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Real-world evidence for regulatory decision-making: updated guidance from around the world

Leveraging the value of real-world evidence (RWE) to make informed regulatory decisions in the field of health care continues to gain momentum. Improving clinical evidence generation by evaluating the outcomes and patient experiences at the point-of-care would help achieve the ultimate aim of ensuri...

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Autores principales: Burns, Leah, Le Roux, Nadege, Kalesnik-Orszulak, Robert, Christian, Jennifer, Dudinak, Jennifer, Rockhold, Frank, Khozin, Sean, O’Donnell, John
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10643567/
https://www.ncbi.nlm.nih.gov/pubmed/38020096
http://dx.doi.org/10.3389/fmed.2023.1236462
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author Burns, Leah
Le Roux, Nadege
Kalesnik-Orszulak, Robert
Christian, Jennifer
Dudinak, Jennifer
Rockhold, Frank
Khozin, Sean
O’Donnell, John
author_facet Burns, Leah
Le Roux, Nadege
Kalesnik-Orszulak, Robert
Christian, Jennifer
Dudinak, Jennifer
Rockhold, Frank
Khozin, Sean
O’Donnell, John
author_sort Burns, Leah
collection PubMed
description Leveraging the value of real-world evidence (RWE) to make informed regulatory decisions in the field of health care continues to gain momentum. Improving clinical evidence generation by evaluating the outcomes and patient experiences at the point-of-care would help achieve the ultimate aim of ensuring that effective and safe treatments are rapidly approved for patient use. In our previous publication, we assessed the global regulatory landscape with respect to RWE and provided a review of the regional availability of frameworks and guidance through May 2021 on the basis of 3 key regulatory elements: regulatory RWE frameworks, data quality guidance, and study methods guidance. In the current review, we have updated and elaborated upon recent developments in the regulatory RWE environment from a regional perspective under the same 3 regulatory elements stated above. In addition, we have also included a new category on procedural guidance. The review also discusses the perceived gaps and potential opportunities for future development and harmonization in this field to support framework establishment in regions without pre-existing RWE policies. Additionally, the article reviews current developments of health technology assessment (HTA) bodies pertaining to RWE and discusses the status of evidentiary alignment among regulators and HTA agencies.
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spelling pubmed-106435672023-10-30 Real-world evidence for regulatory decision-making: updated guidance from around the world Burns, Leah Le Roux, Nadege Kalesnik-Orszulak, Robert Christian, Jennifer Dudinak, Jennifer Rockhold, Frank Khozin, Sean O’Donnell, John Front Med (Lausanne) Medicine Leveraging the value of real-world evidence (RWE) to make informed regulatory decisions in the field of health care continues to gain momentum. Improving clinical evidence generation by evaluating the outcomes and patient experiences at the point-of-care would help achieve the ultimate aim of ensuring that effective and safe treatments are rapidly approved for patient use. In our previous publication, we assessed the global regulatory landscape with respect to RWE and provided a review of the regional availability of frameworks and guidance through May 2021 on the basis of 3 key regulatory elements: regulatory RWE frameworks, data quality guidance, and study methods guidance. In the current review, we have updated and elaborated upon recent developments in the regulatory RWE environment from a regional perspective under the same 3 regulatory elements stated above. In addition, we have also included a new category on procedural guidance. The review also discusses the perceived gaps and potential opportunities for future development and harmonization in this field to support framework establishment in regions without pre-existing RWE policies. Additionally, the article reviews current developments of health technology assessment (HTA) bodies pertaining to RWE and discusses the status of evidentiary alignment among regulators and HTA agencies. Frontiers Media S.A. 2023-10-30 /pmc/articles/PMC10643567/ /pubmed/38020096 http://dx.doi.org/10.3389/fmed.2023.1236462 Text en Copyright © 2023 Burns, Le Roux, Kalesnik-Orszulak, Christian, Dudinak, Rockhold, Khozin and O’Donnell. 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 Medicine
Burns, Leah
Le Roux, Nadege
Kalesnik-Orszulak, Robert
Christian, Jennifer
Dudinak, Jennifer
Rockhold, Frank
Khozin, Sean
O’Donnell, John
Real-world evidence for regulatory decision-making: updated guidance from around the world
title Real-world evidence for regulatory decision-making: updated guidance from around the world
title_full Real-world evidence for regulatory decision-making: updated guidance from around the world
title_fullStr Real-world evidence for regulatory decision-making: updated guidance from around the world
title_full_unstemmed Real-world evidence for regulatory decision-making: updated guidance from around the world
title_short Real-world evidence for regulatory decision-making: updated guidance from around the world
title_sort real-world evidence for regulatory decision-making: updated guidance from around the world
topic Medicine
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10643567/
https://www.ncbi.nlm.nih.gov/pubmed/38020096
http://dx.doi.org/10.3389/fmed.2023.1236462
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