Optum, 1325 Boylston Street, Boston, MA, 02215, USA.
Formerly affiliated with Optum, 5500 North Service Road, Suite 501, Burlington, ON, L7L 6W6, Canada.
Pharmacoeconomics. 2020 Jun;38(6):607-618. doi: 10.1007/s40273-020-00896-5.
Carcinoid syndrome, a rare condition in patients with neuroendocrine tumours, characterised by flushing and diarrhoea, severely affects patients' quality of life. The current carcinoid syndrome standard of care includes somatostatin analogues, but some patients experience uncontrolled symptoms despite somatostatin analogue therapy. Telotristat ethyl is a novel treatment approved by the European Medicines Agency (EMA) and US FDA that significantly reduces bowel movement frequency in patients with uncontrolled carcinoid syndrome.
We developed a model to evaluate the 5-year budget impact of introducing telotristat ethyl to standard care in Swedish patients with uncontrolled carcinoid syndrome.
Treatment response in the 12-week phase III TELESTAR trial (NCT01677910) informed telotristat ethyl efficacy; subsequently, health states were captured by a Markov model using 4-week cycles. TELESTAR open-label extension data informed telotristat ethyl discontinuation. The number of treatment-eligible patients was estimated from literature reviews reporting the prevalence, incidence and mortality of carcinoid syndrome. A Swedish database study informed real-world costs related to carcinoid syndrome and carcinoid heart disease costs. Telotristat ethyl market share was assumed to increase annually from 24% (year 1) to 70% (year 5).
Over the 5-year model horizon, 44 patients were expected to initiate telotristat ethyl treatment. The cumulative net budget impact of adding telotristat ethyl to current standard of care was €172,346; per-year costs decreased from €66,495 (year 1) to €29,818 (year 5). Increased drug costs from adding telotristat ethyl were offset by reduced costs elsewhere.
The expected budget impact of adding telotristat ethyl to the standard of care in Sweden was relatively low, largely because of the rarity of carcinoid syndrome.
类癌综合征是一种罕见的神经内分泌肿瘤患者的病症，其特征为潮红和腹泻，严重影响患者的生活质量。目前的类癌综合征标准治疗包括生长抑素类似物，但一些患者尽管接受了生长抑素类似物治疗，但仍出现症状无法控制的情况。替利曲塞乙酯是一种新型治疗药物，已获得欧洲药品管理局（EMA）和美国食品药品监督管理局（FDA）的批准，可显著降低无法控制的类癌综合征患者的排便频率。
我们开发了一种模型，以评估替利曲塞乙酯引入瑞典无法控制的类癌综合征标准治疗的 5 年预算影响。
TELESTAR 三期试验（NCT01677910）中的 12 周治疗反应为替利曲塞乙酯的疗效提供了信息；随后，使用 4 周周期的 Markov 模型捕捉健康状态。TELESTAR 开放标签扩展数据提供了替利曲塞乙酯停药的信息。根据文献综述中报告的类癌综合征患病率、发病率和死亡率，估计了符合治疗条件的患者人数。一项瑞典数据库研究提供了与类癌综合征和类癌性心脏病相关成本的真实世界成本信息。假设替利曲塞乙酯的市场份额将从第 1 年的 24%逐年增加至第 5 年的 70%。
在 5 年模型预测期内，预计有 44 名患者将开始替利曲塞乙酯治疗。将替利曲塞乙酯添加到当前标准治疗中的累积净预算影响为 172346 欧元；每年的成本从第 1 年的 66495 欧元下降到第 5 年的 29818 欧元。添加替利曲塞乙酯增加了药物成本，但同时也降低了其他方面的成本。
将替利曲塞乙酯添加到瑞典标准治疗中的预期预算影响相对较低，这主要是因为类癌综合征的罕见性。
