Presentation (CTI)

OBJECTIVES: Novel pharmacotherapies are revolutionising obesity treatment. Weight loss continues to be the primary outcome in obesity clinical trials. The Food and Drug Administration’s (FDA) recent draft guidance for industry focuses on the “effectiveness of drugs on sustained weight reduction” but also notes “fit-for-purpose COA measures could also be appropriate secondary endpoints to support a labelling claim.” The aim of this review was to examine the use of patient-reported outcome measures (PROMs) in obesity clinical trials as described in regulatory reviews and submission packages for EMA- and FDA-approved obesity management medications (OMMs).
METHODS: We identified OMM approvals by the EMA and FDA from 1998-2025. FDA drug approval packages, approved product labels, EMA assessment reports, package leaflets and product information were reviewed for PROM information.
RESULTS: Most regulatory OMM submissions included PROMs (FDA: n=6/7, EMA: n=4/6), and six PROMs were identified. The most widely used PROMs were Impact of Weight on Quality of Life (various versions) (n=8 submissions) and 36-Item Short Form Survey (n=5 submissions). The most frequently assessed concepts were quality of life (n=8), physical impacts (n=7) and emotional impacts (n=5). None of the approved OMMs included PROMs in their labels for obesity. However, one sleep PROM was included in an OMM label claim for the Obstructive Sleep Apnoea population. Nine of the 10 submissions with PROMs included regulatory discussions of PROM results: two FDA reviews noted challenges in the PRO content validity evidence, while the majority of the reviews (FDA: n=3, EMA: n=4) noted insufficient evidence for clinical significance in PROM results. Two FDA reviews included explicit recommendations on the design of future studies aiming to demonstrate improvement in functional outcomes.
CONCLUSIONS: There is continued interest in patients’ experiences of OMMs. The use of fit-for-purpose PROMs in carefully designed studies is needed to understand the clinical benefits of PROM-based endpoints.