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

Abstract

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.

Application of multiple validated algorithms for identifying incident breast cancer among individuals with atopic dermatitis

“…1,2 These secondary data sources can be used to generate evidence as part of post-marketing surveillance systems, ad hoc observational studies, and in an increasing number of regulatory obligations. [3][4][5][6] Reliable estimates of disease incidence or prevalence using RWD often require development of validated algorithms (VAs) to identify appropriate patient populations and/or outcomes. [7][8][9] These algorithms are then adapted by other researchers for their specific population or research question.…”

“…Real‐world data (RWD), including electronic health records, medical claims data, and patient surveys, are often used in pharmacosurveillance and pharmacoepidemiologic studies 1,2 . These secondary data sources can be used to generate evidence as part of post‐marketing surveillance systems, ad hoc observational studies, and in an increasing number of regulatory obligations 3–6 …”

Navigating the unknown: how to best ‘reflect’ standard of care in indications without a dedicated treatment pathway in health technology assessment submissions

“…For example, in a NICE appraisal [ID3995] on sebelipase alfa, the lack of clinical agreement among the leading medical centers on patients' management created challenges for the committee to robustly justify the SoC arm in the base-case analysis [ 41 ]. Furthermore, generalizability issues when using data from clinical experts across different jurisdictions, although not uncommon in HTAs for rare or very rare diseases, could also pose challenges for its incorporation in decision-making as it may reflect differences in healthcare systems and allocation of local resources [ 42 ].…”

“…Early collaboration between manufacturers, decision-makers, and other stakeholders can also provide the opportunity to share learnings and encourage dialog across different geographical settings. For example, opportunities for real-world data transferability across borders may be more applicable for these situations, although current related guidance is not comprehensive enough to guide HTA submissions in the near future [ 42 ]. Data transferability will help stakeholders to collaborate and understand the evidentiary requirements early in the process and create efficiencies and consistency in decision-making considering the limited treatment options available for patients with these diseases [ 48 ].…”

Integrated Real-World Study Databases in 3 Diverse Asian Health Care Systems in Taiwan, India, and Thailand: Scoping Review

“…Such a framework is currently absent in Asia [ 6 ]. Considering the diversity and magnitude of health challenges, Asian health systems are expected to tremendously benefit from fit-for-purpose RWE for pharmacoeconomics, pharmacovigilance, and pharmacoepidemiology assessments [ 6 , 7 ].…”