Section of Gastroenterology and Hepatology, Dartmouth-Hitchcock Health, Lebanon, New Hampshire, USA.
Division of Gastroenterology, Massachusetts General Hospital, Boston, Massachusetts, USA.
Am J Gastroenterol. 2021 Oct 1;116(10):2118-2127. doi: 10.14309/ajg.0000000000001403.
Chronic idiopathic constipation (CIC) is a common and burdensome illness. We performed a cost-effectiveness analysis of the US Food and Drug Administration-approved CIC drugs to evaluate and quantify treatment preferences compared with usual care from insurer and patient perspectives.
We evaluated the subset of patients with CIC and documented failure of over-the-counter (OTC) osmotic or bulk-forming laxatives. A RAND/UCLA consensus panel of 8 neurogastroenterologists informed model design. Treatment outcomes and costs were defined using integrated analyses of registered clinical trials and the US Centers for Medicare and Medicaid Services-supported cost databases. Quality-adjusted life years (QALYs) were calculated using health utilities derived from clinical trials. A 12-week time horizon was used.
With continued OTC laxatives, CIC-related costs were $569 from an insurer perspective compared with $3,154 from a patient perspective (considering lost wages and out-of-pocket expenses). CIC prescription drugs increased insurer costs by $618-$1,015 but decreased patient costs by $327-$1,117. Effectiveness of CIC drugs was similar (0.02 QALY gained/12 weeks or ∼7 healthy days gained/year). From an insurer perspective, prescription drugs (linaclotide, prucalopride, and plecanatide) seemed less cost-effective than continued OTC laxatives (incremental cost-effectiveness ratio >$150,000/QALY gained). From a patient perspective, the cost-effective algorithm started with plecanatide, followed by choosing between prucalopride and linaclotide starting at the 145-μg dose (favoring prucalopride among patients whose disease affects their work productivity). The patient perspective was driven by drug tolerability and treatment effects on quality of life.
Addressing costs at a policy level has the potential to enable patients and clinicians to move from navigating barriers in treatment access toward truly optimizing treatment choice.
慢性特发性便秘(CIC)是一种常见且负担沉重的疾病。我们对美国食品和药物管理局批准的 CIC 药物进行了成本效益分析,从保险公司和患者的角度评估和量化了与常规护理相比的治疗偏好。
我们评估了 CIC 患者亚组,这些患者有记录的非处方(OTC)渗透性或容积性泻药治疗失败。由 8 名神经胃肠病学家组成的 RAND/UCLA 共识小组为模型设计提供了信息。使用注册临床试验和美国医疗保险和医疗补助服务支持的成本数据库的综合分析来定义治疗结果和成本。使用来自临床试验的健康效用来计算质量调整生命年(QALY)。使用 12 周的时间范围。
继续使用 OTC 泻药,从保险公司的角度来看,CIC 相关的成本为 569 美元,而从患者的角度来看,成本为 3154 美元(考虑到工资损失和自付费用)。CIC 处方药增加了保险公司的成本 618-1015 美元,但降低了患者的成本 327-1117 美元。CIC 药物的疗效相似(12 周内增加 0.02 QALY 或每年增加约 7 个健康日)。从保险公司的角度来看,与继续使用 OTC 泻药相比,处方药(利那洛肽、普芦卡必利和普卡必利)似乎没有成本效益(增量成本效益比>150000 美元/QALY 增加)。从患者的角度来看,成本效益算法从普卡必利开始,然后在 145μg 剂量下在普芦卡必利和利那洛肽之间进行选择(对于那些疾病影响工作生产力的患者,普芦卡必利更有利)。患者的观点受到药物耐受性和生活质量治疗效果的驱动。
在政策层面上解决成本问题有可能使患者和临床医生能够从治疗途径的障碍转移到真正优化治疗选择。