Cost-effectiveness analysis of telotristat ethyl for treatment of carcinoid syndrome diarrhea inadequately controlled with somatostatin analogs
Cost-effectiveness analysis of telotristat ethyl for treatment of carcinoid syndrome diarrhea inadequately controlled with somatostatin analogs
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DOI：
10.1080/13696998.2017.1387120
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发表时间：
2018-01-01
影响因子：
2.4
通讯作者：
Lapuerta, P.
中科院分区：
文献类型：
--
作者：
Joish, V. N.;Frech, F.;Lapuerta, P.
目的：本研究评估了将替洛司他乙酯 (TE) 添加到生长抑素类似物奥曲肽 (SSA+TE) 与单用奥曲肽 (SSA) 相比，对于单用 SSA 仍无法控制症状的类癌综合征腹泻 (CSD) 患者的成本效益。 材料和方法：确定性马尔可夫模型评估了 SSA+TE 与 SSA 相比的成本和质量调整生命年 (QALY)。美国第三方付款人的观点。该模型反映了基于当前护理标准的临床实践和资源使用估计，以及基于溃疡性结肠炎的类似症状的效用估计。治疗效果基于 SSA+TE 与单独 SSA 的 III 期临床试验 [TELESTAR, NCT01677910]。根据 TELESTAR 的数据，12 周后，44% 的 SSA+TE 患者和 20% 的 SSA 患者对治疗有反应。在每个 4 周评估期，未得到充分控制的 SSA 患者接受增加剂量的 SSA，而 SSA+TE 患者则停止 TE 并仅转至 SSA。充分控制和未充分控制患者的 SSA 药物成本分别为 4,291.75 美元和 5,890.57 美元，SSA+TE 分别为 9,456.07 美元和 5,890.57 美元。 结果：基本案例分析表明，SSA 队列的终生 QALY 为 1.67，成本为 495,125 美元，SSA 队列的成本为 2.33 美元。 （590,087 美元）对于 SSA+TE，SSA+TE 的 QALY 增量为 0.66，另外需要 94,962 美元。每获得 QALY 的增量成本为 142,545 美元。敏感性分析表明，SSA+TE 在罕见疾病和孤儿药 300,000-450,000 美元的阈值下具有成本效益的可能性很高（>99%）。 局限性：近期 TE 的可用性排除了基于现实世界实践模式的临床和经济投入的结合。由于这种罕见疾病缺乏流行病学和实用信息，因此需要使用一些替代估计。结论：这项分析表明，当在 CSD 患者的标准护理基础上使用 TE 时，TE 是一种具有成本效益的治疗选择。
Aims: This study evaluated the cost-effectiveness of telotristat ethyl (TE) added to somatostatin analog octreotide (SSA+TE) compared to octreotide alone (SSA) in patients with carcinoid syndrome diarrhea (CSD) whose symptoms remain uncontrolled with SSA alone.Materials and methods: A deterministic Markov model evaluated the costs and quality-adjusted life-years (QALY) gained with SSA+TE vs SSA per a third-party US payer perspective. The model reflected clinical practice and resource use estimates based on current standards of care, with utility estimates based on similar symptoms from ulcerative colitis. Treatment efficacy was based on the phase III clinical trial of SSA+TE vs SSA alone [TELESTAR, NCT01677910]. According to TELESTAR, 44% of SSA+TE and 20% of SSA patients responded to therapy after 12 weeks. At each 4-week assessment period, SSA patients not adequately controlled received increasing doses of SSA and SSA+TE patients discontinued TE and moved to SSA only. Drug costs for adequately and not adequately controlled patients were $4,291.75 and $5,890.57 for SSA, respectively, and $9,456.07 and $5,890.57 for SSA+TE, respectively.Results: The base-case analysis demonstrated lifetime QALYs of 1.67 at a cost of $495,125 for the SSA cohort and 2.33 ($590,087) for SSA+TE with an incremental QALY for SSA+TE of 0.66 for an additional $94,962. The incremental cost per QALY gained was $142,545. Sensitivity analyses demonstrated high probability (>99%) of SSA+TE being cost-effective at thresholds for rare diseases and orphan drugs of $300,000-$450,000.Limitations: The recent availability of TE precluded the incorporation of clinical and economic inputs based on real-world practice patterns. The scarcity of epidemiology and utility information for this rare condition required the use of some proxy estimates.Conclusions: This analysis demonstrated TE is a cost-effective treatment option when used on top of standard of care in CSD patients.