Manufacturers are increasingly using real-world data (RWD) to generate real-world evidence (RWE) to support health technology assessment- (HTA-) and regulatory-facing assessments and applications. As we think about the benefits of using RWE in this manner, we need to also acknowledge the areas where more comprehensive best practices are needed. One key area for improvement is how to approach situations where local, high-quality data is unavailable. It is important that decision-makers consider when it may be appropriate for researchers to import RWD from one jurisdiction to support HTA or regulatory decision-making in a different jurisdiction (i.e., RWD transferability). In order to be comfortable with RWD transferability, researchers and decision-makers must have a robust understanding of data fitness to facilitate the identification of high-quality and fit-for-purpose RWD to address the research question at hand.
In a recent publication in Frontiers in Medicine, we collaborated with co-authors from the University of Toronto, the Canadian Centre for Applied Research in Cancer Control, Jazz Pharmaceuticals, and the U.K.’s National Institute for Health and Care Excellence (NICE) to discuss the current state of transferability of RWD across borders for regulatory and HTA decision-making.
Goal of the study
The review aimed to catalog which HTAs and regulators specifically discussed the concept of RWD transferability in their guidances and how to approach understanding transferability’s value in decision-making through case studies.
Process for the analysis and initial findings
We conducted a detailed review of RWD guidances from a variety of North American and European stakeholders. We found that four stakeholders published guidances that explicitly mention the transferability of international RWD. These four stakeholders are the U.S.’s Agency for Healthcare Research and Quality (AHRQ) and the Food and Drug Administration (FDA), Germany’s Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG), and the U.K.’s NICE. Each of the four stakeholders discussed the importance of assessing context-specific factors when using RWD from another jurisdiction, such as differences in the treatment pathways and/or healthcare system and local patient context. We also identified three case studies where imported RWD was submitted for safety, efficacy, and/or cost-effectiveness evaluations.
Detailed findings and impact
When aggregating and analyzing common themes across stakeholder guidances and case studies, a few common themes emerged. Sponsors should keep the following factors at the forefront of their analysis when assessing the fitness of RWD from another jurisdiction:
Treatment pathways in the jurisdiction of origin
There are a variety of sources of information available on treatment pathways in different countries. Sponsors should assess everything from evidence-based recommendations in clinical guidelines to actual prescribing patterns and the availability of therapies, all of which can vary by location. This factor is important because when treatment discrepancies become too large, inferences from the transferred data to the population of interest may be problematic.
The healthcare system in the jurisdiction of origin
Each country greatly varies with regards to its care delivery and the ability of its patients to access health care. It is therefore important for researchers to document similarities as well as differences in patient access to care and where care is delivered between jurisdictions.
Incidence/prevalence of indication in the jurisdiction of origin
Due to different population mixes and environmental factors, the incidence/prevalence of an indication can be heterogeneous across geographies. It is important to determine how factors associated with incidence/prevalence in one jurisdiction are similar to or different from the jurisdiction of the HTA or regulator reviewing the potential evidence.
Patient demographics in the jurisdiction of origin
Patient demographics, such as age, sex, race/ethnicity, socioeconomic status, and genetic profile, are all common risk factors for disease and can thus impact the incidence/severity of the indication as well as other health outcomes. Sponsors should therefore be sure to account for differences in patient demographics across jurisdictions when feasible.
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Ashley Jaksa, Market Access Scientific Strategy Lead at Aetion, focuses on partnering with regulatory and HTA agencies to to support their use of RWE in decision-making, and to help set methodological guidance for RWE generation. She also works with Aetion’s biopharma clients to ensure their RWE studies meet decision-makers’ evidence standards. She holds a bachelor’s degree from the University of Michigan-Ann Arbor and a MPH from Yale University.
Patrick Arena, Consultant at Aetion, is currently a doctoral candidate in the epidemiology department at the UCLA Fielding School of Public Health. Prior to UCLA, he graduated from Boston College with a BS in Biology and Columbia University with a MPH in Epidemiology. Patrick previously worked at Pfizer, where he contributed to post-marketing studies as well as Phase II/III vaccine clinical trials.
To connect with one of our scientific experts at Aetion, please contact us at https://aetion.com/contact-us/.