Abstract

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: Canadian Agency for Drugs and Technologies in Health, National Institute for Health and Care Excellence, Haute Autorité de Santé, Gemeinsamer Bundesausschuss/Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, Zorginstituut Nederland, and Pharmaceutical Benefits Advisory Committee. Each agency’s assessment was screened to identify their 8 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 4 HTARs for evaluation. For each reassessment, we extracted drug characteristics, HTAR details, initial assessment details, and if/how the 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 1 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.