REVIEW OF CLINICAL OUTCOME ASSESSMENTS (COA) IN DRUG APPROVALS 2024-25, INSIGHTS FROM FDA AND EMA LABELLING
Author(s)
Isabelle Savre, PhD1, Benoit Arnould, PhD2, Bistra Bonnefon, MBA1, Wiola Racino, Msc1, John Kealy, PhD3, Pujita Vaidya, MPH4, Ella Brookes, BSc, MSc5.
1Mapi Research Trust, LYON, France, 2Sanofi PID-HVT, Lyon, France, 3Mapi Research Trust, LYON, Ireland, 4Sanofi, Alexandria, VA, USA, 5Clinical Outcome Assessment Scientist, Sanofi, Reading, United Kingdom.
1Mapi Research Trust, LYON, France, 2Sanofi PID-HVT, Lyon, France, 3Mapi Research Trust, LYON, Ireland, 4Sanofi, Alexandria, VA, USA, 5Clinical Outcome Assessment Scientist, Sanofi, Reading, United Kingdom.
OBJECTIVES: Clinical Outcome assessments (COAs) in product labelling demonstrate meaningful improvements in patient relevant concepts and ensure treatments address unmet needs. Previously conducted reviews of FDA and EMA approvals (2000-23), showed consistent increase in the number of COAs reaching the label over time. A review of product approvals 2018-23 identified 40% included COAs in the label (Savre et al. 2025). This review aimed to further investigate this trend including recent approvals (2024-25).
METHODS: A search of newly approved drug products between 2024-25 was conducted using FDA and EMA websites. PROLABELS database identified products with COAs mentioned in their labels. Among 324 newly approved products (FDA:217; EMA:107), 148 mentioned COAs in their labels.
RESULTS: Among the 324 approvals, 45% of FDA products (97) and 48% of EMA products (51) included a COA in the label, an increase from previous years. PRO label representation was slightly higher for EMA (28%) than FDA (22%), consistent with previous reviews. PROs were the most prevalent COA overall (78 products, 53%; FDA: 48, 49%, EMA: 30, 59%), followed by ClinROs (54 products, 36%; FDA: 34, 35%, EMA: 20, 39%), Composite measures (53 products, 36%; FDA: 35, 36%, EMA: 18, 35%), PerfOs (18 products, 12%; FDA: 10, 10%, EMA: 8, 16%), and ObsROs (10 products, 7%; FDA: 5, 5%, EMA: 5, 10%). COAs measuring signs/symptoms were most frequent across both agencies (122 products; FDA: 77, EMA: 45), most frequently as primary (40%) or secondary (38%) endpoint. HRQoL-specific COAs were least represented (14 products), exclusively PRO and predominantly secondary endpoints (74%) with notably higher endorsement from EMA (EMA: 12, FDA: 2).
CONCLUSIONS: A continued emphasis on COA in regulatory approvals was identified, with measurement of signs/symptoms dominating regulatory focus. Measurement of impacts and HRQoL remains underrepresented, particularly in FDA approvals, highlighting a divergence between regulators in their approach to patient-centred evidence.
METHODS: A search of newly approved drug products between 2024-25 was conducted using FDA and EMA websites. PROLABELS database identified products with COAs mentioned in their labels. Among 324 newly approved products (FDA:217; EMA:107), 148 mentioned COAs in their labels.
RESULTS: Among the 324 approvals, 45% of FDA products (97) and 48% of EMA products (51) included a COA in the label, an increase from previous years. PRO label representation was slightly higher for EMA (28%) than FDA (22%), consistent with previous reviews. PROs were the most prevalent COA overall (78 products, 53%; FDA: 48, 49%, EMA: 30, 59%), followed by ClinROs (54 products, 36%; FDA: 34, 35%, EMA: 20, 39%), Composite measures (53 products, 36%; FDA: 35, 36%, EMA: 18, 35%), PerfOs (18 products, 12%; FDA: 10, 10%, EMA: 8, 16%), and ObsROs (10 products, 7%; FDA: 5, 5%, EMA: 5, 10%). COAs measuring signs/symptoms were most frequent across both agencies (122 products; FDA: 77, EMA: 45), most frequently as primary (40%) or secondary (38%) endpoint. HRQoL-specific COAs were least represented (14 products), exclusively PRO and predominantly secondary endpoints (74%) with notably higher endorsement from EMA (EMA: 12, FDA: 2).
CONCLUSIONS: A continued emphasis on COA in regulatory approvals was identified, with measurement of signs/symptoms dominating regulatory focus. Measurement of impacts and HRQoL remains underrepresented, particularly in FDA approvals, highlighting a divergence between regulators in their approach to patient-centred evidence.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
CO150
Topic
Clinical Outcomes, Health Policy & Regulatory, Patient-Centered Research
Topic Subcategory
Clinical Outcomes Assessment