Patient-Reported Outcomes Trends in Clinical Trials: Insights from 2008-2023
Patient-reported outcomes trends show a clear surge in clinical trials from 2008 to 2023, where usage has doubled in interventional studies, while tools like EQ-5D lead as the top generic instrument, with PROMIS seeing explosive growth that reflects rising demands from regulators and health technology assessments (HTAs). These shifts emphasize patient-centered research for adults and children, ultimately boosting focus on health-related quality of life (HRQL) data.
Executive Summary: 25% of Trials Now Use PROs
Patient-reported outcome measures in clinical trials rose sharply from 2008 to 2023, reaching nearly one-quarter of adult trials by 2023, up from 12.7% in 2008, according to this study that draws from ClinicalTrials.gov data and shows EQ-5D surpassing SF-36 variants as the main generic tool since 2017. PROMIS grew at a 51.9% compound annual growth rate (CAGR) and pediatric use climbed from 7.9% to 17.7% of trials, highlighting a stronger push for patient views on well-being.
5 Key Findings
Patient-reported outcomes trends reveal key patterns in HRQL assessments and patient-focused endpoints, as the study analyzed 475,844 interventional trials to uncover what stands out:
- Rapid Overall Growth: 18.6% of trials included at least one PRO, with adult studies nearly doubling their PRO use and pediatric ones more than doubling, showing more attention to personal health experiences.
- EQ-5D’s Dominance: This tool covers areas like mobility and anxiety, growing at 16.2% CAGR to appear in 8,142 trials by 2023, when it overtook SF-36/12/8/6D (8,872 trials at 7.0% CAGR) around 2017, with its value lying in economic evaluations.
- PROMIS’s Surge: From one trial in 2008, PROMIS hit 524 uses by 2023, including profiles like PROMIS-29 and PROPr scoring, where adaptive testing and flexible domains drive its rise for chronic conditions.
- Condition-Specific Trends: In oncology, EORTC QLQ-C30/QLU-C10D appeared in 4,270 trials (16.4% CAGR), FACIT showed 3,063 trials (8.8% CAGR), and HUI2/HUI3 stayed flat despite some HTA support.
- Pediatric Highlights: PedsQL topped with 1,055 trials (15.9% CAGR), EQ-5D-Y followed at 647 trials (17.9% CAGR), PROMIS Pediatric reached 115 trials (22.3% CAGR), though use still trails adults.
Methodology: 96% Accuracy via ClinicalTrials.gov API
Patient-reported outcomes (PROs) are self-assessments of symptoms, function, and well-being that evaluate treatments from the patient’s angle, and this study by Maja Kuharic and colleagues at Northwestern University and the University of Illinois Chicago extends prior work up to 2013 by tracking changes to 2023 amid FDA and EMA updates.
The approach used ClinicalTrials.gov, a U.S. National Library of Medicine database that covers Phase 1-3 trials registered after 2008 FDA rules, where researchers listed common PROs from PROQOLID (over 7,000 tools), excluded clinician reports, and added HTA picks like EQ-5D (five HRQL domains plus VAS), SF family surveys, PROMIS (NIH system with 300+ items for physical and mental health), and specifics like EORTC QLQ-C30, FACIT, PedsQL, and CHU9D.
A Python algorithm queried the API with 96% sensitivity and 97% specificity, using Boolean searches and pattern-matching for variants like plurals or abbreviations, including general terms like “patient-reported outcome,” while manual checks on 200 sample trials boosted accuracy. Data split by age (pediatric: birth-17; adult: 18+), region (e.g., Europe, North America), and disease (via NIH MeSH, such as neoplasms), with growth analyzed via CAGR and annual changes in Python, Excel, and SAS to provide solid evidence of PRO expansion.
Dive deeper into these PRO families and trial uses in this detailed ScienceDirect analysis.
Real-World Impact: HTAs, Approvals, Reimbursements
Patient-reported outcomes trends shape health economics and outcomes research, as their doubling boosts patient input in cost-effectiveness, approvals, and value-based care, with EQ-5D’s growth—backed by NICE—providing utility scores for cost-utility models that standardize evaluations in Europe and support global pricing.
PROMIS’s fast rise offers strong, adaptable domains that track chronic burdens well, yet regional gaps—like lower use in Asia—call for cross-cultural checks to cut biases, while in pediatrics, 17.7% adoption by 2023 closes adult-child gaps, sways policies like the Orphan Drug Act for rare diseases, and may align HTA rules next to promote fair PRO use. Patient data can then guide reimbursements and guidelines, improving decisions.
FAQ
What drives the growth in patient-reported outcomes trends over 2008-2023?
Regulatory pushes from FDA and EMA, plus HTA needs, doubled PRO use by prioritizing patient-centered data in trials for better real-world insights.
How do patient-reported outcomes trends differ between adults and pediatrics?
Adult trials hit 25% PRO use by 2023, up from 12.7%, while pediatrics reached 17.7% from 7.9%, with tools like PedsQL growing fast but facing standardization hurdles.
What role does EQ-5D play in patient-reported outcomes trends for economic evaluations?
EQ-5D leads generics with 16.2% CAGR, as its domains enable utility scoring that’s vital for cost-utility analyses in HTAs like NICE, influencing pricing and approvals.
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