OBJECTIVES: Although randomised controlled trials (RCTs) remain the gold standard, there is a trend toward trying to incorporate real-world evidence (RWE) in health technology assessments where appropriate. In this light, the National Institute for Health and Care Excellence (NICE) released a framework in June 2022 to optimise RWE quality and address data uncertainties. This study evaluates the impact of NICE's framework on RWE adoption in reimbursement decisions and identifies key issues and opportunities associated with its implementation.

METHODS: A review and synthesis of NICE HTA submissions from June 23, 2022, to April 25, 2024, was conducted. Extracted information included: disease area, RWE type, rationale for inclusion in the submission, primary objections from NICE, and final recommendation outcome.

RESULTS: Approximately 64% (91/141) of submissions incorporated RWE during the NICE process. However, only 56% (51/91) explicitly mentioned RWE in the final NICE guidance. Forty-five percent (64/141) and 40% (56/141) of submissions related to oncology and rare diseases respectively. Of these, 61% (39/64) and 62% (35/56) utilized RWE. We observed a positive correlation between RWE inclusion and favorable NICE recommendations: 88% (124/141) of drugs were recommended, with 70% (83/124) utilizing RWE. Main reasons for RWE inclusion were generalisability to the UK population (34%, 31/91), reassurance of trial outcomes in routine practice (30%, 27/91) and to support extrapolated outcomes in economic modelling (25%, 21/83). Primary objections included issues with population representativeness (20%, 18/91), concerns about statistical methods (15%, 14/91) and uncertainty due to data immaturity (12%, 11/91).

CONCLUSIONS: The NICE RWE framework appears to demonstrate its potential to enhance reimbursement decisions and decrease uncertainty in the evidence base. Despite ongoing challenges with data representativeness and maturity, 70% of the recommended medicines utilized RWE. Future improvements could focus on strengthening the RWE methodology and ensuring consistent integration of RWE discussions in the final guidance.