Challenges with integrating early-stage cancer trial endpoints into economic models: review of health technology recommendations for adjuvant or neoadjuvant therapies in Canada.

IF 1.8 4区 医学 Q3 HEALTH CARE SCIENCES & SERVICES
Jaclyn M Beca, Stephanie Gosselin, Don Husereau, Eon Ting
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Abstract

Objectives: Adjuvant and neoadjuvant therapies for early-stage cancers demonstrate early clinical benefit in delaying disease recurrence. Health technology assessments require economic evaluations modeling lifetime disease trajectories. We examined modeling approaches used in Canadian health technology reviews to understand relevant challenges and identify opportunities for methodological improvements.

Methods: From CADTH reimbursement recommendations for adjuvant/neo-adjuvant treatment of solid tumors, we collected outcomes and details of submitted clinical and economic evidence. We classified issues raised during economic review related to data maturity, surrogacy, treatment pathways, and assumptions surrounding extrapolation, duration of benefit and cure.

Results: Reviews from Jul/2015-Mar/2023 were identified. Reimbursement was recommended in 14/18 (78%) reviews. All assessments described OS as immature. Most (9/10, 90%) reviews with interim comparative OS data recommended reimbursement, while several (3/8, 38%) without OS data were not recommended. CADTH revisions changed implications for cost-effectiveness ($50,000/QALY threshold) in 10/18 (56%) reviews. Duration of benefit assumptions were inconsistent among both submitters and reviewers. Cure-time was consistently revised to ≥5 years from initiation.

Conclusions: Despite surrogate endpoints and immature survival data, positive reimbursement recommendations were common. CADTH re-analyses frequently had modest impacts on cost-effectiveness. Further guidance is needed to capture benefits and assess uncertainties with more consistency for early-stage cancers.

将早期癌症试验终点纳入经济模型的挑战:加拿大辅助或新辅助治疗的卫生技术建议综述
目的:早期癌症的辅助和新辅助治疗在延缓疾病复发方面显示出早期临床益处。卫生技术评估需要建立终身疾病轨迹模型的经济评估。我们研究了加拿大卫生技术审查中使用的建模方法,以了解相关挑战并确定方法改进的机会。方法:从辅助/新辅助治疗实体瘤的CADTH报销建议中,我们收集了提交的临床和经济证据的结果和细节。我们对经济回顾中提出的问题进行了分类,这些问题与数据成熟度、替代、治疗途径以及围绕外推的假设、受益和治愈的持续时间有关。结果:对2015年7月- 2023年3月的综述进行了识别。14/18次(78%)审查建议报销。所有评估都将OS描述为不成熟的。大多数(9/10,90%)有中期比较OS数据的综述推荐报销,而一些(3/8,38%)没有OS数据的综述不推荐报销。CADTH修订在10/18(56%)审查中改变了成本效益(50,000美元/QALY阈值)的含义。在提交者和审稿人中对获益持续时间的假设是不一致的。治疗时间一致修改为起始后≥5年。结论:尽管有替代终点和未成熟生存数据,积极的报销建议是常见的。CADTH重新分析通常对成本效益影响不大。需要进一步的指导,以更一致地获取早期癌症的益处和评估不确定性。
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来源期刊
Expert Review of Pharmacoeconomics & Outcomes Research
Expert Review of Pharmacoeconomics & Outcomes Research HEALTH CARE SCIENCES & SERVICES-PHARMACOLOGY & PHARMACY
CiteScore
4.00
自引率
4.30%
发文量
68
审稿时长
6-12 weeks
期刊介绍: Expert Review of Pharmacoeconomics & Outcomes Research (ISSN 1473-7167) provides expert reviews on cost-benefit and pharmacoeconomic issues relating to the clinical use of drugs and therapeutic approaches. Coverage includes pharmacoeconomics and quality-of-life research, therapeutic outcomes, evidence-based medicine and cost-benefit research. All articles are subject to rigorous peer-review. The journal adopts the unique Expert Review article format, offering a complete overview of current thinking in a key technology area, research or clinical practice, augmented by the following sections: Expert Opinion – a personal view of the data presented in the article, a discussion on the developments that are likely to be important in the future, and the avenues of research likely to become exciting as further studies yield more detailed results Article Highlights – an executive summary of the author’s most critical points.
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