Analysis of Intensity of Use of Real-World Data in Single Technology Appraisals of Cancer Drugs By the National Institute for Health and Care Excellence

OBJECTIVES: Studies reviewing use of real-world data (RWD) by simply counting how often an appraisal uses RWD can only offer a limited understanding of how intensively RWD are used in drug appraisals. This study investigates the intensity of use of RWD and what factors are associated with more intensive use of RWD in the economic modelling informing single technology appraisals of cancer drugs by the National Institute for Health and Care Excellence (NICE).

METHODS: Data were extracted from NICE appraisals of cancer drugs (January 2011 - December 2022, n=267). These appraisals were classified into intensity groups by how many times RWD were used for major (choice of comparator, estimating overall survival, estimating volume of treatment) and minor components of the economic evaluation (other components of the economic model). Generalised ordered regression was used to test hypotheses concerning the higher level of intensity of using RWD.

RESULTS: Half of the appraisals either did not use RWD or only made minor use of RWD in the economic models. While major use of RWD increased over time, there has been no clear change in intensity. RWD were more often used for major components in appraisals of treatments for skin cancer, than for other types of cancer. The availability of direct treatment comparisons and the maturity of survival data are inversely associated with the intensity with which RWD are used.

CONCLUSIONS: Intensive use of RWD was not made in appraisals despite fairly wide use of RWD. The intensity of using RWD in economic modelling is increased when direct treatment comparison is not available. The intensity with which RWD are used may be expected to increase as medical regulators base more of their decision-making on single-arm trials. However, the potential for bias associated with RWD may limit its more intensive use in economic modelling.