PATIENT-REPORTED OUTCOMES IN UNITED STATES PROMOTIONAL COMMUNICATIONS FOR DRUGS AND BIOLOGICS: A REVIEW OF DRUG PROMOTION UNTITLED LETTERS FROM THE FDA
Author(s)
Yelizaveta Yagudayev, PharmD1, Alexander Niyazov, MPH, PharmD, JD1, Paul Cislo, PhD1, Joseph C. Cappelleri, MPH, MS, PhD2;
1Pfizer, New York, NY, USA, 2Pfizer, Newington, CT, USA
1Pfizer, New York, NY, USA, 2Pfizer, Newington, CT, USA
Presentation Documents
OBJECTIVES: The Food and Drug Administration (FDA) guidance on Patient-Reported Outcomes (PROs) provided a framework on the use of PROs to support medical product development. Currently, there are limited assessments of enforcement communications regarding promotional activities and claims regarding PROs. This study aimed to analyze attributes of PRO promotional content in FDA’s Untitled Letters (initial informal warning that serves as the FDA's first notification of non-compliant promotional materials found in ads, websites, brochures, etc.).
METHODS: A review of the Office of Prescription Drug Promotion Untitled Letters available on the FDA website from January 1, 2017 to December 31, 2025, was conducted to identify promotional communications that contained references to PROs. Common attributes of PRO content in promotional communications were reported descriptively.
RESULTS: In total, 93 Untitled Letters were reviewed. Eight Untitled Letters included promotional communications containing PROs; oncology (n=4), cardiology (n=1), dermatology (n=1), pulmonology (n=1), and endocrinology (n=1). Out of the eight letters, four were for small molecules and four were for biologics. Themes of enforcement communication included (1) claims not supported by data (n=5), (2) lack of alpha (Type 1 error) allocation (n =2), (3) infrequent collection of PRO assessments (n=2), (4) recall period bias (n=2), (5) reliance on global quality of life endpoints (n=2), (6) difficulty in interpreting time to event analyses (n=2), and (7) improper PRO instruments (n=2).
CONCLUSIONS: Our review of FDA communications found that the FDA's Office of Prescription Drug Promotion identified key findings regarding attributes of enforcement for promotional communications containing PROs. Understanding FDA’s criteria for promotional communications of PROs and adopting early planning to ensure the study design and PRO strategy satisfy the FDA criteria are warranted if PRO endpoints are to be used in promotional communications. Funding: Pfizer Inc.
METHODS: A review of the Office of Prescription Drug Promotion Untitled Letters available on the FDA website from January 1, 2017 to December 31, 2025, was conducted to identify promotional communications that contained references to PROs. Common attributes of PRO content in promotional communications were reported descriptively.
RESULTS: In total, 93 Untitled Letters were reviewed. Eight Untitled Letters included promotional communications containing PROs; oncology (n=4), cardiology (n=1), dermatology (n=1), pulmonology (n=1), and endocrinology (n=1). Out of the eight letters, four were for small molecules and four were for biologics. Themes of enforcement communication included (1) claims not supported by data (n=5), (2) lack of alpha (Type 1 error) allocation (n =2), (3) infrequent collection of PRO assessments (n=2), (4) recall period bias (n=2), (5) reliance on global quality of life endpoints (n=2), (6) difficulty in interpreting time to event analyses (n=2), and (7) improper PRO instruments (n=2).
CONCLUSIONS: Our review of FDA communications found that the FDA's Office of Prescription Drug Promotion identified key findings regarding attributes of enforcement for promotional communications containing PROs. Understanding FDA’s criteria for promotional communications of PROs and adopting early planning to ensure the study design and PRO strategy satisfy the FDA criteria are warranted if PRO endpoints are to be used in promotional communications. Funding: Pfizer Inc.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PCR79
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Oncology, STA: Multiple/Other Specialized Treatments