Key Components of a Strong Real World Data Quality and Relevance Package for Regulatory or Health Technology Assessment Submission

OBJECTIVES: Real world data (RWD) is increasingly used to generate evidence on drugs and biologics for regulatory and Health Technology Assessment (HTA) decisions. To facilitate this growing reliance on RWE, the U.S. Food & Drug Administration has issued guidance for relevance and data quality to use RWD for regulatory purposes. TransCelerate has released related considerations for potential auditing, particularly when RWD are not owned by the Sponsor.

METHODS: After careful review of TransCelerate Considerations, a working group of ISPE, ISPOR and industry leaders convened to craft a tangible view of what should be included in submissions to justify relevance and data quality of data sources considered and selected to implement real-world evidence (RWE) protocols for regulatory purposes. Using the TransCelerate work as foundation, we sought specificity and structure for a potential data feasibility package.

RESULTS: The relevance and quality of RWD is contextual, depending on the specific data needed to address the research question and regulatory decision. Multiple data sources may be evaluated for feasibility to address the research question using RWD. Each must be described as part of the rationale for selection of specific data sources to be used. A table of contents (TOC) and a dynamic item list were developed to provide tactical guidance and ensure inclusion of pertinent details needed for regulators and HTA assessors to evaluate the utility of each data source. The TOC and dynamic itemized form were reviewed and piloted by members of the working group’s respective organizations, and then sent to Duke-Margolis Center for Health Policy for review and comment.

CONCLUSIONS: A submission package template should help researchers justify the evaluation and selection of RWD sources for addressing a specific research question and regulatory/HTA decision and ensure the inclusion in submissions of pertinent information needed for evaluation by regulators and HTAs .