Fischer Aren, Mac Stephen, Freiman Erica Stivelman, Marshall John K, Rand Kim, Ramos-Goñi Juan M
Takeda Canada Inc., Toronto, ON, Canada.
Alexion Pharmaceuticals, Mississauga, ON, Canada.
Pharmacoecon Open. 2025 Jan;9(1):41-56. doi: 10.1007/s41669-024-00523-5. Epub 2024 Oct 8.
Vedolizumab is a gut-selective anti-lymphocyte trafficking biologic indicated for the treatment of adult patients with moderately to severely active ulcerative colitis (UC) and Crohn's disease (CD) in Canada.
The objective of this study was to evaluate the cost effectiveness of treatment sequencing for UC and CD from a public healthcare payer perspective, leveraging new real-world evidence from the literature and the EVOLVE study, a retrospective chart review.
Using separate decision tree/Markov models to assess cost effectiveness for UC and CD, two sequencing approaches were estimated for adult patients (≥ 18 years) diagnosed with UC or CD who were biologic-naïve: vedolizumab as first-line biologic followed by anti-tumor necrosis factor (TNF)-α versus first-line anti-TNFα followed by vedolizumab. Treatment effectiveness (response and remission), surgery rates, dose escalation and regain of response and safety inputs were estimated from EVOLVE, a retrospective chart review of real-world data, and evidence synthesis from the literature, whereas costs and utilities were estimated from health technology assessment reports, clinical trials, and the literature. Biosimilar costs were used for anti-TNFα. Both models simulated a 5-year time horizon and discounted costs and outcomes at 1.5%. Probabilistic base-case analyses (n = 10,000) reported total costs (2023 Canadian dollars) and quality-adjusted life-years (QALYs). Several scenario analyses were conducted to explore robustness of results.
In UC, vedolizumab as a first-line biologic followed by anti-TNFα resulted in an incremental gain of 0.09 QALYs (2.46 vs. 2.55) and saved $7179 ($134,028 vs. $126,848), making this a dominant strategy compared with first-line anti-TNFα followed by vedolizumab. In CD, use of vedolizumab as a first-line biologic resulted in an incremental gain of 0.04 QALYs (3.35 vs. 3.39) at an incremental cost of $50,631 ($89,850 vs. $140,381) versus first-line anti-TNFα followed by vedolizumab (incremental cost-effectiveness ratio of $1,265,775 per QALY).
Based on this analysis, sequencing vedolizumab as a first-line biologic prior to anti-TNFα in UC and CD provided additional clinical benefit to patients. In UC, vedolizumab as a first-line biologic also saved healthcare system costs compared with anti-TNFα, whereas in CD, vedolizumab provided incremental benefit at an incremental cost, which was not considered cost effective at a threshold of $50,000/QALY.
维多珠单抗是一种肠道选择性抗淋巴细胞迁移生物制剂，在加拿大被批准用于治疗中度至重度活动性溃疡性结肠炎（UC）和克罗恩病（CD）的成年患者。
本研究的目的是从公共医疗支付方的角度，利用文献中的新真实世界证据和回顾性图表审查的EVOLVE研究，评估UC和CD治疗顺序的成本效益。
使用单独的决策树/马尔可夫模型评估UC和CD的成本效益，针对初治生物制剂的成年患者（≥18岁）诊断为UC或CD估计了两种治疗顺序方法：维多珠单抗作为一线生物制剂，随后使用抗肿瘤坏死因子（TNF）-α，与一线抗TNFα随后使用维多珠单抗。治疗效果（反应和缓解）、手术率、剂量递增以及反应恢复和安全性数据来自EVOLVE（一项对真实世界数据的回顾性图表审查）以及文献证据综合，而成本和效用则根据卫生技术评估报告、临床试验和文献进行估计。抗TNFα使用生物类似药成本。两个模型均模拟了5年的时间范围，并以1.5%的贴现率对成本和结果进行贴现。概率性基础案例分析（n = 10,000）报告了总成本（2023加元）和质量调整生命年（QALY）。进行了多项情景分析以探索结果的稳健性。
在UC中，维多珠单抗作为一线生物制剂随后使用抗TNFα导致QALY增加0.09（2.46对2.55），并节省了7179加元（134,028加元对126,848加元），与一线抗TNFα随后使用维多珠单抗相比，这是一种优势策略。在CD中，使用维多珠单抗作为一线生物制剂导致QALY增加0.04（3.35对3.39），增量成本为50,631加元（89,850加元对140,381加元），而一线抗TNFα随后使用维多珠单抗（每QALY的增量成本效益比为1,265,775加元）。
基于该分析，在UC和CD中，在抗TNFα之前将维多珠单抗作为一线生物制剂进行治疗顺序安排为患者提供了额外的临床益处。在UC中，与抗TNFα相比，维多珠单抗作为一线生物制剂还节省了医疗系统成本，而在CD中，维多珠单抗以增量成本提供了增量益处，在每QALY 50,000加元的阈值下，这被认为不具有成本效益。
