Using Real-World Evidence in Health Technology Assessment Submissions: A NICE Case Study

OBJECTIVES: There is a lack of consensus and transparency on using real-world evidence (RWE) in health technology assessment (HTA). In 2022, the National Institute for Health and Care Excellence (NICE) developed an RWE framework. This study explores the use of RWE databases in NICE technology appraisals (TAs) from April 2019.

METHODS: The NICE website was searched to source TAs from April 2019 to May 2024 that mentioned at least 1 of 10 identified RWE databases. Information on how RWE was used and how it was viewed by the External Assessment Group (EAG) and NICE committee were extracted.

RESULTS: 13 TAs mentioned at least 1 of the databases: Clinical Practice Research Datalink (12 TAs), OpenSAFELY (3 TAs), The Health Improvement Network (1 TA) and General Practice Extraction Service Data for Pandemic Planning and Research (1 TA). In 12 TAs, RWE data were used to inform or validate model parameters. In all TAs screened, the committee and EAG did not criticize the use of RWE data extracted from databases. Moreover, in three cases the EAG utilized RWE data to inform alternative base case parameter estimates. In two cases, the committee recommended the manufacturer use RWE data to validate a model assumption. Comments from the committee focused on the relevance and applicability of the RWE to the decision problem.

CONCLUSIONS: It is evident that decision makers respond positively to RWE when it has been correctly employed. For RWE to aid the decision-making process, data must be applicable and relevant to the population of interest. Correct utilization of RWE yields the potential to reduce parameter uncertainty. This study supports the use of RWE in HTA and demonstrates that a standard framework can ensure appropriate use of RWE. Future research could explore RWE use in other HTA agencies to strengthen the current study and broaden its applicability.