Comment Now on FDA Draft Guidance on Use of EHR, Claims Data as RWD

Oct. 4, 2021 – As part of its real-world evidence (RWE) program, the Food and Drug Administration last week issued a draft guidance to provide sponsors, researchers and other stakeholders with the agency’s current thinking about the use of electronic health records (EHRs) or medical claims data in clinical studies “to support a regulatory decision for effectiveness or safety.” Public comments on the draft guidance are due by Nov. 29.

The FDA committed to publish guidance on how RWE can contribute to the assessment of safety and effectiveness in regulatory submissions under the Prescription Drug User Fee Amendments of 2017. A 21st Century Cures Act mandate established a program to evaluate the potential use of RWE to help support the approval of a new indication for an already approved drug or to help support or satisfy post-approval study requirements. “FDA is issuing the draft guidance as part of a series of guidance documents to satisfy the Cures Act mandate and the PDUFA VI goal,” according to a Sept. 30 Federal Register notice.

The September 2021 draft guidance, “Real-World Data: Assessing Electronic Health Records and Medical Claims Data To Support Regulatory Decision-Making for Drug and Biological Products,” builds on an FDA guidance document issued in 2013, “Best Practices for Conducting and Reporting Pharmacoepidemiological Safety Studies Using Electronic Healthcare Data.”

The new draft guidance discusses the following topics related to the use of EHR and medical claims data as real-world data (RWD) :

  • Selection of data sources that appropriately address the study question and sufficiently characterize study populations, exposure(s), outcome(s) of interest, and key covariates;
  • Development and validation of definitions for study design elements (e.g., exposure, outcomes, covariates); and
  • Data provenance and quality during data accrual, data curation, and into the final study-specific dataset.

The FDA stipulates that because EHR and medical claims data were not designed to support regulatory submissions, they have some limitations. Examples of these limitations include:

  • The purpose of medical claims data is to support payment for care; claims may not accurately reflect a particular disease, or a patient may have a particular disease or condition that is not reflected in claims data.
  • EHR data are generated for use in clinical care and may also serve as a basis for billing and for auditing of practice quality measures. Data recorded in an EHR system depend on each health care system’s practices for patient care and the clinical practices of its providers. In addition, data collection is limited to the data captured within an EHR system or network, and may not represent comprehensive care (e.g., care obtained outside of the health care system).
  • For prospective clinical studies proposing to use EHRs, it may be possible to modify the EHR system for the purpose of collecting additional patient data during routine care through an add-on module to the EHR system. However, given the limited ability to add modules to collect extensive additional information, EHR-based data collection may still not be comprehensive.

Sponsors also should be aware of the differences that data from “around the world or between health care systems” present, and how those differences “may affect the relevance of the data source to the study question,” the document states. Those differences include age, socioeconomic status, health conditions, and risk factors, as well as variations in how medications are covered and administered in other countries.

The draft guidance does not include recommendations on choice of study design or type of statistical analysis, and the FDA does not endorse any type of data source or study methodology in the document. For all study designs, “it is important to ensure the reliability and relevance of the data used to help support a regulatory decision,” the guidance states. The agency also recommends that sponsors should seek FDA input by requesting comments from or a meeting with the appropriate FDA review division to discuss the proposed study.

Following the Nov. 29 comment deadline, the FDA will begin work on the final version of the guidance.