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Use of Real‐World Evidence to Drive Drug Development Strategy and Inform Clinical Trial Design
Interest in real‐world data (RWD) and real‐world evidence (RWE) to expedite and enrich the development of new biopharmaceutical products has proliferated in recent years, spurred by the 21st Century Cures Act in the United States and similar policy efforts in other countries, willingness by regulato...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9299990/ https://www.ncbi.nlm.nih.gov/pubmed/34839524 http://dx.doi.org/10.1002/cpt.2480 |
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author | Dagenais, Simon Russo, Leo Madsen, Ann Webster, Jen Becnel, Lauren |
author_facet | Dagenais, Simon Russo, Leo Madsen, Ann Webster, Jen Becnel, Lauren |
author_sort | Dagenais, Simon |
collection | PubMed |
description | Interest in real‐world data (RWD) and real‐world evidence (RWE) to expedite and enrich the development of new biopharmaceutical products has proliferated in recent years, spurred by the 21st Century Cures Act in the United States and similar policy efforts in other countries, willingness by regulators to consider RWE in their decisions, demands from third‐party payers, and growing concerns about the limitations of traditional clinical trials. Although much of the recent literature on RWE has focused on potential regulatory uses (e.g., product approvals in oncology or rare diseases based on single‐arm trials with external control arms), this article reviews how biopharmaceutical companies can leverage RWE to inform internal decisions made throughout the product development process. Specifically, this article will review use of RWD to guide pipeline and portfolio strategy; use of novel sources of RWD to inform product development, use of RWD to inform clinical development, use of advanced analytics to harness “big” RWD, and considerations when using RWD to inform internal decisions. Topics discussed will include the use of molecular, clinicogenomic, medical imaging, radiomic, and patient‐derived xenograft data to augment traditional sources of RWE, the use of RWD to inform clinical trial eligibility criteria, enrich trial population based on predicted response, select endpoints, estimate sample size, understand disease progression, and enhance diversity of participants, the growing use of data tokenization and advanced analytical techniques based on artificial intelligence in RWE, as well as the importance of data quality and methodological transparency in RWE. |
format | Online Article Text |
id | pubmed-9299990 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | John Wiley and Sons Inc. |
record_format | MEDLINE/PubMed |
spelling | pubmed-92999902022-07-22 Use of Real‐World Evidence to Drive Drug Development Strategy and Inform Clinical Trial Design Dagenais, Simon Russo, Leo Madsen, Ann Webster, Jen Becnel, Lauren Clin Pharmacol Ther Reviews Interest in real‐world data (RWD) and real‐world evidence (RWE) to expedite and enrich the development of new biopharmaceutical products has proliferated in recent years, spurred by the 21st Century Cures Act in the United States and similar policy efforts in other countries, willingness by regulators to consider RWE in their decisions, demands from third‐party payers, and growing concerns about the limitations of traditional clinical trials. Although much of the recent literature on RWE has focused on potential regulatory uses (e.g., product approvals in oncology or rare diseases based on single‐arm trials with external control arms), this article reviews how biopharmaceutical companies can leverage RWE to inform internal decisions made throughout the product development process. Specifically, this article will review use of RWD to guide pipeline and portfolio strategy; use of novel sources of RWD to inform product development, use of RWD to inform clinical development, use of advanced analytics to harness “big” RWD, and considerations when using RWD to inform internal decisions. Topics discussed will include the use of molecular, clinicogenomic, medical imaging, radiomic, and patient‐derived xenograft data to augment traditional sources of RWE, the use of RWD to inform clinical trial eligibility criteria, enrich trial population based on predicted response, select endpoints, estimate sample size, understand disease progression, and enhance diversity of participants, the growing use of data tokenization and advanced analytical techniques based on artificial intelligence in RWE, as well as the importance of data quality and methodological transparency in RWE. John Wiley and Sons Inc. 2021-11-28 2022-01 /pmc/articles/PMC9299990/ /pubmed/34839524 http://dx.doi.org/10.1002/cpt.2480 Text en © 2021 The Authors. Clinical Pharmacology & Therapeutics published by Wiley Periodicals LLC on behalf of American Society for Clinical Pharmacology and Therapeutics. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ (https://creativecommons.org/licenses/by-nc-nd/4.0/) License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made. |
spellingShingle | Reviews Dagenais, Simon Russo, Leo Madsen, Ann Webster, Jen Becnel, Lauren Use of Real‐World Evidence to Drive Drug Development Strategy and Inform Clinical Trial Design |
title | Use of Real‐World Evidence to Drive Drug Development Strategy and Inform Clinical Trial Design |
title_full | Use of Real‐World Evidence to Drive Drug Development Strategy and Inform Clinical Trial Design |
title_fullStr | Use of Real‐World Evidence to Drive Drug Development Strategy and Inform Clinical Trial Design |
title_full_unstemmed | Use of Real‐World Evidence to Drive Drug Development Strategy and Inform Clinical Trial Design |
title_short | Use of Real‐World Evidence to Drive Drug Development Strategy and Inform Clinical Trial Design |
title_sort | use of real‐world evidence to drive drug development strategy and inform clinical trial design |
topic | Reviews |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9299990/ https://www.ncbi.nlm.nih.gov/pubmed/34839524 http://dx.doi.org/10.1002/cpt.2480 |
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