(ASPOT02) Contents of a Strong Real World Data Quality and Relevance Package for Regulatory or Health Technology Assessment Submission

Background: Regulatory and health technology assessment (HTA) decisions are increasing relying on real world data (RWD) for drugs, biologics and devices. In 2023, TransCelerate released a series of considerations for audit readiness, particularly when RWD are not owned by the Sponsor, that should be addressed when submitting evidence to support regulatory or HTA decisions. Through a literature review and FDA RWE guidance documents, they identified issues across conceptual data domains of relevance, accrual, provenance, completeness and accuracy.

Objectives: To clarify the components of a real world data quality and relevance submission package for regulators and health technology assessment agencies, and present a potential framework for documentation to be submitted with RWE to regulators and payers

Methods: After careful review of the considerations released by TransCelerate, we convened a working group of ISPE/ISPOR/TransCelerate leaders to craft a more tangible view of what more specifically should be included in submission packages to justify data relevance and data quality of RWD sources considered for use in implementing a real-world evidence (RWE) protocol. Using the TransCelerate work as foundation, we sought clarity and specificity in a data management and processing package and how it might be structured.

Results: The relevance and quality of RWD is contextual, depending on the specific data elements needed to address the research question and regulatory decision. Multiple data sources are often evaluated for feasibility in addressing the research question using RWD. Each of these must be described as part of the rationale for selection of the specific data sources to be used. A table of contents (TOC) for a potential data submission package was created. In addition, a dynamic itemized framework was developed to ensure inclusion of pertinent details needed for regulators and HTA assessors to evaluate the utility of each data source. After review and revision, the TOC and dynamic checklist were sent to Duke-Margolis for feedback. The structure of a data package for submission along with the nature of the dynamic framework will be reviewed.

Conclusions: A more detailed submission package framework will help researchers understand what they need to justify in terms of the evaluation and selection of RWD sources for a specific research question and regulatory/HTA decision, to better ensure that pertinent information is included. It can also be useful for negotiating with data providers, and early discussions with regulators.