Özdemir Dilay, Büssgen Melanie
University of Hamburg, Hamburg, Germany.
Hamburg Center for Health Economics, University of Hamburg, Hamburg, Germany.
J Pharm Policy Pract. 2023 Sep 25;16(1):106. doi: 10.1186/s40545-023-00611-7.
Until 2010, stage III or IV malignant melanoma (MM) had a poor prognosis. The discovery of immune checkpoint inhibitors (ICIs) in 2011 changed the treatment landscape. Promising results in patient survival with a checkpoint inhibitor prompted research into combination therapies. In 2016, the first combination therapy has been approved as first-line therapy for advanced MM.
The aim of this work is to investigate to what extent combination therapy is (cost-)effective compared to monotherapy in stage III or IV MM.
A systematic literature search was performed (Web of Science, PubMed, PubPharm, EconLit, and Cochrane Library); searching for publications published over the past decade that examine the cost-effectiveness in terms of cost/QALY and the effectiveness in terms of survival and response of combination therapy in comparison to monotherapy in stage III or IV MM patients.
A total of 11 randomized controlled trials (RCTs) and five cost-utility analyses met our inclusion criteria. Nine clinical trials demonstrated superiority of combination therapy over monotherapy. The combination of B-rapidly accelerated fibrosarcoma (BRAF) protein and mitogen-activated kinase (MEK) protein inhibitors is not cost-effective in any country. Three analyses demonstrate the cost-effectiveness of combination therapy with ICI compared to monotherapy.
Combination therapy is more effective compared to monotherapy. While combined ICIs are cost-effective compared to monotherapy, this is not the case for the combination of BRAF and MEK inhibitors.
在2010年之前,III期或IV期恶性黑色素瘤(MM)的预后较差。2011年免疫检查点抑制剂(ICI)的发现改变了治疗格局。检查点抑制剂在患者生存方面取得的良好结果促使人们对联合疗法展开研究。2016年,首个联合疗法被批准作为晚期MM的一线治疗方案。
本研究的目的是探讨在III期或IV期MM中,联合疗法与单药疗法相比在(成本)效益方面的程度。
进行了系统的文献检索(科学网、PubMed、PubPharm、EconLit和考科蓝图书馆);搜索过去十年发表的研究,这些研究从成本/质量调整生命年的角度考察联合疗法相对于单药疗法在III期或IV期MM患者中的成本效益,以及从生存和反应的角度考察其有效性。
共有11项随机对照试验(RCT)和5项成本效用分析符合我们的纳入标准。9项临床试验证明联合疗法优于单药疗法。BRAF(快速加速纤维肉瘤)蛋白和丝裂原活化蛋白激酶(MEK)蛋白抑制剂的联合在任何国家都不具有成本效益。三项分析表明,与单药疗法相比,ICI联合疗法具有成本效益。
与单药疗法相比,联合疗法更有效。虽然与单药疗法相比,联合ICI具有成本效益,但BRAF和MEK抑制剂的联合并非如此。