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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...

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Autores principales: Yap, Timothy A., Jacobs, Ira, Baumfeld Andre, Elodie, Lee, Lauren J., Beaupre, Darrin, Azoulay, Laurent
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8771908/
https://www.ncbi.nlm.nih.gov/pubmed/35070951
http://dx.doi.org/10.3389/fonc.2021.695936
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author Yap, Timothy A.
Jacobs, Ira
Baumfeld Andre, Elodie
Lee, Lauren J.
Beaupre, Darrin
Azoulay, Laurent
author_facet Yap, Timothy A.
Jacobs, Ira
Baumfeld Andre, Elodie
Lee, Lauren J.
Beaupre, Darrin
Azoulay, Laurent
author_sort Yap, Timothy A.
collection PubMed
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.
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spelling pubmed-87719082022-01-21 Application of Real-World Data to External Control Groups in Oncology Clinical Trial Drug Development Yap, Timothy A. Jacobs, Ira Baumfeld Andre, Elodie Lee, Lauren J. Beaupre, Darrin Azoulay, Laurent Front Oncol Oncology 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. Frontiers Media S.A. 2022-01-06 /pmc/articles/PMC8771908/ /pubmed/35070951 http://dx.doi.org/10.3389/fonc.2021.695936 Text en Copyright © 2022 Yap, Jacobs, Baumfeld Andre, Lee, Beaupre and Azoulay https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Oncology
Yap, Timothy A.
Jacobs, Ira
Baumfeld Andre, Elodie
Lee, Lauren J.
Beaupre, Darrin
Azoulay, Laurent
Application of Real-World Data to External Control Groups in Oncology Clinical Trial Drug Development
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 Oncology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8771908/
https://www.ncbi.nlm.nih.gov/pubmed/35070951
http://dx.doi.org/10.3389/fonc.2021.695936
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