Regulator‐Requested Non‐Interventional Postauthorization Safety and Effectiveness Studies for Oncology Drugs: A Systematic Review

There has been a growing number of oncology drug approvals. Non‐interventional postauthorization safety/effectives studies (PASSs/PAESs) aim to provide real‐world evidence on the safety/effectiveness of oncology drugs postapproval. To understand the current landscape, a comprehensive search as of March 1, 2021, was conducted in major register/databases. We found that requested studies increased from 1 in 2006–2010 to 47 in 2016–2020. Of 78 total studies identified, 50 focused on safety/risk assessment (64.1%), 6 on effectiveness (7.7%), 3 on drug utilization (3.8%), 1 on disease epidemiology (1.3%), and 18 on effectiveness of additional risk minimization measures (23.1%). For safety/risk assessment studies, 58.9% focused on nonspecific end points (e.g., frequency of adverse events). For effectiveness studies, the leading primary outcome was overall survival. Overall, safety/risk assessment studies concerning nonspecific end points for vascular endothelial growth factor (receptor) inhibitors were most requested. Regarding data sources, though a majority (71.8%) used primary data collection, a growing proportion utilized secondary data sources. Among 23 studies with information available on study design, 10 (43.5%) were single‐arm cohort studies, 9 (39.1%) were cross‐sectional studies, 3 (13.1%) were comparative cohort studies, and 1 (4.3%) was a nested‐case‐control study. In conclusion, there was an increasing number of oncology‐specific non‐interventional PASSs/PAESs, with a majority on safety/risk assessment. Although most utilized primary data collection, there was an increasing use of secondary real‐world data sources. Few conducted comparative analyses, and most used single‐arm designs. Future efforts are needed to assess how findings of these studies would impact regulatory decisions and improve knowledge of toxicity for clinical/translational development.