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Harnessing Real-World Evidence to Advance Cancer Research
Abstract
Randomized controlled trials (RCTs) form a cornerstone of oncology research by generating evidence about the efficacy of therapies in selected patient populations. However, their implementation is often resource- and cost-intensive, and their generalisability to patients treated in routine practice may be limited. Real-world evidence leverages data collected about patients receiving clinical care in routine practice outside of clinical trial settings and provides opportunities to identify and address gaps in clinical trial evidence. This review outlines the strengths and limitations of real-world and RCT evidence and proposes a framework for the complementary use of the two bodies of evidence to advance cancer research. There are challenges to the implementation of real-world research in oncology, including heterogeneity of data sources, timely access to high-quality data, and concerns about the quality of methods leveraging real-world data, particularly causal inference. Improved understanding of the strengths and limitations of real-world data and ongoing efforts to optimise the conduct of real-world evidence research will improve its reliability, understanding and acceptance, and enable the full potential of real-world evidence to be realised in oncology practice.
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The study findings of observational studies are susceptible to confounding and selection biases, which could result in biased estimates of intervention effects compared with smaller RCTs.
“Thus, the study findings are susceptible to confounding and selection biases, which could result in biased estimates of intervention effects compared with smaller RCTs [5,6].”
From RoBANS 2: A Revised Risk of Bias Assessment Tool for Nonrandomized Studies of Interventions (2023) · cites this paper as [5]
A crucial limitation of observational studies on intervention effects and adverse reactions is that the intervention of interest is not randomly assigned, blinding is lacking, and there is often no comparison.
“However, a crucial limitation of observational studies on intervention effects and adverse reactions is that the intervention of interest is not randomly assigned, blinding is lacking, and there is often no comparison.”
From RoBANS 2: A Revised Risk of Bias Assessment Tool for Nonrandomized Studies of Interventions (2023) · cites this paper as [5]
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Benchimol, E. I., Smeeth, L., Guttmann, A., Harron, K., Moher, D., Petersen, I., Sørensen, H. T., von Elm, E., Langan, S. M., & RECORD Working Committee (2015) · PLOS Medicine · doi:10.1371/journal.pmed.1001885 · Cited by 5,749
Ford, I., & Norrie, J. (2016) · New England Journal of Medicine · doi:10.1056/nejmra1510059 · Cited by 1,405
Understanding controlled trials: Why are randomised controlled trials important?
Sibbald, B., & Roland, M. (1998) · BMJ · doi:10.1136/bmj.316.7126.201 · Cited by 807
When and how can real world data analyses substitute for randomized controlled trials?
Franklin, J. M., & Schneeweiss, S. (2017) · Clinical Pharmacology & Therapeutics · doi:10.1002/cpt.857 · Cited by 316
Strom, J. B., Faridi, K. F., Butala, N. M., Zhao, Y., Tamez, H., Valsdottir, L. R., Brennan, J. M., Shen, C., Popma, J. J., Kazi, D. S., et al. (2020) · Circulation · doi:10.1161/circulationaha.120.046159 · Cited by 37
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Sorted by citations
RoBANS 2: A Revised Risk of Bias Assessment Tool for Nonrandomized Studies of Interventions
Seo, H.-J., Kim, S. Y., Lee, Y. J., & Park, J.-E. (2023) · Korean Journal of Family Medicine · doi:10.4082/kjfm.23.0034 · Cited by 128
Metadata
- Authors
- Tang, M., Pearson, S.-A., Simes, R. J., & Chua, B. H.
- Venue
- Current Oncology
- Publisher
- MDPI AG
- Published
- 2023-02-02
- Volume
- 30
- Issue
- 2
- Pages
- 1844-1859
- ISSN
- 1718-7729
- PMC
- PMC9955401
- Cited by
- 88
- References
- 5
- Language
- en
- Funders
- National Health and Medical Research Council (1151479, DS-18-01)
- National Breast Cancer Foundation (1151479, DS-18-01)
- Translational Cancer Research Network (1151479, DS-18-01)
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