Fawsitt Christopher G, Gallagher Elaine, Singh Alka, Baker Hannah, Kayongo Edward, Thom Howard, Paracha Noman
Clifton Insight, Bristol, UK.
Bayer Pharmaceuticals AG, Basel, Switzerland.
Pharmacoeconomics. 2025 Aug 18. doi: 10.1007/s40273-025-01532-w.
Metastatic castration-sensitive prostate cancer (mCSPC) imposes a significant economic burden and necessitates more cost-effective treatment strategies. The variability among the components of published economic evaluation models leads to methodological inconsistencies, underscoring the need for an optimal framework to minimise unwarranted structural variation. This paper reviews existing economic evaluations, establishes a comprehensive framework and aims to support future economic evaluations and decision-making in mCSPC.
A systematic literature review (SLR) was conducted to identify relevant economic evaluations in mCSPC. Health technology assessments (HTAs) by the National Institute for Health and Care Excellence, and Canada's Drug Agency were reviewed to gather insights on critiques and limitations. On the basis of these findings, a comprehensive cost-effectiveness modelling framework was established. Furthermore, two additional SLRs were conducted to identify cost and resource utilisation inputs, as well as health state utility scores derived from published studies and HTA assessments.
Markov models and partitioned survival models (PSMs) were commonly reported in literature and published HTA evaluations. Despite the strong precedence of PSMs, we propose an optimal framework for mCSPC utilising a semi-Markov structure. This approach offers increased flexibility, allowing transition rates from progressed states to depend on time since progression occurred. We also present key sources of cost and utility data identified in the SLR.
This work aligns with methodologies recommended by the Innovative Medicine Initiative (IMI) PIONEER external group and published studies. The optimal framework, including healthcare resource utilisation and utility data, consolidates existing modelling precedents in mCSPC and will assist the cost-effectiveness assessment of treatments for this condition.
转移性去势敏感性前列腺癌（mCSPC）带来了巨大的经济负担，因此需要更具成本效益的治疗策略。已发表的经济评估模型各组成部分之间的差异导致方法上的不一致，这凸显了需要一个优化框架以尽量减少不必要的结构变异。本文回顾了现有的经济评估，建立了一个全面的框架，旨在支持未来mCSPC的经济评估和决策。
进行了一项系统文献综述（SLR），以识别mCSPC中相关的经济评估。对英国国家卫生与临床优化研究所和加拿大药品管理局的卫生技术评估（HTA）进行了回顾，以收集有关批评意见和局限性的见解。基于这些发现，建立了一个全面的成本效益建模框架。此外，还进行了另外两项SLR，以识别已发表研究和HTA评估中得出的成本和资源利用投入以及健康状态效用评分。
文献和已发表的HTA评估中普遍报告了马尔可夫模型和分区生存模型（PSM）。尽管PSM有很强的先例，但我们为mCSPC提出了一个采用半马尔可夫结构的优化框架。这种方法提供了更大的灵活性，使进展状态的转移率能够取决于进展发生后的时间。我们还介绍了SLR中确定的成本和效用数据的关键来源。
这项工作与创新药物倡议（IMI）先锋外部小组推荐的方法以及已发表的研究一致。这个优化框架，包括医疗资源利用和效用数据，整合了mCSPC中现有的建模先例，并将有助于对这种疾病治疗的成本效益评估。
