OBJECTIVES: Real world data (RWD) generated from non-interventional studies are gradually being utilised alongside evidence from randomised controlled trials (RCT) to inform funding and licensing decision-making for new technologies. This review aimed to describe the recent utilisation of RWD in National Institute for Health and Care Excellence (NICE) health technology assessment (HTA) submissions in England.

METHODS: We conducted a review of all NICE HTA submissions and committee appraisals since 2018 (as of 17 March 2022). Relevant information, including a description of study characteristics (i.e., therapy area, study design and data source) and committee comments relating to RWD was extracted and recorded in an electronic database.

RESULTS: Of 219 submissions appraised by NICE since 2018, 69 (32%) included RWD, most commonly with an oncology focus (68%). Of the studies involving RWD, 46% were retrospective studies and 36% were prospective (12% incorporated both; 6% unknown). Sources used to generate RWD included bespoke chart reviews (28%), existing databases (e.g., HES, CPRD, SACT; 26%), patient access schemes (19%) and registries/other (14%), while 12% of submissions included RWD from multiple sources. The committee specifically appraised the quality and robustness of RWD in 48% of RWD submissions, focusing on areas such as rationale provided, generalisability/representativeness, analytical methodology (e.g., confounder adjustment), sample size and follow-up duration. Guidance recommended further RWD collection for 13% (29/219) of submissions (19/29 had not yet submitted any RWD).

CONCLUSIONS: These findings highlight the increasing importance of RWD for technology appraisals, with approximately one-third of submissions since 2018 including RWD. This represents a considerable increase compared with our previous review of HTA submissions in 2015-16 (Glen and Pulfer, ISPOR Europe 2017). These findings also support the requirement for standardised guidance, such as the upcoming NICE Real World Evidence Framework, to inform best practices for the collection and reporting of RWD for HTA submissions.