Trends Underlying Positive and Negative Decision-Making for New Oncology Treatments Appraised by the National Institute for Health and Care Excellence (NICE) in 2023

OBJECTIVES: Oncology is a rapidly evolving therapeutic area, accounting for 41% of Health Technology Assessments by NICE in 2023. This research aimed to identify trends in decision-making for emerging oncology treatments in England.

METHODS: NICE technology appraisals for oncology indications published in 2023 were identified. Pre-defined topics, including NICE recommendation, clinical and economic evidence submissions, and decision drivers, were extracted with spot checks by a second reviewer.

RESULTS: NICE published guidance on 33 oncology treatments in 2023. Although 79% were recommended (26/33), only 58% of these were recommended in line with EMA marketing authorization (MA;15/26). Most recommended not in accordance with MA were either restricted by treatment line (55%;6/11) or to a patient subgroup by biomarker, treatment history/contraindications, or prognostic index (55%;6/11; not mutually exclusive). While most submissions included evidence from Phase 3 trials (73%;24/33), 3 relied on Phase 1/2 evidence. These 3 were all recommended and were: restricted by treatment line (1), orphan medicines (2), considered innovative (1), supplemented with real-world evidence (RWE;2), and relied on funding within the Cancer Drugs Fund (CDF;2). For TA specifying a treatment line (19/33;58%), more first/second-line treatments were recommended (12/13;92%) compared to third-line plus (3/6;50%); despite the committee’s recognition of the unmet need in later-lines. Overall, negative recommendations (21%;7/33) were driven by lack of cost-effectiveness (100%;7/7), lack of direct comparative clinical evidence (43%;3/7), and uncertainty in clinical data (57%;4/7), cost-effectiveness estimates (100%;7/7), and indirect treatment comparisons (57%;4/7).

CONCLUSIONS: Negative decisions were frequently driven by data uncertainty, impacting the reliability of cost-effectiveness estimates. Despite significant unmet need, price justification in later-lines can be difficult given the challenge in demonstrating survival benefit. Recommendations based on early-phase studies suggests a willingness to accept uncertainty to accelerate patient access, facilitated with use of the CDF. Therefore, manufacturers should consider innovative approaches to mitigate uncertainties for conditions facing data generation challenges.