies was compared with cDMARDs. Incremental cost, incremental quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) were calculated for each treatment sequence relative to cDMARDs. The cost-effectiveness of each strategy was determined using a US willingness-to-pay (WTP) threshold of $150,000/QALY.
For the base-case scenario, bDMARD treatment sequences were associated with greater treatment benefit (i.e. more QALYs), lower lost productivity costs, and greater treatment-related costs than cDMARDs. The expected ICERs for bDMARD sequences ranged from ∼$126,000 to $140,000 per QALY gained, which is below the US-specific WTP. Alternative scenarios examining the effects of homogeneous patients, dose increases, increased costs of hospitalization for severely physically impaired patients, and a lower baseline Health Assessment Questionnaire (HAQ) Disability Index score resulted in similar ICERs.
bDMARD treatment sequences are cost-effective from a US societal perspective.
从美国社会角度确定生物性病情缓解抗风湿药物或 Janus 激酶/信号转导与转录激活因子通路抑制剂（统称为生物 DMARDs）与传统 DMARDs（cDMARDs）治疗序列对中度至重度活动性类风湿关节炎（RA）且对 cDMARDs 反应不足患者的成本效益。
开发了一个个体患者模拟模型，该模型基于临床试验数据和真实世界证据评估治疗对疾病的影响。治疗策略包括以依那西普、阿达木单抗、赛妥珠单抗或阿巴西普起始的序列。将这些治疗策略中的每一种与 cDMARDs 进行比较。计算每个治疗序列相对于 cDMARDs 的增量成本、增量质量调整生命年（QALY）和增量成本效益比（ICER）。使用美国每 QALY 支付意愿（WTP）阈值 150,000 美元确定每种策略的成本效益。
对于基础病例情景，生物 DMARDs 治疗序列与更大的治疗效益（即更多 QALY）、更低的生产力损失成本以及比 cDMARDs 更高的治疗相关成本相关。生物 DMARDs 序列的预期 ICER 范围为每获得一个 QALY 约 126,000 美元至 140,000 美元，低于美国特定的 WTP。检查同质患者、剂量增加、严重身体受损患者住院成本增加以及较低基线健康评估问卷（HAQ）残疾指数评分影响的替代情景导致类似的 ICER。
从美国社会角度来看，生物 DMARDs 治疗序列具有成本效益。
