OBJECTIVES

Recent scientific advances in precision and personalised medicine have shown curative potential in areas of high unmet need within oncology. The EMA has recognised this evolution by introducing adaptive licencing, however, NICE has maintained the same cost-effectiveness methods for 20 years without any significant change to valuing curative innovation. This targeted review aims to identify key areas of NICE methods that impact value assessment, uncertainty and innovation recognition in England for EMA-approved oncology treatments with curative intent.

METHODS

Targeted electronic literature search of committee papers and final appraisal documents in the online NICE guidance database.

• Search term: ‘cure model’.
• Inclusion criteria: technology appraisals in oncology, January 2009-September 2019.
• Main data extraction topics: definition of cure, clinical trial design, key areas of uncertainty, end of life (EoL) criteria and recommendations for inclusion in the Cancer Drugs Fund (CDF).

RESULTS

Data extraction was conducted on 19 technology appraisals. The definition of cure was heterogenous across technology appraisals. In TA589 and TA541 both in acute lymphoblastic leukaemia, cure was defined by patients remaining alive at 5 and 3 years respectively. Clinical effectiveness evidence was often from single arm phase II trials (37%). The main drivers of cost-effectiveness were cure assumptions. Eighty-nine percent of the interventions identified were recommended for use in the CDF and 32% met NICE’s EoL criteria

CONCLUSIONS

Uncertainty was a key issue for cure modelling in all appraisals, as evidenced by the high proportion of CDF recommendations. These are subjected to lower ICER thresholds in an attempt to alleviate the decision risk attributed to uncertainty. Results suggest that innovative drugs with curative intent are penalised on their evidence base, which is often from single arm trials, identify aspects of NICE methods for reform and highlight misalignment between HTA requirements and EMA adaptive licensing.