Role of Real-World Evidence for Treatments Granted Access Under Cancer Drugs Fund: Lessons and Insights From Review of NICE Technology Assessment Reports (2019-2022)

OBJECTIVES: In oncology, surrogate endpoints are commonly used by regulatory agencies to grant approval to new drugs, enabling early access. Many of these treatments are reimbursed under the Cancer Drugs Fund (CDF), allowing patients to access the treatment while real-world evidence (RWE) is collected. The objective of this analysis was to understand the role of RWE in NICE's final recommendations for drugs covered under the CDF.

METHODS: A systematic review of all 2019-2022 NICE HTAs was conducted using NOVEL-HTA database. Cancer drug assessments with a mention of CDF in the Final recommendations were reviewed for RWE evidence that NICE had cited. For selected reports, case studies were developed for insights, and lessons learned.

RESULTS: During 2019-June 2022, there were 148 HTA reports published by NICE. Vast majority of the reports were for oncology (53%). 40 (~50%) of all reviewed reports included mention of “Cancer Drugs Fund” in the final recommendation. In 20 assessments, NICE recommended the treatment to be covered under the CDF due to lack of overall survival (OS) data or uncertainty in long-term clinical benefits. Interestingly, in 12 re-assessments, NICE mentioned that access under CDF enabled real-world evidence generation and confirmed the overall survival benefit, leading to recommendations to grant full access. In 3 assessments, NICE said CDF provided limited but supportive evidence confirming trial data. In 1 assessment, CDF data was not useful because standard of care in the UK had changed. NICE mentioned in 1 assessment that lower dose usage was observed in the real-world setting. Only 1 treatment was mentioned an unable to show OS benefit.

CONCLUSIONS: Review of assessments shows that for several treatments RWE was useful to confirm OS and long-term benefits. Reimbursement through special funding channels and RWE helps patients gain early access to life saving treatments.