Prevalence of immature survival data for anti-cancer drugs presented to the National Institute for Health and Care Excellence between 2018-2022

Objectives

Between 2015 and 2017, 41% of 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 importantly different conclusions, compared to cost-effectiveness re-estimations 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. 54% (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 one 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.