Pochopien Michal, Dziedzic Jakub Wladyslaw, Aballea Samuel, Clay Emilie, Zerda Iwona, Toumi Mondher, Borissov Borislav
Assignity, Wadowicka 8a, 30-415 Krakow, Poland.
InovIntell, 3023GJ Rotterdam, Zuid-Holland, The Netherlands.
J Mark Access Health Policy. 2024 Apr 2;12(2):35-57. doi: 10.3390/jmahp12020005. eCollection 2024 Jun.
Currently there are no disease-specific approved therapies for non-alcoholic fatty liver (NAFL) and non-alcoholic steatohepatitis (NASH); however, several treatments are under development. This study aimed to estimate the cost-effectiveness of hypothetical innovative therapies compared with lifestyle intervention alone and combined with pioglitazone, and assess the health economic consequences of their future availability for patients.
A Markov cohort model was developed, considering fourteen disease health states and one absorbing state representing death. Transition probabilities, costs, utilities, and treatment efficacy were based on published data and assumptions. Four treatment strategies were considered, including two existing therapies (lifestyle intervention, small molecule treatment) and two hypothetical interventions (biological and curative therapy). The analysis was performed from the US third-party payer perspective.
The curative treatment with the assumed efficacy of 70% of patients cured and assumed price of $500,000 was the only cost-effective option. Although it incurred higher costs (a difference of $188,771 vs. lifestyle intervention and $197,702 vs. small molecule), it generated more QALYs (a difference of 1.58 and 1.38 QALYs, respectively), resulting in an ICER below the willingness-to-pay threshold of $150,000 per QALY. The sensitivity analyses showed that the results were robust to variations in model parameters.
This study highlighted the potential benefits of therapies aimed at curing a disease rather than stopping its progression. Nonetheless, each of the analyzed therapies could be cost-effective compared with lifestyle intervention at a relatively high price.
目前尚无针对非酒精性脂肪肝（NAFL）和非酒精性脂肪性肝炎（NASH）的疾病特异性获批疗法；然而，有几种治疗方法正在研发中。本研究旨在评估与单纯生活方式干预以及与吡格列酮联合使用相比，假设的创新疗法的成本效益，并评估其未来可用性对患者的健康经济影响。
开发了一个马尔可夫队列模型，考虑了14种疾病健康状态和1种代表死亡的吸收状态。转移概率、成本、效用和治疗效果基于已发表的数据和假设。考虑了四种治疗策略，包括两种现有疗法（生活方式干预、小分子治疗）和两种假设干预（生物疗法和治愈性疗法）。分析是从美国第三方支付方的角度进行的。
假设疗效为70%的患者治愈且假设价格为50万美元的治愈性治疗是唯一具有成本效益的选择。尽管它产生了更高的成本（与生活方式干预相比相差188,771美元，与小分子治疗相比相差197,702美元），但它产生了更多的质量调整生命年（分别相差1.58和1.38个质量调整生命年），导致增量成本效果比低于每质量调整生命年15万美元的支付意愿阈值。敏感性分析表明，结果对模型参数的变化具有稳健性。
本研究强调了旨在治愈疾病而非阻止其进展的疗法的潜在益处。尽管如此，与生活方式干预相比，每种分析的疗法在相对较高的价格下都可能具有成本效益。
