Application of Real-World Data to External Control Groups in Oncology Clinical Trial Drug Development
Randomized controlled trials (RCTs) that assess overall survival are considered the “gold standard” when evaluating the efficacy and safety of a new oncology intervention. However, single-arm trials that use surrogate endpoints (e.g., objective response rate or duration of response) to evaluate clin...
| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2022-01-01
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| Series: | Frontiers in Oncology |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fonc.2021.695936/full |
| _version_ | 1798035273277243392 |
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| author | Timothy A. Yap Ira Jacobs Elodie Baumfeld Andre Lauren J. Lee Darrin Beaupre Laurent Azoulay Laurent Azoulay |
| author_facet | Timothy A. Yap Ira Jacobs Elodie Baumfeld Andre Lauren J. Lee Darrin Beaupre Laurent Azoulay Laurent Azoulay |
| author_sort | Timothy A. Yap |
| collection | DOAJ |
| description | Randomized controlled trials (RCTs) that assess overall survival are considered the “gold standard” when evaluating the efficacy and safety of a new oncology intervention. However, single-arm trials that use surrogate endpoints (e.g., objective response rate or duration of response) to evaluate clinical benefit have become the basis for accelerated or breakthrough regulatory approval of precision oncology drugs for cases where the target and research populations are relatively small. Interpretation of efficacy in single-arm trials can be challenging because such studies lack a standard-of-care comparator arm. Although an external control group can be based on data from other clinical trials, using an external control group based on data collected outside of a trial may not only offer an alternative to both RCTs and uncontrolled single-arm trials, but it may also help improve decision-making by study sponsors or regulatory authorities. Hence, leveraging real-world data (RWD) to construct external control arms in clinical trials that investigate the efficacy and safety of drug interventions in oncology has become a topic of interest. Herein, we review the benefits and challenges associated with the use of RWD to construct external control groups, and the relevance of RWD to early oncology drug development. |
| first_indexed | 2024-04-11T20:55:55Z |
| format | Article |
| id | doaj.art-c0a4a1ac7a1d4799ae548861fb16cf51 |
| institution | Directory Open Access Journal |
| issn | 2234-943X |
| language | English |
| last_indexed | 2024-04-11T20:55:55Z |
| publishDate | 2022-01-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Oncology |
| spelling | doaj.art-c0a4a1ac7a1d4799ae548861fb16cf512022-12-22T04:03:41ZengFrontiers Media S.A.Frontiers in Oncology2234-943X2022-01-011110.3389/fonc.2021.695936695936Application of Real-World Data to External Control Groups in Oncology Clinical Trial Drug DevelopmentTimothy A. Yap0Ira Jacobs1Elodie Baumfeld Andre2Lauren J. Lee3Darrin Beaupre4Laurent Azoulay5Laurent Azoulay6Department of Investigational Cancer Therapeutics (Phase I Program), Division of Cancer Medicine, the University of Texas MD Anderson Cancer Center, Houston, TX, United StatesPfizer Inc., New York, NY, United StatesPfizer Inc., New York, NY, United StatesPfizer Inc., New York, NY, United StatesPfizer Inc., New York, NY, United StatesCentre for Clinical Epidemiology Lady Davis Institute, Jewish General Hospital, Montreal, QC, CanadaDepartment of Epidemiology, Biostatistics, and Occupational Health and Gerald Bronfman Department of Oncology, McGill University, Montreal, QC, CanadaRandomized controlled trials (RCTs) that assess overall survival are considered the “gold standard” when evaluating the efficacy and safety of a new oncology intervention. However, single-arm trials that use surrogate endpoints (e.g., objective response rate or duration of response) to evaluate clinical benefit have become the basis for accelerated or breakthrough regulatory approval of precision oncology drugs for cases where the target and research populations are relatively small. Interpretation of efficacy in single-arm trials can be challenging because such studies lack a standard-of-care comparator arm. Although an external control group can be based on data from other clinical trials, using an external control group based on data collected outside of a trial may not only offer an alternative to both RCTs and uncontrolled single-arm trials, but it may also help improve decision-making by study sponsors or regulatory authorities. Hence, leveraging real-world data (RWD) to construct external control arms in clinical trials that investigate the efficacy and safety of drug interventions in oncology has become a topic of interest. Herein, we review the benefits and challenges associated with the use of RWD to construct external control groups, and the relevance of RWD to early oncology drug development.https://www.frontiersin.org/articles/10.3389/fonc.2021.695936/fullcancerclinical trialdesigndrug developmentexternal control grouponcology |
| spellingShingle | Timothy A. Yap Ira Jacobs Elodie Baumfeld Andre Lauren J. Lee Darrin Beaupre Laurent Azoulay Laurent Azoulay Application of Real-World Data to External Control Groups in Oncology Clinical Trial Drug Development Frontiers in Oncology cancer clinical trial design drug development external control group oncology |
| title | Application of Real-World Data to External Control Groups in Oncology Clinical Trial Drug Development |
| title_full | Application of Real-World Data to External Control Groups in Oncology Clinical Trial Drug Development |
| title_fullStr | Application of Real-World Data to External Control Groups in Oncology Clinical Trial Drug Development |
| title_full_unstemmed | Application of Real-World Data to External Control Groups in Oncology Clinical Trial Drug Development |
| title_short | Application of Real-World Data to External Control Groups in Oncology Clinical Trial Drug Development |
| title_sort | application of real world data to external control groups in oncology clinical trial drug development |
| topic | cancer clinical trial design drug development external control group oncology |
| url | https://www.frontiersin.org/articles/10.3389/fonc.2021.695936/full |
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