Implementation of patient-reported outcome measures in real-world evidence studies: Analysis of ClinicalTrials.gov records (1999-2021)

Konrad Maruszczyk; Olalekan Lee Aiyegbusi; Victor Roth Cardoso; Georgios V Gkoutos; Karin Slater; Philip Collis; Thomas Keeley; Melanie J Calvert

doi:10.1016/j.cct.2022.106882

Implementation of patient-reported outcome measures in real-world evidence studies: Analysis of ClinicalTrials.gov records (1999-2021)

Contemp Clin Trials. 2022 Sep:120:106882. doi: 10.1016/j.cct.2022.106882. Epub 2022 Aug 13.

Authors

Konrad Maruszczyk¹, Olalekan Lee Aiyegbusi², Victor Roth Cardoso³, Georgios V Gkoutos³, Karin Slater³, Philip Collis⁴, Thomas Keeley⁵, Melanie J Calvert⁶

Affiliations

¹ Centre for Patient Reported Outcome Research, Institute of Applied Health Research, University of Birmingham, Birmingham, UK; Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK.
² Centre for Patient Reported Outcome Research, Institute of Applied Health Research, University of Birmingham, Birmingham, UK; Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK; National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre, University of Birmingham, Birmingham, UK; NIHR Surgical Reconstruction and Microbiology Research Centre, University of Birmingham, Birmingham, UK; NIHR Applied Research Collaboration (ARC) West Midlands, University of Birmingham, Birmingham, UK. Electronic address: o.l.aiyegbusi@bham.ac.uk.
³ Institute of Cancer and Genomic Sciences, University of Birmingham, Birmingham, UK.
⁴ Centre for Patient Reported Outcome Research, Institute of Applied Health Research, University of Birmingham, Birmingham, UK; NIHR Applied Research Collaboration (ARC) West Midlands, University of Birmingham, Birmingham, UK; Patient partner, UK.
⁵ Centre for Patient Reported Outcome Research, Institute of Applied Health Research, University of Birmingham, Birmingham, UK; GlaxoSmithKline (GSK), Patient Centered Outcome, Value Evidence and Outcomes, Brentford, UK.
⁶ Centre for Patient Reported Outcome Research, Institute of Applied Health Research, University of Birmingham, Birmingham, UK; Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK; National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre, University of Birmingham, Birmingham, UK; NIHR Surgical Reconstruction and Microbiology Research Centre, University of Birmingham, Birmingham, UK; NIHR Applied Research Collaboration (ARC) West Midlands, University of Birmingham, Birmingham, UK.

PMID: 35973663
DOI: 10.1016/j.cct.2022.106882

Abstract

Background: Real-world evidence (RWE) plays an increasingly important role within global regulatory and reimbursement processes. RWE generation can be enhanced by collecting and using patient-reported outcomes (PROs), which can provide valuable information on the effectiveness, safety, and tolerability of health interventions from the patient perspective. This analysis aims to examine and summarise the utilisation of patient-reported outcomes measures (PROMs) in real-world studies.

Methods: Descriptions of phase IV trials were downloaded on July 22, 2021 from the Clinicaltrials.gov database since its inception. An automated algorithm was built to detect trials utilising PROMs and composite measures including patient-reported components. Search terms were developed based on the PROQOLID database.

Results: Of 27,976 phase IV clinical trials posted on Clinicaltrials.gov between 1999 and July 2021, 21% and 4% used PROMs and composite measures, respectively. Recent years demonstrated a steady increase in the utilisation of PROMs in phase IV trials.

Conclusions: The use of PROMs in phase IV trials seems to be lower than its use in earlier phases of clinical research. Increased uptake of PROMs in RWE studies can be facilitated in a number of ways including the development of standards for their collection, analysis and use.

Keywords: Clinical trial; PRO; PROM; Phase IV; RWD; RWE.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Clinical Trials, Phase IV as Topic*
Humans
Patient Reported Outcome Measures*