OBJECTIVES: To assess the extent of use of endpoints based on survival, biomarker and clinical outcome assessments (COAs) consisting of Clinician-reported outcomes (ClinROs), Observer-reported outcomes (ObsROs), Patient-reported outcomes (PROs) and Performance outcomes (PerfOs) as primary endpoints in confirmatory studies of new drugs approve from 2011 through 2015.
METHODS: New drugs approved between January 2011 and December 2015 were identified using Drugs@ FDA database. Labeling and medical review sections from FDA DAPs were reviewed to identify indication and the primary endpoint of confirmatory studies. ICD-10 codes were used to classify disease and the primary endpoints were classified based on the type of outcome assessment. Descriptive data were recorded in Microsoft Excel; frequency of measured characteristics was analyzed.
RESULTS: Of the 182 new drugs approved the majority of the approvals were for drugs related to cancer (27.5%), anti-infective (15.9%) and endocrine, nutritional and metabolic diseases (15.4%). ClinROs and biomarkers constituted 47.8% and 40.7% of the primary endpoints respectively. PROs, survival and PerfO constituted 13.2%, 14.3% and 1.1% of the primary endpoints respectively. PROs were commonly found to be the primary endpoint in diseases related to genitourinary (80%), musculoskeletal (66.7%) and digestive systems (50%). Approvals related to cancer drugs relied on ClinROs (76%), biomarkers (14%) and survival (36%) as primary endpoints. Only six approvals (3.2%) were based on primary endpoints that were composite of PRO and another type of outcome.
CONCLUSIONS: ClinROs and biomarkers are used as primary endpoints in the majority of confirmatory studies of recent FDA approvals. PROs were key to assess treatment benefit in diseases such as those related to genitourinary, digestive and musculoskeletal systems as per respective regulatory guidance.
