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 Gkoutos; Luke Slater; Philip  Collis; Thomas Keeley; Melanie 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)

Konrad Maruszczyk, Olalekan Lee Aiyegbusi, Victor Roth Cardoso, Georgios Gkoutos, Luke Slater, Philip Collis, Thomas Keeley, Melanie Calvert

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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.

Original language	English
Article number	106882
Number of pages	7
Journal	Contemporary Clinical Trials
Volume	120
Early online date	13 Aug 2022
DOIs	https://doi.org/10.1016/j.cct.2022.106882
Publication status	Published - 23 Aug 2022

Bibliographical note

Funding Information:
OLA receives funding from the NIHR Birmingham Biomedical Research Centre (BRC), NIHR Applied Research Collaboration (ARC) West Midlands at the University of Birmingham and University Hospitals Birmingham NHS Foundation, Innovate UK (part of UK Research and Innovation), Gilead Sciences Ltd, Janssen Pharmaceuticals, Inc., and Sarcoma UK. OLA declares personal fees from Gilead Sciences Ltd, GSK and Merck outside the submitted work.

Funding Information:
MC is Director of the Birmingham Health Partners Centre for Regulatory Science and Innovation, Director of the Centre for Patient-Reported Outcomes Research and is a National Institute for Health and Care Research (NIHR) Senior Investigator. She receives funding from the NIHR Birmingham Biomedical Research Centre, the NIHR Surgical Reconstruction and Microbiology Research Centre and NIHR Applied Research Collaboration (ARC) West Midlands at the University of Birmingham and University Hospitals Birmingham NHS Foundation Trust, Health Data Research UK, Innovate UK (part of UK Research and Innovation), Macmillan Cancer Support, UCB and GSK. MC has received personal fees from Astellas, Takeda, Merck, Daiichi Sankyo, Glaukos, CIS Oncology, Aparito Ltd, GSK, Genentech and the Patient-Centered Outcomes Research Institute (PCORI) outside the submitted work.

Funding Information:
This research was conducted as part of a PhD programme funded by GSK . TK is a co-supervisor of KM (the holder of the GSK PhD grant) and is a Director at GSK Ltd. In his role as co-supervisor TK inputted to all stages of this research.

Funding Information:
This research was conducted as part of a PhD programme funded by GSK. TK is a co-supervisor of KM (the holder of the GSK PhD grant) and is a Director at GSK Ltd. In his role as co-supervisor TK inputted to all stages of this research.

Publisher Copyright:
© 2022 The Authors

Keywords

Clinical trial
RWE
PRO
PROM
Phase IV
RWD

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Maruszczyk, K., Aiyegbusi, O. L., Roth Cardoso, V., Gkoutos, G., Slater, L., Collis, P., Keeley, T., & Calvert, M. (2022). Implementation of patient-reported outcome measures in real-world evidence studies: analysis of ClinicalTrials.gov records (1999-2021). Contemporary Clinical Trials, 120, Article 106882. Advance online publication. https://doi.org/10.1016/j.cct.2022.106882

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title = "Implementation of patient-reported outcome measures in real-world evidence studies: analysis of ClinicalTrials.gov records (1999-2021)",

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, RWE, PRO, PROM, Phase IV, RWD",

author = "Konrad Maruszczyk and Aiyegbusi, {Olalekan Lee} and {Roth Cardoso}, Victor and Georgios Gkoutos and Luke Slater and Philip Collis and Thomas Keeley and Melanie Calvert",

