Evaluating the Role of Patient-Reported Outcomes in Phase 4 Clinical Trials: Trends, Applications, and Implications for Postmarketing Surveillance
Author(s)
Heather Wellam, BSc, MSc1, Clare Foy, BSc, MSc1, Kimrin Kaur Pannu, BSc, MSc1, Catherine Beecher, .2.
1Petauri Evidence, Oxfordshire, United Kingdom, 2Petauri Evidence, Bicester, United Kingdom.
1Petauri Evidence, Oxfordshire, United Kingdom, 2Petauri Evidence, Bicester, United Kingdom.
OBJECTIVES: Explore trends in the adoption of patient-reported outcomes (PROs) in Phase 4 clinical trials.
METHODS: A search of ClinicalTrials.gov identified Phase 4 clinical trials registered between 2020-2024. Start date, condition, intervention, and primary outcome were quantitively analysed.
RESULTS: Between 2020-2024, 17% (1,179 of 6,981) of Phase 4 clinical trials incorporated a PRO as the primary endpoint. The annual inclusion of PROs ranged from 18-22% of trials registered over the 5-year period: 2020 (218 trials, 18%), 2021 (242 trials, 21%), 2022 (254 trials, 22%), 2023 (243 trials, 21%), and 2024 (222 trials, 19%). PROs were applied to different interventions including drug (1,018 trials, 86%), device (55 trials, 5%), procedure (41 trials, 3%), and other (65 trials, 5%). PROs were applied to conditions affecting body systems including nervous (26%), musculoskeletal (18%), mental health (15%), reproductive and excretory (10%), integumentary (9%), digestive (7%), respiratory (5%), cardiovascular (3%), endocrine (3%), and others (4%). PRO domains included symptoms (77%), function (7%), behaviour (6%), experience (6%), and quality of life (4%).
CONCLUSIONS: Integration of PROs in clinical trials remained stable between 2020-2024. The predominant application was the assessment of drugs, while use in device and procedural studies remained limited. Conditions affecting the nervous system, musculoskeletal system, and mental health represented the largest categories utilising PROs due to the subjective nature of symptoms, including pain, mobility, and mood, and the difficulty in quantifying their impact through clinical measures. This research demonstrates that PROs are valuable in demonstrating patient-centred benefits in a real-world setting across a range of therapeutic areas. Future research could explore how PRO data contribute to long-term surveillance, support comparative analysis of treatments, and inform market access, such as pricing, and coverage decisions by demonstrating the real-world impact of therapies from the patient perspective.
METHODS: A search of ClinicalTrials.gov identified Phase 4 clinical trials registered between 2020-2024. Start date, condition, intervention, and primary outcome were quantitively analysed.
RESULTS: Between 2020-2024, 17% (1,179 of 6,981) of Phase 4 clinical trials incorporated a PRO as the primary endpoint. The annual inclusion of PROs ranged from 18-22% of trials registered over the 5-year period: 2020 (218 trials, 18%), 2021 (242 trials, 21%), 2022 (254 trials, 22%), 2023 (243 trials, 21%), and 2024 (222 trials, 19%). PROs were applied to different interventions including drug (1,018 trials, 86%), device (55 trials, 5%), procedure (41 trials, 3%), and other (65 trials, 5%). PROs were applied to conditions affecting body systems including nervous (26%), musculoskeletal (18%), mental health (15%), reproductive and excretory (10%), integumentary (9%), digestive (7%), respiratory (5%), cardiovascular (3%), endocrine (3%), and others (4%). PRO domains included symptoms (77%), function (7%), behaviour (6%), experience (6%), and quality of life (4%).
CONCLUSIONS: Integration of PROs in clinical trials remained stable between 2020-2024. The predominant application was the assessment of drugs, while use in device and procedural studies remained limited. Conditions affecting the nervous system, musculoskeletal system, and mental health represented the largest categories utilising PROs due to the subjective nature of symptoms, including pain, mobility, and mood, and the difficulty in quantifying their impact through clinical measures. This research demonstrates that PROs are valuable in demonstrating patient-centred benefits in a real-world setting across a range of therapeutic areas. Future research could explore how PRO data contribute to long-term surveillance, support comparative analysis of treatments, and inform market access, such as pricing, and coverage decisions by demonstrating the real-world impact of therapies from the patient perspective.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR84
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas