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Transferability of real-world data across borders for regulatory and health technology assessment decision-making

Recently, there has been increased consideration of real-world data (RWD) and real-world evidence (RWE) in regulatory and health technology assessment (HTA) decision-making. Due to challenges in identifying high-quality and relevant RWD sources, researchers and regulatory/HTA bodies may turn to RWD...

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Autores principales: Jaksa, Ashley, Arena, Patrick J., Chan, Kelvin K. W., Ben-Joseph, Rami H., Jónsson, Páll, Campbell, Ulka B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9709526/
https://www.ncbi.nlm.nih.gov/pubmed/36465931
http://dx.doi.org/10.3389/fmed.2022.1073678
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author Jaksa, Ashley
Arena, Patrick J.
Chan, Kelvin K. W.
Ben-Joseph, Rami H.
Jónsson, Páll
Campbell, Ulka B.
author_facet Jaksa, Ashley
Arena, Patrick J.
Chan, Kelvin K. W.
Ben-Joseph, Rami H.
Jónsson, Páll
Campbell, Ulka B.
author_sort Jaksa, Ashley
collection PubMed
description Recently, there has been increased consideration of real-world data (RWD) and real-world evidence (RWE) in regulatory and health technology assessment (HTA) decision-making. Due to challenges in identifying high-quality and relevant RWD sources, researchers and regulatory/HTA bodies may turn to RWD generated in locales outside of the locale of interest (referred to as “transferring RWD”). We therefore performed a review of stakeholder guidance as well as selected case studies to identify themes for researchers to consider when transferring RWD from one jurisdiction to another. Our review highlighted that there is limited consensus on defining decision-grade, transferred RWD; certain stakeholders have issued relevant guidance, but the recommendations are high-level and additional effort is needed to generate comprehensive guidance. Additionally, the case studies revealed that RWD transferability has not been a consistent concern for regulatory/HTA bodies and that more focus has been put on the evaluation of internal validity. To help develop transferability best practices (alongside internal validity best practices), we suggest that researchers address the following considerations in their justification for transferring RWD: treatment pathways, nature of the healthcare system, incidence/prevalence of indication, and patient demographics. We also recommend that RWD transferability should garner more attention as the use of imported RWD could open doors to high-quality data sources and potentially reduce methodological issues that often arise in the use of local RWD; we thus hope this review provides a foundation for further dialogue around the suitability and utility of transferred RWD in the regulatory/HTA decision-making space.
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spelling pubmed-97095262022-12-01 Transferability of real-world data across borders for regulatory and health technology assessment decision-making Jaksa, Ashley Arena, Patrick J. Chan, Kelvin K. W. Ben-Joseph, Rami H. Jónsson, Páll Campbell, Ulka B. Front Med (Lausanne) Medicine Recently, there has been increased consideration of real-world data (RWD) and real-world evidence (RWE) in regulatory and health technology assessment (HTA) decision-making. Due to challenges in identifying high-quality and relevant RWD sources, researchers and regulatory/HTA bodies may turn to RWD generated in locales outside of the locale of interest (referred to as “transferring RWD”). We therefore performed a review of stakeholder guidance as well as selected case studies to identify themes for researchers to consider when transferring RWD from one jurisdiction to another. Our review highlighted that there is limited consensus on defining decision-grade, transferred RWD; certain stakeholders have issued relevant guidance, but the recommendations are high-level and additional effort is needed to generate comprehensive guidance. Additionally, the case studies revealed that RWD transferability has not been a consistent concern for regulatory/HTA bodies and that more focus has been put on the evaluation of internal validity. To help develop transferability best practices (alongside internal validity best practices), we suggest that researchers address the following considerations in their justification for transferring RWD: treatment pathways, nature of the healthcare system, incidence/prevalence of indication, and patient demographics. We also recommend that RWD transferability should garner more attention as the use of imported RWD could open doors to high-quality data sources and potentially reduce methodological issues that often arise in the use of local RWD; we thus hope this review provides a foundation for further dialogue around the suitability and utility of transferred RWD in the regulatory/HTA decision-making space. Frontiers Media S.A. 2022-11-16 /pmc/articles/PMC9709526/ /pubmed/36465931 http://dx.doi.org/10.3389/fmed.2022.1073678 Text en Copyright © 2022 Jaksa, Arena, Chan, Ben-Joseph, Jónsson and Campbell. 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
Jaksa, Ashley
Arena, Patrick J.
Chan, Kelvin K. W.
Ben-Joseph, Rami H.
Jónsson, Páll
Campbell, Ulka B.
Transferability of real-world data across borders for regulatory and health technology assessment decision-making
title Transferability of real-world data across borders for regulatory and health technology assessment decision-making
title_full Transferability of real-world data across borders for regulatory and health technology assessment decision-making
title_fullStr Transferability of real-world data across borders for regulatory and health technology assessment decision-making
title_full_unstemmed Transferability of real-world data across borders for regulatory and health technology assessment decision-making
title_short Transferability of real-world data across borders for regulatory and health technology assessment decision-making
title_sort transferability of real-world data across borders for regulatory and health technology assessment decision-making
topic Medicine
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9709526/
https://www.ncbi.nlm.nih.gov/pubmed/36465931
http://dx.doi.org/10.3389/fmed.2022.1073678
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