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

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

doi:10.1016/j.cct.2022.106882

Cited by 15 publications

(11 citation statements)

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“…The ClinicalTrials.gov database was searched for records of relevant studies. The database is designed to provide easy access to summary information on publicly and privately funded clinical trials and observational studies [ 27 ]. The terms ‘healthy ageing’, ‘ageing well’, ‘ageing’, ‘anti-aging’ and ‘aging process’ were searched from inception to May 2022.…”

Section: Methodsmentioning

confidence: 99%

“…The authors thank all the participants who participated in the workshop conducted at UK SPINE annual conference 27 Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material.…”

Section: Acknowledgementsmentioning

confidence: 99%

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Optimising the selection of outcomes for healthy ageing trials: a mixed methods study

et al. 2022

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Advancing age is associated with chronic diseases which are the largest cause of death and disability in developed countries. With increasing life expectancy and an ageing population, there is a need to conduct trials to extend healthy ageing, including targeting biological ageing processes, and prevent ageing-related diseases. The main objectives of the study are as follows: (i) to review outcome measures utilised in healthy ageing trials focusing on pharmacological therapies, nutritional supplements and medical devices; (ii) to summarise the views of key stakeholders on outcome selection for healthy ageing trials. An analysis of records from the Clinicaltrials.gov database pertaining to healthy ageing trials from inception to May 2022 was conducted. In addition, the findings of a workshop attended by key stakeholders at the 2022 annual UKSPINE conference were qualitatively analysed. Substantial heterogeneity was found in the interventions evaluated and the outcomes utilised by the included studies. Recruitment of participants with diverse backgrounds and the confounding effects of multi-morbidity in older adults were identified as the main challenges of measuring outcomes in healthy ageing trials by the workshop participants. The development of a core outcome set for healthy ageing trials can aid comparability across interventions and within different settings. The workshop provided an important platform to garner a range of perspectives on the challenges with measuring outcomes in this setting. It is critical to initiate such discussions to progress this field and provide practical answers to how healthy ageing trials are designed and structured in the future.

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Section: Methodsmentioning

confidence: 99%

Section: Acknowledgementsmentioning

confidence: 99%

Optimising the selection of outcomes for healthy ageing trials: a mixed methods study

et al. 2022

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“…Efforts have been made to standardize RWD/RWE for some time. In guiding researchers to design and implement RWD research, some standards have been put forth, such as guidelines, 7,8 processes, [9][10][11] and templates. 12,13 Although RWSs differ greatly from traditional clinical trials in terms of research characteristics, researchers still adopt the habit of writing traditional randomized controlled trial (RCT) research protocols.…”

Section: Introductionmentioning

confidence: 99%

Worldwide Trends in Registering Real-World Studies at ClinicalTrials.gov: A Cross-Sectional Analysis

Li¹,

Tian²,

Pei³

et al. 2023

IJGM

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The purpose of this study was to characterize real-world studies (RWSs) registered at ClinicalTrials.gov to help investigators better conduct relevant research in clinical practice. Methods: A retrospective analysis of 944 studies was performed on February 28, 2023. Results: A total of 944 studies were included. The included studies involved a total of 48 countries. China was the leading country in terms of the total number of registered studies (37.9%, 358), followed by the United States (19.7%, 186). Regarding intervention type, 42.4% (400) of the studies involved drugs, and only 9.1% (86) of the studies involved devices. Only 8.5% (80) of the studies mentioned both the detailed study design type and data source in the "Brief Summary". A total of 49.4% (466) of studies had a sample size of 500 participants and above. Overall, 63% (595) of the studies were single-center studies. A total of 213 conditions were covered in the included studies. One-third of the studies (32.7%, 309) involved neoplasms (or tumors). China and the United States were very different regarding the study of different conditions. Conclusion:Although the pandemic has provided new opportunities for RWSs, the rigor of scientific research still needs to be emphasized. Special attention needs to be given to the correct and comprehensive description of the study design in the Brief Summary of registered studies, thereby promoting communication and understanding. In addition, deficiencies in ClinicalTrials.gov registration data remain prominent.

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“…This has changed the data landscape, with growing recognition of the value of using real-world evidence that is applicable for regulatory and reimbursement purposes. Frameworks from regulatory bodies, such as the United States Food and Drug Administration (FDA) and European Medicines Agency (EMA), and reimbursement agencies, such as the and patient perspectives [9][10][11]. Although outside of the scope of this viewpoint, data from historical clinical trials have been found to be influential in comparative efficacy analyses and have been found to have a role in supplementing an external control study through hybrid study designs [12,13].…”

Section: Introductionmentioning

confidence: 99%

“…Real-world data are data related to patient health status or delivery of health care outside of RCTs where sources commonly originate from electronic health records (EHRs), medical claims data, and product and disease registries [ 8 ]. In addition to what may be considered common forms, real-world data from outside of traditional medical charting, including data from mobile phones, wearables, and patient-reported outcomes, have provided an abundance of data, allowing comprehensive capture of the natural course of a disease from both the physician and patient perspectives [ 9 - 11 ]. Although outside of the scope of this viewpoint, data from historical clinical trials have been found to be influential in comparative efficacy analyses and have been found to have a role in supplementing an external control study through hybrid study designs [ 12 , 13 ].…”

Section: Introductionmentioning

confidence: 99%

Data Challenges for Externally Controlled Trials: Viewpoint

Velummailum¹,

McKibbon²,

Brenner³

et al. 2023

J Med Internet Res

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The preferred evidence of a large randomized controlled trial is difficult to adopt in scenarios, such as rare conditions or clinical subgroups with high unmet needs, and evidence from external sources, including real-world data, is being increasingly considered by decision makers. Real-world data originate from many sources, and identifying suitable real-world data that can be used to contextualize a single-arm trial, as an external control arm, has several challenges. In this viewpoint article, we provide an overview of the technical challenges raised by regulatory and health reimbursement agencies when evaluating comparative efficacy, such as identification, outcome, and time selection challenges. By breaking down these challenges, we provide practical solutions for researchers to consider through the approaches of detailed planning, collection, and record linkage to analyze external data for comparative efficacy.

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

Cited by 15 publications

References 10 publications

Optimising the selection of outcomes for healthy ageing trials: a mixed methods study

Optimising the selection of outcomes for healthy ageing trials: a mixed methods study

Worldwide Trends in Registering Real-World Studies at ClinicalTrials.gov: A Cross-Sectional Analysis

Data Challenges for Externally Controlled Trials: Viewpoint

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