Patient-Reported Outcomes: Observations of Regulatory Approvals by the Center for Biologics Evaluation and Research

• Activity Number: 113 - Clinical Outcome Assessments: Measurement, Evaluation, and Interpretation
• Type: Topic Contributed
• Date/Time: Monday, July 31, 2017 : 8:30 AM to 10:20 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Hussein Ezzeldin* and Megan Moncur and Yuqun Abigail Luo and Telba Irony
• Companies: FDA/CBER and FDA/CBER and FDA/CBER and Office of Biostatistics and Epidemiology, CBER, FDA
• Keywords: patient-reported outcomes ; quality-of-life ; Biologics License Application

Abstract

Objective: Present past experience and the future prospects of the Center for Biologics Evaluation and Research (CBER) with Patient-Reported Outcomes (PROs). Examine the history of PROs in CBER and their role in establishing primary and secondary endpoints. Methods: All biologics License Applications (BLAs) approved by CBER with a PRO were identified from the FDA website [1] for the period 1998 - 2016. For each approved BLA with a PRO, we reviewed the publicly available clinical and statistical reviews and the package insert to characterize the endpoints, PRO instruments used, and claims. Results: Of the 296 approved BLAs, 79 (26.7%) included clinical studies, of which 21 (26.6%) included PROs for primary or secondary endpoint. Conclusion: Progress has been made in the utilization of PROs, from using simple scales to complex instruments. Despite the few PRO-supported labeling claims to date, we have seen in recent years (2007-2016) an increase to 32.7% after the issuance of the 2006 draft PRO guidance, compared to 12.5% prior to 2006.

1.http://www.fda.gov/BiologicsBloodVaccines/DevelopmentApprovalProcess/BiologicalApprovalsbyYear/default.htm