Prevalence of Immature Survival Data for Anticancer Drugs Presented to the National Institute for Health and Care Excellence Between 2018 and 2022.

Abstract

Objectives: 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.

Methods: 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.

Results: 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.

Conclusion: 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.