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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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2022-11-01
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| Series: | Frontiers in Medicine |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fmed.2022.1073678/full |
| _version_ | 1797988810200449024 |
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| author | Ashley Jaksa Patrick J. Arena Patrick J. Arena Kelvin K. W. Chan Kelvin K. W. Chan Rami H. Ben-Joseph Páll Jónsson Ulka B. Campbell |
| author_facet | Ashley Jaksa Patrick J. Arena Patrick J. Arena Kelvin K. W. Chan Kelvin K. W. Chan Rami H. Ben-Joseph Páll Jónsson Ulka B. Campbell |
| author_sort | Ashley Jaksa |
| collection | DOAJ |
| 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. |
| first_indexed | 2024-04-11T08:08:56Z |
| format | Article |
| id | doaj.art-26eb172ca3ad4da8aeae11e78d6660b7 |
| institution | Directory Open Access Journal |
| issn | 2296-858X |
| language | English |
| last_indexed | 2024-04-11T08:08:56Z |
| publishDate | 2022-11-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Medicine |
| spelling | doaj.art-26eb172ca3ad4da8aeae11e78d6660b72022-12-22T04:35:25ZengFrontiers Media S.A.Frontiers in Medicine2296-858X2022-11-01910.3389/fmed.2022.10736781073678Transferability of real-world data across borders for regulatory and health technology assessment decision-makingAshley Jaksa0Patrick J. Arena1Patrick J. Arena2Kelvin K. W. Chan3Kelvin K. W. Chan4Rami H. Ben-Joseph5Páll Jónsson6Ulka B. Campbell7Scientific Research and Strategy, Aetion, Inc., New York, NY, United StatesScientific Research and Strategy, Aetion, Inc., New York, NY, United StatesDepartment of Epidemiology, University of California, Los Angeles, Los Angeles, CA, United StatesSunnybrook Health Sciences Centre, University of Toronto, Toronto, ON, CanadaCanadian Centre for Applied Research in Cancer Control, Toronto, ON, CanadaBig Data Real World Evidence, Jazz Pharmaceuticals, Palo Alto, CA, United StatesNational Institute for Health and Care Excellence, Manchester, United KingdomScientific Research and Strategy, Aetion, Inc., New York, NY, United StatesRecently, 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.https://www.frontiersin.org/articles/10.3389/fmed.2022.1073678/fulltransferabilityreal-world dataregulatory decision-makinghealth technology assessmentsdata relevance |
| spellingShingle | Ashley Jaksa Patrick J. Arena Patrick J. Arena Kelvin K. W. Chan Kelvin K. W. Chan Rami H. Ben-Joseph Páll Jónsson Ulka B. Campbell Transferability of real-world data across borders for regulatory and health technology assessment decision-making Frontiers in Medicine transferability real-world data regulatory decision-making health technology assessments data relevance |
| 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 | transferability real-world data regulatory decision-making health technology assessments data relevance |
| url | https://www.frontiersin.org/articles/10.3389/fmed.2022.1073678/full |
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