Challenges with integrating early-stage cancer trial endpoints into economic models: review of health technology recommendations for adjuvant or neoadjuvant therapies in Canada.
Jaclyn M Beca, Stephanie Gosselin, Don Husereau, Eon Ting
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引用次数: 0
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.
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.