Joish V N, Frech F, Lapuerta P
a Lexicon Pharmaceuticals, Inc. , Basking Ridge , NJ , USA.
b Former Employee of Lexicon Pharmaceuticals , Basking Ridge , NJ , USA.
J Med Econ. 2018 Feb;21(2):182-188. doi: 10.1080/13696998.2017.1387120. Epub 2017 Oct 9.
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.
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.
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.
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.
This analysis demonstrated TE is a cost-effective treatment option when used on top of standard of care in CSD patients.
本研究评估了对于类癌综合征腹泻（CSD）且单独使用生长抑素类似物奥曲肽（SSA）症状仍无法得到控制的患者，添加乙基替加曲肽（TE）至奥曲肽（SSA + TE）与单独使用奥曲肽（SSA）相比的成本效益。
一个确定性马尔可夫模型从美国第三方支付方的角度评估了SSA + TE与SSA相比所获得的成本和质量调整生命年（QALY）。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，效用估计基于溃疡性结肠炎的类似症状。治疗效果基于SSA + TE与单独使用SSA的III期临床试验[TELESTAR，NCT01677910]。根据TELESTAR试验，12周后，44%接受SSA + TE治疗的患者和20%接受SSA治疗的患者对治疗有反应。在每4周的评估期，未得到充分控制的SSA患者接受递增剂量的SSA，而接受SSA + TE治疗的患者停用TE并仅改用SSA。对于得到充分控制和未得到充分控制的患者，SSA的药物成本分别为4,291.75美元和5,890.57美元，SSA + TE的药物成本分别为9,456.07美元和5,890.57美元。
基础病例分析表明，SSA队列的终身QALY为1.67，成本为495,125美元；SSA + TE队列的终身QALY为2.33（590,087美元），SSA + TE的增量QALY为0.66，额外成本为94,962美元。每获得一个QALY的增量成本为142,545美元。敏感性分析表明，在300,000 - 450,000美元的罕见病和孤儿药阈值下，SSA + TE具有成本效益的概率很高（>99%）。
TE最近才上市，无法纳入基于实际临床实践模式的临床和经济数据。针对这种罕见疾病的流行病学和效用信息稀缺，需要使用一些替代估计。
该分析表明，对于CSD患者，在标准治疗基础上使用TE是一种具有成本效益的治疗选择。