note = "Funding Information: OLA receives funding from the NIHR Birmingham Biomedical Research Centre (BRC), NIHR Applied Research Collaboration (ARC) West Midlands at the University of Birmingham and University Hospitals Birmingham NHS Foundation, Innovate UK (part of UK Research and Innovation), Gilead Sciences Ltd, Janssen Pharmaceuticals, Inc., and Sarcoma UK. OLA declares personal fees from Gilead Sciences Ltd, GSK and Merck outside the submitted work. Funding Information: MC is Director of the Birmingham Health Partners Centre for Regulatory Science and Innovation, Director of the Centre for Patient-Reported Outcomes Research and is a National Institute for Health and Care Research (NIHR) Senior Investigator. She receives funding from the NIHR Birmingham Biomedical Research Centre, the NIHR Surgical Reconstruction and Microbiology Research Centre and NIHR Applied Research Collaboration (ARC) West Midlands at the University of Birmingham and University Hospitals Birmingham NHS Foundation Trust, Health Data Research UK, Innovate UK (part of UK Research and Innovation), Macmillan Cancer Support, UCB and GSK. MC has received personal fees from Astellas, Takeda, Merck, Daiichi Sankyo, Glaukos, CIS Oncology, Aparito Ltd, GSK, Genentech and the Patient-Centered Outcomes Research Institute (PCORI) outside the submitted work. Funding Information: This research was conducted as part of a PhD programme funded by GSK . TK is a co-supervisor of KM (the holder of the GSK PhD grant) and is a Director at GSK Ltd. In his role as co-supervisor TK inputted to all stages of this research. Funding Information: This research was conducted as part of a PhD programme funded by GSK. TK is a co-supervisor of KM (the holder of the GSK PhD grant) and is a Director at GSK Ltd. In his role as co-supervisor TK inputted to all stages of this research. Publisher Copyright: {\textcopyright} 2022 The Authors",

year = "2022",

month = aug,

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doi = "10.1016/j.cct.2022.106882",

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T1 - Implementation of patient-reported outcome measures in real-world evidence studies

T2 - analysis of ClinicalTrials.gov records (1999-2021)

AU - Maruszczyk, Konrad

AU - Aiyegbusi, Olalekan Lee

AU - Roth Cardoso, Victor

AU - Gkoutos, Georgios

AU - Slater, Luke

AU - Collis, Philip

AU - Keeley, Thomas

AU - Calvert, Melanie

N1 - Funding Information: OLA receives funding from the NIHR Birmingham Biomedical Research Centre (BRC), NIHR Applied Research Collaboration (ARC) West Midlands at the University of Birmingham and University Hospitals Birmingham NHS Foundation, Innovate UK (part of UK Research and Innovation), Gilead Sciences Ltd, Janssen Pharmaceuticals, Inc., and Sarcoma UK. OLA declares personal fees from Gilead Sciences Ltd, GSK and Merck outside the submitted work. Funding Information: MC is Director of the Birmingham Health Partners Centre for Regulatory Science and Innovation, Director of the Centre for Patient-Reported Outcomes Research and is a National Institute for Health and Care Research (NIHR) Senior Investigator. She receives funding from the NIHR Birmingham Biomedical Research Centre, the NIHR Surgical Reconstruction and Microbiology Research Centre and NIHR Applied Research Collaboration (ARC) West Midlands at the University of Birmingham and University Hospitals Birmingham NHS Foundation Trust, Health Data Research UK, Innovate UK (part of UK Research and Innovation), Macmillan Cancer Support, UCB and GSK. MC has received personal fees from Astellas, Takeda, Merck, Daiichi Sankyo, Glaukos, CIS Oncology, Aparito Ltd, GSK, Genentech and the Patient-Centered Outcomes Research Institute (PCORI) outside the submitted work. Funding Information: This research was conducted as part of a PhD programme funded by GSK . TK is a co-supervisor of KM (the holder of the GSK PhD grant) and is a Director at GSK Ltd. In his role as co-supervisor TK inputted to all stages of this research. Funding Information: This research was conducted as part of a PhD programme funded by GSK. TK is a co-supervisor of KM (the holder of the GSK PhD grant) and is a Director at GSK Ltd. In his role as co-supervisor TK inputted to all stages of this research. Publisher Copyright: © 2022 The Authors

PY - 2022/8/23

Y1 - 2022/8/23

N2 - 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.

AB - 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.

KW - Clinical trial

KW - RWE

KW - PRO

KW - PROM

KW - Phase IV

KW - RWD

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DO - 10.1016/j.cct.2022.106882

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SN - 1551-7144

VL - 120

JO - Contemporary Clinical Trials

JF - Contemporary Clinical Trials

M1 - 106882

ER -

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

Abstract

Bibliographical note

Keywords

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Cite this