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

OBJECTIVES: Health technology assessment (HTA) agencies can evaluate drugs at multiple time points across the lifecycle (i.e., reassessments) to ensure a drug’s value to the health system. Real-world evidence (RWE) is an important tool for HTA reassessments (HTARs), but there is limited guidance on how RWE should be used. An evaluation of the use of RWE in HTARs is needed to inform evidence-generation strategies and promote successful patient access; however, this literature is limited.

METHODS: Six agencies were chosen for inclusion based on predefined criteria: CADTH, NICE, HAS, G-BA/IQWiG, ZIN, and PBAC. Each agency’s assessments were screened to identify their most recent eight HTARs. HTARs were then evaluated to determine if they used RWE. If for a given agency, less than 50% of the screened HTARs used RWE, we identified an additional four HTARs for evaluation. For the HTARs that used RWE, we extracted the type of RWE and how it was used in HTA decision-making.

RESULTS: We identified 40 HTARs across the six HTA agencies. Over 50% of 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 uncertainties being the primary and secondary endpoints. Registry data were the most commonly utilized. Moreover, 59% of HTARs that used RWE resulted in no change in patient access, a proportion similar to the HTARs that did not use RWE (56%).

CONCLUSIONS: RWE was used in more than half of the identified HTARs to address (mostly clinical) uncertainties stemming from the initial assessment. This finding implies that manufacturers should proactively determine how RWE can address uncertainties in clinical development in anticipation of post-launch evidence needs, and agencies and sponsors should collaborate/align on evidence needs and study feasibility.