11/30/2021

The US Food and Drug Administration (FDA) released a draft guidance “Data Standards for Drug and Biological Product Submissions Containing Real-World Data” for public comment in October 2021. This draft guidance is published in line with the 21st Century Cures Act (Cures Act). Part of the Cures Act is related to the use of real-world evidence (RWE) to help support regulatory decision making, including the approval of a new indication for an approved drug or to support post approval study requirements. According to the FDA, RWE is “data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than traditional clinical trials” and the “clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of real word data (RWD).” RWD comes from medical billing claims, electronic health records (EHR), clinical registries, patient-generated data including from in-home use, and other sources. RWE provides valuable information on the safety and efficacy of a drug, biologic, or device when it is used as intended, outside the constraints of a clinical trial.

Regulatory Background

The guidance document “Providing Regulatory Submissions In Electronic Format — Standardized Study Data” (Study Data Guidance) references a data specification document, the Data Standards Catalog (Catalog). Both the Study Data Guidance and the Catalog describe the requirements for electronic submissions of standardized clinical study data and confirm that data must be submitted in a format that the FDA is able to process, review, and archive. The draft guidance describes how a sponsor should submit study data obtained from RWE sources while following the specifications in the Catalog.

Summary of Draft Guidance

There are many challenges to standardizing RWE data for regulatory submissions, including differences in terminology, format, methods of data aggregation, or processes that can impact data quality such as de-identification. If there are challenges in transforming RWE data to an FDA supported format, they need to be documented including the rationale and justification for approaches used to reconcile any challenges. Examples of challenges include inconsistent coding or changes in data collection practices. Also, if data need to be changed to support the appropriate format, this should also be documented as well as any potential impacts resulting from the change.

Sponsors should ensure there are adequate processes in place to demonstrate confidence in the RWE data collected and transformed, and this should be documented. The FDA recommends that sponsors contact the applicable FDA review division early on to discuss the proposed approach for using RWE data in their submissions, including reviewing the protocol, data management plan, and study reports.

The draft guidance appendix provides several examples of how to map RWE to the study data submission standards, the Clinical Data Interchange Standards Consortium (CDISC) Study Data Tabulation Model (SDTM).

Comment now through January 21, 2022 HERE.

Using RWE to support applications for drug or biologics can be expected to expedite the data collection process, thus leading to safe and effective products getting to market faster. For more information on the FDA’s framework for RWE, visit our blog “FDA Framework for Use of Real-World Evidence.” In September, the FDA published a draft guidance “Real-World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision Making for Drug and Biological Products”. Read our blog HERE for more information. Additional guidance on the use of RWE can be expected. Also, the FDA plans to issue guidance or to update the Catalog with standards for study data that are derived from RWD sources.

If you are preparing a regulatory submission and need assistance with utilizing RWE, Clinical Pathways can provide training as it is important to know how to apply these methods. Contact us at info@clinicalpathwaysresearch.com.

- The Clinical Pathways Team

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