An Assessment of the Maturity of Cancer Survival Data Used in Economic Models for the National Institute for Health and Care Excellence's Single Technology Appraisals.

Abstract

Objectives: 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.

Methods: 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.

Results: 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).

Conclusions: 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.