Use of Real-World Evidence in Health Technology Reassessments Across Six Health Technology Assessment Agencies

Objectives: To review health technology assessment reassessments (HTARs) and characterize the use of real-world evidence (RWE) in HTARs.

Methods: Six agencies were chosen for inclusion in this targeted review: CADTH, NICE, HAS, G-BA/IQWiG, ZIN, and PBAC. Each agency's assessments were screened to identify their eight most recent HTARs, which were evaluated to determine if they used RWE. If for a given agency, less than half of the screened HTARs used RWE, we identified an additional four HTARs for evaluation. For each reassessment, we extracted drug characteristics, HTAR details, initial assessment details, and if/how RWE was used. Narrative synthesis in conjunction with descriptive statistics were used to characterize the findings.

Results: We identified 40 HTARs across the agencies. Over half of the HTARs were for oncology therapies. Additionally, 55% used RWE; these reassessments tended to be for orphan therapies. RWE was submitted to address at least one clinical uncertainty, with the most common being related to the primary/secondary endpoints. The majority of RWE studies came from registry data (57.1%). Moreover, the proportion of HTARs resulting in no change in patient access was similar between HTARs that did and did not use RWE. Lastly, no de novo RWE comparative effectiveness studies were identified.

Conclusions: Our findings imply that RWE can play a role in addressing uncertainties identified at launch, especially in addition to clinical trial evidence; agencies and sponsors should collaborate/align on evidence needs and study feasibility to ensure RWE can be effectively used in reassessments.