OBJECTIVES: Clinical outcome assessments (COAs) are measurements that represent aspects of a patient’s health status and are used to evaluate clinical benefit when developing a therapy for a disease or condition. Types of COAs include patient-reported outcomes (PROs), clinician-reported outcomes (ClinROs), and performance outcomes (PerfOs). Given the increasing emphasis on patient-centered care, PROs have become especially important in recent years. The objective of the current study was to assess the use of different endpoint types, particularly PROs, in confirmatory studies of new molecular entities (NMEs) approved from 2016-2019.

METHODS: A descriptive analysis was conducted utilizing data from NMEs approved by the Food and Drug Administration (FDA) for treatment between January 1, 2016 and December 31, 2019. FDA approval drug labels were reviewed to identify the drug indication and endpoints of confirmatory studies. ICD-10 codes were used to classify the disease type, and the primary and secondary endpoints were classified based on the type of endpoint (survival, biomarker, ClinRO, ObsRO, PRO, PerfO).

RESULTS: A total of 122 NMEs were approved by the FDA for treatment during 2016-2019. Biomarkers (either alone or in conjunction with other endpoint types, allowing for summation of >100%) were the most common primary endpoint (56.2%), followed by ClinROs (48.8%), PROs (24.8%), survival (14.9%), and PerfOs (5.8%). PROs as a primary endpoint were most prominent in pivotal studies related to nervous system diseases (57.1%). The use of PROs as a primary endpoint has almost doubled from what was previously reported in 2011-2015 (13.2%; Gnanasakthy 2016).

CONCLUSIONS: Although the majority of pivotal studies were largely based on biomarkers and ClinROs in the past 4 years, PROs have seen a sharp increase in use as the primary endpoint in pivotal studies, almost doubling from 2011-2015 to 2016-2019, indicating the rising importance of the patient perspective in trial outcomes.