Abstract
BACKGROUND: With the growing notion of patient-focused drug development, the quality of life and other patient-reported outcomes (PROs) of cancer patients are gaining considerable attention. Several drug regulatory agencies, including the U.S. Food and Drug Administration (FDA), are calling attention to PROs. This review aims to comprehensively characterise the application of PROs and regulatory considerations for PROs in the FDA-approved novel oncology drugs. METHODS: The FDA review documents and labels for novel oncology drugs approved from July 2017 to July 2022 were retrieved. We collected and analysed drug approval information, types of endpoints for PROs, PRO measures, designs of trials including PROs, and regulatory comments on PRO-related contents. FINDINGS: Results demonstrated that PROs were used more commonly for solid tumours than hematologic malignancies, which might be correlated with the disease characteristics. We further categorised and analysed existing PRO measures, providing insight for tool selection in future oncology trial design. Our findings also indicated that PROs currently do not play a significant role in oncology drug approvals. The major deficiencies related to PROs commented on by FDA reviewers were analysed, followed by recommendations for improvements. INTERPRETATION: This review demonstrates that PROs currently do not play a significant role in oncology drug marketing review, and how they can be used to support the approval of new oncology drugs is still in the exploratory stage. This current situation is not only related to the deficiencies in the design and implementation of PRO-related contents in oncology trials, but more importantly, it is a reminder that we should pay more attention to patient experience in the development of oncology drugs. FUNDING: This study was not supported by any funding.