Gibbons Cara L, Latimer Nicholas R
Sheffield Centre for Health and Related Research, University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA, England, UK.
Sheffield Centre for Health and Related Research, University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA, England, UK; Delta Hat Limited, Nottingham, England, UK.
Value Health. 2025 Mar;28(3):406-414. doi: 10.1016/j.jval.2024.11.013. Epub 2024 Dec 24.
Between 2015 and 2017, 41% of National Institute for Health and Care Excellence (NICE) cancer single-technology appraisal (STA) decisions relied upon immature survival data. This occurs when clinical trials that form the evidence base in support of new or existing technologies suffer from limited follow-up. During this period, NICE did not negatively recommend any cancer technologies that used immature data. This suggests a potential incentive to submit to NICE with immature data to avoid rejection. Using immature survival data in cost-effectiveness evaluations has resulted in significantly different conclusions compared with cost-effectiveness reestimations using matured data. We assessed the reliance on immature survival data in NICE decision making of cancer treatments, appraised after 2017.
A structured literature review of NICE cancer STAs published between 2018 and 2022 was conducted. The relationship between data maturity and NICE recommendations was assessed, and the extent to which past decisions were later reviewed was explored.
56% (n = 57) of NICE's cancer recommendations relied upon immature survival data. Fifty-four percent (n = 31) of these received a positive recommendation, 39% (n = 22) were placed into the Cancer Drugs Fund (CDF), and 7% (n = 4) received a negative recommendation. STAs with mature data received a similar proportion of negative recommendations. Only 1 non-CDF recommendation based on immature data was reappraised using updated survival data.
The majority of NICE cancer technology decisions are based on immature survival data and receive positive recommendations. Non-CDF decisions are unlikely to be reappraised. Consequently, many technologies could receive an inappropriate recommendation based on immature data and not be subsequently rectified.
在2015年至2017年期间，英国国家卫生与临床优化研究所（NICE）41%的癌症单一技术评估（STA）决策依赖于不成熟的生存数据。当作为支持新技术或现有技术证据基础的临床试验随访时间有限时，就会出现这种情况。在此期间，NICE没有对任何使用不成熟数据的癌症技术给出负面推荐。这表明存在一种潜在的动机，即提交不成熟数据给NICE以避免被拒。与使用成熟数据进行成本效益重新估计相比，在成本效益评估中使用不成熟的生存数据得出的结论有显著差异。我们评估了2017年以后NICE对癌症治疗决策中对不成熟生存数据的依赖情况。
对2018年至2022年期间发布的NICE癌症STA进行了结构化文献综述。评估了数据成熟度与NICE推荐之间的关系，并探讨了过去决策后来被重新审查的程度。
NICE 56%（n = 57）的癌症推荐依赖于不成熟的生存数据。其中54%（n = 31）获得了正面推荐，39%（n = 22）被纳入癌症药物基金（CDF），7%（n = 4）获得了负面推荐。有成熟数据的STA获得负面推荐的比例相似。只有1项基于不成熟数据的非CDF推荐使用更新后的生存数据进行了重新评估。
NICE大多数癌症技术决策基于不成熟的生存数据并获得正面推荐。非CDF决策不太可能被重新评估。因此，许多技术可能基于不成熟数据获得不恰当的推荐，且随后无法得到纠正。
