Kang Jiyeon, Cairns John, Latimer Nicholas R, Duffield Stephen, Grieve Richard
Department of Health Service Research and Policy, Faculty of Public Health and Policy, London School of Hygiene and Tropical Medicine, London, UK; Centre for Cancer Biomarkers, University of Bergen, Bergen, Norway.
Department of Health Service Research and Policy, Faculty of Public Health and Policy, London School of Hygiene and Tropical Medicine, London, UK; Centre for Cancer Biomarkers, University of Bergen, Bergen, Norway.
Value Health. 2025 Jul 22. doi: 10.1016/j.jval.2025.07.010.
This study examines the maturity of survival data used in cancer drug appraisals by the National Institute for Health and Care Excellence (NICE) and the implications for decision making.
We assessed the maturity of survival data used in economic models within NICE single technology appraisals published between January 1, 2011 and December 31, 2023 (n = 301). We categorized these survival data according to whether they were "highly immature" (<20% of events), "immature" (20%-50%), or "mature" (>50%). We applied multinomial logistic regression analysis to assess the association of factors such as time period, the introduction of the Cancer Drugs Fund (CDF), cancer type, disease severity/stage, technology type, and trial design (single-arm or randomized controlled trial), with the maturity of the survival data. We then assessed the association of the maturity of the survival data with the subsequent recommendation of the NICE appraisal committee.
After adjusting for potential confounders, the percentage of appraisals with highly immature survival data increased from 25.1% (pre-CDF) to 40.4% (post-CDF) (P = .105). Appraisals that used single-arm trials or were for early-stage cancers were more likely to use highly immature survival data. Those technologies with highly immature data were more likely to receive CDF recommendations (30.4% vs 11.5%, P = .007).
The trend toward more NICE single technology appraisals of cancer drugs relying on immature survival data are consistent with moves by regulatory agencies to encourage expedited approvals for innovative therapies. For Health Technology Assessment decision-making, it is essential to balance early drug access with the use of robust evidence.
本研究考察了英国国家卫生与临床优化研究所（NICE）在癌症药物评估中使用的生存数据的成熟度及其对决策的影响。
我们评估了2011年1月1日至2023年12月31日期间NICE单一技术评估中经济模型所使用的生存数据的成熟度（n = 301）。我们根据这些生存数据是“极不成熟”（事件发生比例<20%）、“不成熟”（20%-50%）还是“成熟”（>50%）进行分类。我们应用多项逻辑回归分析来评估诸如时间段、癌症药物基金（CDF）的设立、癌症类型、疾病严重程度/分期、技术类型和试验设计（单臂试验或随机对照试验）等因素与生存数据成熟度之间的关联。然后，我们评估了生存数据成熟度与NICE评估委员会随后的建议之间的关联。
在对潜在混杂因素进行调整后，使用极不成熟生存数据的评估比例从CDF设立前的25.1%增加到了CDF设立后的40.4%（P = 0.105）。使用单臂试验或针对早期癌症的评估更有可能使用极不成熟的生存数据。那些数据极不成熟的技术更有可能获得CDF的推荐（30.4%对11.5%，P = 0.007）。
NICE对癌症药物进行更多单一技术评估时越来越依赖不成熟生存数据的趋势与监管机构鼓励加快创新疗法审批的举措相一致。对于卫生技术评估决策而言，平衡早期药物可及性与使用有力证据至关重要。
