PMS25 Patient-Reported Outcome Labeling for Rheumatoid Arthritis Drugs Approved in the United States: 2010-2019

Abstract

Patient-reported outcomes (PROs), defined as any report on the status of a patient’s health condition that comes directly from the patient, have played an increasingly important role in drug development. In 2009 the Food and Drug Administration (FDA) issued final guidance on using PRO measures to support labeling claims, as a means of incorporating the patient perspective into the drug approval process. For rheumatoid arthritis (RA), a chronic inflammatory disease associated with physical symptoms like pain and stiffness, but also changes to functional capacity and quality of life (QOL), PROs are critical to demonstrate the treatment benefit of new drugs. This analysis evaluates the incorporation of PROs into labeling for recent primary approvals of RA drugs. The FDA’s novel drug approvals (2010-2019) were reviewed to identify RA drugs. Drug approval packages and product labeling were accessed via the [email protected] database and analyzed for PRO endpoints, measures, and labeling claims. Clinical trial designs and published study results were retrieved via the ClinicalTrials.gov website and PubMed. Five novel drugs received primary approval for RA indications between 2010 and 2019. Trials supporting approval of all 5 products assessed patients’ perception of their disease activity, functional capacity, QOL, pain and fatigue. Three of the 5 products (60%) evaluated patients’ work limitations/productivity during pivotal trials, while 1 of the 5 (20%) evaluated quality of sleep. All 5 products incorporated PROs in their labeling, with more recent approvals having a greater number of PRO claims. All RA drugs approved in the past decade used PRO assessments during development, and all acquired labeling claims. Our findings underscore the significant impact RA symptoms have on patients’ day-to-day functioning and QOL, the importance of PROs in RA drug development, and the need to standardize functional domains and validated instruments used in registrational trials.