Real-World Data for Clinical Trials: Brand New FDA Expectations for Use of Real-World Evidence for Regulatory Decision-Making

This roundtable will address the major challenges for sponsors posed by FDA’s recent final guidance on the assessment and use of electronic health record (EHR) and medical claims data in clinical studies to support regulatory decision-making. Although the final guidance focuses on clinical studies of drug and biological products, it is also pertinent to clinical studies of medical devices that intend to use these data sources. EHR and medical claims data represent an enormous trove of real-world health outcomes data to support approval of a new indication for an approved drug, the approval of a medical device, and postapproval studies. The final guidance reflects the FDA’s rapidly evolving major concerns about the reliability and relevance of these clinical data sources. We will address current FDA expectations regarding:

• Predefinition and validation of all study elements (relevant populations, exposures, outcomes of interest, and covariates) for the specific study question;
• Identifying and addressing limitations of incomplete, absent, or misclassified information;
• Validation of outcomes of interest, including confounding variables and effect modifiers, and justification of the validation process;
• Applying formal methods in a QA/QC plan for implementing quality assurance and quality checks for data accrual, curation, transformation, and analysis; and
• Knowing how to identify when EHR or medical claims data are not “good enough” to support regulatory decision-making for your product.