OBJECTIVES

Real-world evidence (RWE) can complement randomised clinical trial data and inform healthcare decision making^1,2,3. This study aims to identify the level of utilisation of RWE, as well as its impact, within the NICE appraisal process, for the most significant oncology drugs.

METHODS

The top 20 oncology drugs, by worldwide revenue, were identified from published data. The relevant Single Technology Appraisals (STAs) were found on the NICE website and reviewed. The manufacturer submissions, committee discussions and Evidence Review Group (ERG) reports were inspected, and all elements of RWE were identified, extracted and analysed to draw conclusions regarding the purpose of RWE use, data source and its impact on the appraisal (incorporation in the model, committee consideration).

RESULTS

64 NICE oncology STAs were identified. 16 submissions of 11 drugs included at least one instance of RWE (19 instances in total) and 14 of them (87.5%) resulted in approvals. More than half of RWE were sourced from institutional electronic records (10). 8 RWE sources referred to survival outcomes, 5 to health-related quality of life (HRQoL), 2 to defining the current standard of care, 2 to surrogate marker measurement, 1 to resource use for health states and 1 to time until treatment discontinuation. Overall, the RWE utilised had an impact on 8 out of the 16 appraisals. The main reasons for RWE being criticised in the appraisals were uncertainty due to small patient cohorts, and study patient populations not relevant to the specific indication assessed. RWE was found to be increasingly utilised with time, following a similar pattern of the increase in the number of oncology submissions.

CONCLUSIONS

This study provides an overview of the utilisation of RWE in oncology appraisals. RWE has a growing presence in NICE oncology submissions, yet, tackling the uncertainty that often comes with it, remains a challenge.