Clinical Outcome Assessments in Drug Approvals: Trends and Insights From FDA; EMA Labeling 2018-2023
Author(s)
Isabelle Savre, PhD1, Petar Markov, PhD1, Bistra Bonnefon, MBA1, Sandra Desruol-Allardin, MSc1, John Kealy, PhD1, Pujita Vaidya, MPH2, Julie Guery, PhD3, Amaya Clemente, PharmD4, Sonia Bothorel, MBA1, Benoit Arnould, PhD5.
1Mapi Research Trust, Lyon, France, 2SANOFI, Alexandria, VA, USA, 3SANOFI, Morristown, NJ, USA, 4SANOFI, Barcelona, Spain, 5SANOFI, Lyon, France.
1Mapi Research Trust, Lyon, France, 2SANOFI, Alexandria, VA, USA, 3SANOFI, Morristown, NJ, USA, 4SANOFI, Barcelona, Spain, 5SANOFI, Lyon, France.
OBJECTIVES: Clinical outcome assessments (COAs) in drug labeling play a crucial role in regulatory decisions influencing how therapies are perceived by healthcare providers and patients. They help ensure treatments address patient priorities and improve their quality of life. A 2013-2017 review of FDA and EMA drug approvals showed that around 40% of EMA and FDA products have COA in their label. This study explores the trends for newly approved drug products between 2018 and 2023.
METHODS: FDA and EMA websites were systematically reviewed to identify new drug approvals (2018-2023). We collected information on COAs included in drug labeling using the PROLABELSTM database and by reviewing these regulatory websites.
RESULTS: Between 2018 and 2023, 711 and 342 new drug approvals were granted by the FDA and the EMA, respectively, and remained on the market. COAs appeared in 44% of FDA (n=312) and 53% of EMA approvals (n=180). Among these, PRO was the most common COA type, present in 46% of FDA (n=142) and in 62% of EMA (n=112) approvals with COA in the labels, followed by ClinROs (42% vs 41%), Composite (31% vs 48%), PerfOs (13% vs 15%), and ObsROs (8% vs 9%). Most frequently assessed concepts included signs/symptoms (FDA n=263, EMA n=142), and less frequently treatment response/remission (FDA n=64, EMA n=37), functional status (FDA n=53, EMA n=47), and quality of life/health status (FDA n=17, EMA=53). The PROs primarily relied on validated instruments (n=147), and secondarily on study-specific scales (n=71).
CONCLUSIONS: Regulatory emphasis on patient-centered research for demonstrating clinical benefit and drug approval is growing. Our results showed that while PROs are the most common COA type retrieved, overall, COA-labeled drugs still accounted for only half of EMA approvals and fewer than half of FDA approvals. This highlights the need for more education on COA use to better reflect patient experience in clinical trials.
METHODS: FDA and EMA websites were systematically reviewed to identify new drug approvals (2018-2023). We collected information on COAs included in drug labeling using the PROLABELSTM database and by reviewing these regulatory websites.
RESULTS: Between 2018 and 2023, 711 and 342 new drug approvals were granted by the FDA and the EMA, respectively, and remained on the market. COAs appeared in 44% of FDA (n=312) and 53% of EMA approvals (n=180). Among these, PRO was the most common COA type, present in 46% of FDA (n=142) and in 62% of EMA (n=112) approvals with COA in the labels, followed by ClinROs (42% vs 41%), Composite (31% vs 48%), PerfOs (13% vs 15%), and ObsROs (8% vs 9%). Most frequently assessed concepts included signs/symptoms (FDA n=263, EMA n=142), and less frequently treatment response/remission (FDA n=64, EMA n=37), functional status (FDA n=53, EMA n=47), and quality of life/health status (FDA n=17, EMA=53). The PROs primarily relied on validated instruments (n=147), and secondarily on study-specific scales (n=71).
CONCLUSIONS: Regulatory emphasis on patient-centered research for demonstrating clinical benefit and drug approval is growing. Our results showed that while PROs are the most common COA type retrieved, overall, COA-labeled drugs still accounted for only half of EMA approvals and fewer than half of FDA approvals. This highlights the need for more education on COA use to better reflect patient experience in clinical trials.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HPR45
Topic
Clinical Outcomes, Health Policy & Regulatory, Patient-Centered Research
Topic Subcategory
Approval & Labeling
Disease
No Additional Disease & Conditions/Specialized Treatment Areas