FDA Unveils Criteria for Reporting Real-World Evidence (RWE) Submissions

The U.S. Food and Drug Administration (FDA) recently published its criteria for reporting on the use of real-world evidence (RWE) in drug and biologic submissions, as part of its commitments under the Prescription Drug User Fee Act (PDUFA). This new reporting initiative aims to enhance transparency by publicly sharing aggregated data on how companies are using real-world data (RWD) to support drug and biologic regulatory submissions. Understanding what types of submissions will be reported can provide valuable insights into how other companies are utilizing RWE.

How Does This Impact Drug and Biologic Companies?

Understanding what types of submissions will be reported can provide valuable insights into how other companies are utilizing RWE. This transparency allows companies to benchmark against industry practices, learning how other companies are incorporating RWE into their regulatory submissions. By gaining insights from aggregated data, you can refine your own submission approaches and enhance your strategies. Staying informed on the latest trends and regulatory expectations related to RWE helps improve decision-making regarding the use of real-world data in product development and regulatory strategies. Leveraging the increased transparency from the FDA’s reporting enables your company to optimize its use of RWE, supporting more robust and compliant submissions.

What is Real Word Evidence (RWE) and Real World Data (RWD)?

The FDA defines real-world evidence as, “clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of [real-world data].” Real-world data (RWD) are, “data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources. . . . includ[ing] data derived from electronic health records, medical claims data, data from product or disease registries, and data gathered from other sources (such as digital health technologies) that can inform on health status.”

Why is RWE Important?

The FDA uses RWE, “to monitor and evaluate the postmarket safety of approved drugs.” With the reauthorization of the PDUFA in 2022, the FDA took on the responsibility to enhance its use of RWE, placing particular emphasis on reporting. The FDA’s real-world evidence submission reporting framework builds upon its existing framework for evaluating potential uses of RWE, which has been in place since 2018.

FDA’s RWE Submission Reporting Framework

The FDA's new framework for RWE submission reporting includes three main criteria categories:

1. Type of Regulatory Submission:
   • Initial protocol submission (e.g., submission of an interventional study protocol to an IND)
   • Protocol amendment adding RWD to a study
   • New drug application (NDA) or biologics license application (BLA) (original or supplemental)
   • Final study report to satisfy a postmarketing requirement (PMR) or postmarketing commitment (PMC)
2. Role of Study:
   • Serve as an adequate and well-controlled study to support effectiveness
   • Serve as confirmatory evidence for approval based on a single adequate and well-controlled study
   • Provide safety data necessary for approval
   • Satisfy a PMR or PMC
3. Contribution of RWD:
   • Data for primary endpoint assessment
   • Data for external control arm
   • Safety data necessary for approval or to satisfy a PMR or PMC

Public Reporting of RWE Submissions

The FDA will publicly report on submissions that meet the criteria above in an aggregate and anonymized manner to protect proprietary information. Annual totals of RWE submissions for fiscal years 2024 through 2027 will be published on dedicated pages for the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER).

In 2023 fiscal year, the FDA received a total of 18 RWE submissions that met its reporting criteria. These included 10 protocol submissions, four new drug applications/biologics license applications submitted to CDER, and four protocol submissions to CBER. The FDA will be publishing annual totals for fiscal years 2024 through 2027 on separate pages dedicated to CDER and CBER RWE submissions.

Need More Information?

Contract Gardner Law if you need further assistance or have specific questions about how this impacts your submissions or RWE strategies.