Dartmouth-Hitchcock Medical Center, Section of Allergy and Immunology, Lebanon, New Hampshire.
Dartmouth Geisel School of Medicine, Hanover, New Hampshire.
JAMA Netw Open. 2019 May 3;2(5):e193242. doi: 10.1001/jamanetworkopen.2019.3242.
Commercial epicutaneous peanut immunotherapy (EPIT) and peanut oral immunotherapy (POIT) may offer significant quality-of-life improvements for patients with peanut allergy, but the cost-effectiveness of commercial peanut immunotherapies is uncharacterized.
To evaluate critical inputs associated with the cost-effectiveness of EPIT and POIT from a societal perspective.
DESIGN, SETTING, AND PARTICIPANTS: Economic evaluation in which microsimulations with Markov modeling were performed evaluating virtual children aged 4 years over an 80-year time horizon. The base-case costs included a caregiver-reported willingness to pay of $3839 annually for safe and effective food allergy treatment. Estimates of predictive biomarkers or oral challenges were incorporated after the first year of therapy with additional analyses of immunotherapy risk reduction of anaphylaxis and probability of sustained unresponsiveness (SU) to peanut after 4 years.
Children received EPIT, POIT, or no immunotherapy treatment (n = 10 000 per treatment strategy).
Rates of therapy-associated adverse reactions and quality-of-life improvements associated with changes in eliciting or tolerated peanut doses were modeled along with quality-adjusted life-years (QALYs), anaphylaxis, therapy-associated anaphylaxis, and fatalities.
In the base-case analysis without SU to peanut, the EPIT strategy cost less than POIT (mean [SD] cost, $154 662 [$46 716] vs $163 524 [$56 800]) and had fewer total episodes of anaphylaxis (mean [SD], 1.33 [1.55] vs 3.83 [5.02] episodes) and fewer episodes of therapy-associated anaphylaxis (mean [SD], 0.62 [1.30] vs 3.10 [4.94] episodes) but had lower QALY accumulation (mean [SD], 26.932 [2.241] vs 26.945 [2.320] QALYs). The incremental cost-effectiveness ratio was $216 061 for EPIT and $255 431 for POIT. Models were sensitive to therapy cost, SU rates, health state utility, and risk reduction of anaphylaxis. With health state utility sensitivity analyses, the ceiling value-based cost (willingness-to-pay threshold $100 000/QALY) was between $1568 and $6568 for EPIT and between $1235 and $5235 for POIT. If high rates of SU to peanut can be achieved in longer-term models, EPIT and POIT could produce savings in terms of both cost and QALY.
In this simulated analysis, findings showed that EPIT and POIT may be cost-effective under some assumptions. Further research is needed to understand the degree of health state utility improvement associated with each therapy, degree of protection against anaphylaxis, and rates of SU.
商业性经皮食入性花生免疫疗法（EPIT）和花生口服免疫疗法（POIT）可能会为花生过敏患者带来显著的生活质量改善，但商业性花生免疫疗法的成本效益尚不清楚。
从社会角度评估 EPIT 和 POIT 的成本效益的关键投入。
设计、地点和参与者：经济评估中，采用马尔可夫模型进行了微模拟，评估了 4 岁的虚拟儿童在 80 年的时间内的情况。基础病例成本包括照顾者每年报告的愿意为安全有效的食物过敏治疗支付 3839 美元。在治疗的第一年之后，对预测生物标志物或口服挑战进行了评估，并进行了 4 年后过敏反应发生率和持续无反应（SU）的概率的免疫治疗风险降低的额外分析。
儿童接受 EPIT、POIT 或无免疫治疗（每种治疗策略 10000 名儿童）。
与花生剂量的诱导或耐受相关的治疗相关不良反应和生活质量改善的发生率，以及调整后的生命年（QALY）、过敏反应、治疗相关过敏反应和死亡率进行建模。
在没有 SU 到花生的基础病例分析中，EPIT 策略的成本低于 POIT（平均[标准差]成本，154662 美元[46716 美元] vs 163524 美元[56800 美元]），且过敏反应总发作次数更少（平均[标准差]，1.33[1.55] vs 3.83[5.02]次），治疗相关过敏反应次数更少（平均[标准差]，0.62[1.30] vs 3.10[4.94]次），但 QALY 积累较低（平均[标准差]，26.932[2.241] vs 26.945[2.320]QALY）。EPIT 的增量成本效益比为 216061 美元，POIT 为 255431 美元。模型对治疗成本、SU 率、健康状态效用和过敏反应发生率敏感。在健康状态效用敏感性分析中，基于上限价值的成本（愿意支付的阈值为 100000 美元/QALY）在 EPIT 为 1568 美元至 6568 美元之间，在 POIT 为 1235 美元至 5235 美元之间。如果在长期模型中可以实现高 SU 到花生的比率，EPIT 和 POIT 可能会在成本和 QALY 方面产生节约。
在这项模拟分析中，结果表明，在某些假设下，EPIT 和 POIT 可能具有成本效益。需要进一步研究以了解每种治疗方法与健康状态改善相关的程度、对过敏反应的保护程度以及 SU 的发生率。
