By Clinical Research News Staff

May 17, 2019 | Last week, FDA released a draft guidance on submitting documents using real-world data and real-world evidence to FDA for drugs and biologics. The guidance is, “intended to encourage sponsors and applicants who are using real-world data (RWD) to generate real-world evidence (RWE) as part of a regulatory submission to FDA to provide information on their use of RWE in a simple, uniform format,” according to the document. It applies to submissions for investigational new drug applications, new drug applications, and biologics license application that contain RWE used to support regulatory decisions regarding safety and/or effectiveness.

In 2016, the 21^st Century Cures Act mandated that FDA explore the use of real-world evidence in assessing drug approvals. In a 2018 article published in the Journal of the American Medical Association (doi:10.1001/jama.2017.9991), Janet Woodcock and co-authors listed several ways real-world data can improve efficiency and cost-savings in clinical trials. “Real-world data may be used to aid in the design of a clinical trial by assisting in the selection of study sites that are more likely to enroll study participants, provide a basis for power calculations, provide a prior for a Bayesian statistical analysis, provide an external control group, and guide enrichment. Real-world data may also be used during the conduct of a trial to reduce duplication of data input such as baseline medical history, automated adverse event reporting, and end-point ascertainment.”

Thus the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) are tracking the use of RWE in IND submissions for randomized trials, single arm trials using RWE as an external control, or observational studies that generate RWE for either an efficacy supplement or for postmarketing safety or efficacy studies.

The Centers do not plan to track RWE used natural history studies, feasibility studies, or hypothesis-generating studies.

In tracking how and when RWE is used, sponsors are encouraged to include a cover letter, explaining why they used RWE, the type of study, and what types of real-world data sources were used to generate RWE. FDA lists possible data sources including data from EHRs, medical claims or billing data, disease registry data, and other sources that can inform on health status including patient-generated data and mobile devices.

The draft guidance is open for comment for 60 days.