Wang Si-Tien, Anderson Ian M, Mitchell Dominic, Johnson Scott J, Shiozawa Aki
Medicus Economics, LLC, Boston, MA, USA.
Neuroscience and Psychiatry Unit, University of Manchester and Manchester Academic Health Science Centre, Manchester, UK.
Clinicoecon Outcomes Res. 2019 Mar 14;11:257-270. doi: 10.2147/CEOR.S181718. eCollection 2019.
Patients with treatment-resistant major depressive disorder (TRD) have limited treatment options. We developed an early stage cost-effectiveness model of TRD to explore the potential value of a hypothetical monotherapy relative to the standard of care (SOC). The relative impacts of the monotherapy's three differentiating features over SOC are explored: efficacy advantage, tolerability advantage, and price premium.
We adapted an existing economic model of TRD to evaluate the cost-effectiveness of a hypothetical monotherapy for TRD with a 25% efficacy advantage, a 10% tolerability advantage, and a 50% price premium over SOC (selective serotonin reuptake inhibitor plus atypical antipsychotics [SSRI + AAP]). The model is a hybrid of a decision tree that captures patients' outcomes after an 8-week acute treatment phase and a Markov model that simulates patients' depression course through a 10-month maintenance phase. Sensitivity (deterministic and probabilistic) and scenario analyses were conducted to characterize the relative impacts of the monotherapy's three differentiating features over SOC.
Over the 12-month time horizon, the hypothetical monotherapy is shown to dominate SOC; it generates lower costs and higher quality-adjusted life years in comparison to SSRI + AAP. Sensitivity and scenario analyses showed that this dominance depends largely on the monotherapy's efficacy and tolerability advantages over SOC. Specifically, a monotherapy with ≥ 12% efficacy or ≥70% tolerability advantage (and a 50% price premium) will always be superior to SSRI + AAP. Between these two extremes, most profiles, nonetheless, generate incremental cost-utility ratios for the monotherapy, which fall below common payer willingness-to-pay thresholds.
Our adaptation of an existing economic model of TRD provides a flexible platform for researchers to evaluate the efficacy/tolerability improvements required for a successful new TRD product and for decision-makers to assess the cost-effectiveness impact of uncertainties inherent in early stage product development in TRD.
难治性重度抑郁症（TRD）患者的治疗选择有限。我们开发了一种TRD的早期成本效益模型，以探讨一种假设的单一疗法相对于标准治疗（SOC）的潜在价值。探讨了该单一疗法相对于SOC的三个区别特征的相对影响：疗效优势、耐受性优势和价格溢价。
我们采用现有的TRD经济模型，评估一种假设的TRD单一疗法的成本效益，该疗法相对于SOC（选择性5-羟色胺再摄取抑制剂加非典型抗精神病药物[SSRI+AAP]）具有25%的疗效优势、10%的耐受性优势和50%的价格溢价。该模型是一个决策树和马尔可夫模型的混合体，决策树捕捉8周急性治疗阶段后患者的结局，马尔可夫模型模拟患者在10个月维持阶段的抑郁病程。进行了敏感性（确定性和概率性）和情景分析，以描述该单一疗法相对于SOC的三个区别特征的相对影响。
在12个月的时间范围内，该假设的单一疗法显示出优于SOC；与SSRI+AAP相比，它产生的成本更低，质量调整生命年更高。敏感性和情景分析表明，这种优势很大程度上取决于单一疗法相对于SOC的疗效和耐受性优势。具体而言，具有≥12%疗效或≥70%耐受性优势（以及50%价格溢价）的单一疗法总是优于SSRI+AAP。在这两个极端之间，尽管如此，大多数情况仍会产生单一疗法的增量成本效用比，该比值低于常见支付方的支付意愿阈值。
我们对现有TRD经济模型的改编为研究人员评估成功的新型TRD产品所需的疗效/耐受性改善提供了一个灵活的平台，并为决策者评估TRD早期产品开发中固有不确定性的成本效益影响提供了平台。
