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Treatment effectiveness in a rare oncology indication: Lessons from an external control cohort study

Real‐world data (RWD) reflecting patient treatment in routine clinical practice can be used to develop external control groups for single‐arm trials. External controls can provide valuable benchmark results on potential comparator drug effectiveness, particularly in rare indications when randomized...

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Autores principales: Oksen, Dina, Prince, Patricia, Boutmy, Emmanuelle, Garry, Elizabeth M., Ellers‐Lenz, Barbara, Estrin, Adina, Johne, Andreas, Verpillat, Patrice, Gatto, Nicolle M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9372419/
https://www.ncbi.nlm.nih.gov/pubmed/35661422
http://dx.doi.org/10.1111/cts.13315
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author Oksen, Dina
Prince, Patricia
Boutmy, Emmanuelle
Garry, Elizabeth M.
Ellers‐Lenz, Barbara
Estrin, Adina
Johne, Andreas
Verpillat, Patrice
Gatto, Nicolle M.
author_facet Oksen, Dina
Prince, Patricia
Boutmy, Emmanuelle
Garry, Elizabeth M.
Ellers‐Lenz, Barbara
Estrin, Adina
Johne, Andreas
Verpillat, Patrice
Gatto, Nicolle M.
author_sort Oksen, Dina
collection PubMed
description Real‐world data (RWD) reflecting patient treatment in routine clinical practice can be used to develop external control groups for single‐arm trials. External controls can provide valuable benchmark results on potential comparator drug effectiveness, particularly in rare indications when randomized controlled trials are either infeasible or unethical. This paper describes lessons learned from a descriptive real‐world external control cohort study conducted to provide benchmark data for a single‐arm clinical trial in a rare oncology biomarker driven disease. Conducting external control cohort studies to evaluate treatment effectiveness in rare indications likely will present data and analysis challenges as seen in the example study. However, there are mitigating measures that can be applied in the study design, identification of RWD sources, and data analysis. The lessons learned and reported here with a proposal of an external control study framework can provide guidance for future research in this area, and may be applicable as well in other rare indications. Taking these learnings into consideration, the use of real‐world external controls to contextualize treatment effectiveness in rare indications is a valuable approach and warrants further application in the future.
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spelling pubmed-93724192022-08-16 Treatment effectiveness in a rare oncology indication: Lessons from an external control cohort study Oksen, Dina Prince, Patricia Boutmy, Emmanuelle Garry, Elizabeth M. Ellers‐Lenz, Barbara Estrin, Adina Johne, Andreas Verpillat, Patrice Gatto, Nicolle M. Clin Transl Sci Research Real‐world data (RWD) reflecting patient treatment in routine clinical practice can be used to develop external control groups for single‐arm trials. External controls can provide valuable benchmark results on potential comparator drug effectiveness, particularly in rare indications when randomized controlled trials are either infeasible or unethical. This paper describes lessons learned from a descriptive real‐world external control cohort study conducted to provide benchmark data for a single‐arm clinical trial in a rare oncology biomarker driven disease. Conducting external control cohort studies to evaluate treatment effectiveness in rare indications likely will present data and analysis challenges as seen in the example study. However, there are mitigating measures that can be applied in the study design, identification of RWD sources, and data analysis. The lessons learned and reported here with a proposal of an external control study framework can provide guidance for future research in this area, and may be applicable as well in other rare indications. Taking these learnings into consideration, the use of real‐world external controls to contextualize treatment effectiveness in rare indications is a valuable approach and warrants further application in the future. John Wiley and Sons Inc. 2022-06-11 2022-08 /pmc/articles/PMC9372419/ /pubmed/35661422 http://dx.doi.org/10.1111/cts.13315 Text en © 2022 The Authors. Clinical and Translational Science published by Wiley Periodicals LLC on behalf of American Society for Clinical Pharmacology and Therapeutics. https://creativecommons.org/licenses/by-nc/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc/4.0/ (https://creativecommons.org/licenses/by-nc/4.0/) License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.
spellingShingle Research
Oksen, Dina
Prince, Patricia
Boutmy, Emmanuelle
Garry, Elizabeth M.
Ellers‐Lenz, Barbara
Estrin, Adina
Johne, Andreas
Verpillat, Patrice
Gatto, Nicolle M.
Treatment effectiveness in a rare oncology indication: Lessons from an external control cohort study
title Treatment effectiveness in a rare oncology indication: Lessons from an external control cohort study
title_full Treatment effectiveness in a rare oncology indication: Lessons from an external control cohort study
title_fullStr Treatment effectiveness in a rare oncology indication: Lessons from an external control cohort study
title_full_unstemmed Treatment effectiveness in a rare oncology indication: Lessons from an external control cohort study
title_short Treatment effectiveness in a rare oncology indication: Lessons from an external control cohort study
title_sort treatment effectiveness in a rare oncology indication: lessons from an external control cohort study
topic Research
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9372419/
https://www.ncbi.nlm.nih.gov/pubmed/35661422
http://dx.doi.org/10.1111/cts.13315
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