Girman CJ, Ritchey ME, Martin D, Zhou WJ, Dreyer NJ. How do we assess when electronic health records or claims databases are fit for a specific research or regulatory purpose? Presented at the 35th Annual ICPE Conference; August 26, 2019. Philadelphia, PA. [abstract] Pharmacoepidemiol Drug Saf. 2019 Aug 20; 28(S2):2. doi: 10.1002/pds.4864.


BACKGROUND: There is escalating interest in the use of real-world data (RWD) not only for safety but also for effectiveness evaluation. FDA released a framework for public comment at the end of 2018 which focused on randomized trials in clinical practice, potential observational designs, and data quality aspects for the use of real world evidence (RWE) in regulatory and clinical contexts. There is no 'one size fits all' approach to qualifying a data source for broad research or regulatory purposes as the feasibility of using RWD depends on how the results will be used, the anticipated effect size and the quality of the data for the study components critical to addressing the specific research question.

OBJECTIVES: To review the FDA framework on RWD and provide a new structured study-specific context by which to assess the feasibility of using RWD for a specific research question or regulatory purpose (new indication or expanded labeling). This should be of interest to all pharmacoepidemiologists and researchers involved in the design and conduct of real world studies for potential regulatory or other purposes.

DESCRIPTION: Accuracy and reliability of data (including extent of missing data) to define specific components of the research question, such as the population, intervention, comparator, and outcome), fundamentally drive whether RWD in electronic health record or claims databases can be useful for a specific envisioned study. The adequacy of ascertainment of these components along with sample size and anticipated treatment effect size leads to a practical structured approach for assessing utility of RWD in the context of the specific research question. Discussion will consider the perspective of the use of RWD for regulatory labeling or new indications, as well as for other research purposes. Dr. Zhou will introduce the concepts of data quality, relevance and validation based on the FDA Framework followed by Dr. Ritchey presenting a new practical approach to assess RWD feasibility in the context of the specific research question. Dr. Martin and Dr. Girman will review the utility of this study-specific contextual approach for assessing feasibility of RWD for fit-forregulatory purposes and for general research studies, respectively. A 20-minute discussion led by Dr. Dreyer, involving audience participation with the speaker panel, will conclude the session.

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