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.
The study highlights an important issue in the decision-making process for cancer treatments in the United Kingdom. This research is significant as the National Institute for Health and Care Excellence (NICE) plays a crucial role in determining which cancer drugs are available to patients through the National Health Service (NHS). The findings reveal that 56% of NICE recommendations for cancer technologies during this period relied on immature survival data, which means that the data was incomplete or not fully developed at the time of evaluation.
The use of immature survival data can lead to misleading conclusions regarding the cost-effectiveness of new treatments. The study found that the majority of recommendations based on this type of data were positive, despite not having fully matured evidence. This raises the risk that the resources may not be allocated efficiently.
Furthermore, the research indicates that decisions made based on immature data are rarely reviewed or updated, particularly for those not placed in the Cancer Drugs Fund. Only one out of the many drug approvals based on immature data was revisited when more mature data became available. This lack of reappraisal means that many potentially inappropriate recommendations could remain uncorrected, affecting both patients and the healthcare system.
Overall, the study emphasizes the need for vigilance by NICE and other health technology assessment authorities when making recommendations based on incomplete data. It suggests that while the pressure to provide quick access to new treatments is understandable, it could lead to a misallocation of resources in the long-term if these decisions are not systematically reevaluated as more data becomes available. Therefore, it is crucial for decision makers to ensure that recommendations reflect complete and mature evidence to promote effective and efficient healthcare.