What to Know About the FDA’s Real World Evidence Framework

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This framework is for evaluating the potential use of Real World Evidence (RWE) to help support the approval of a new indication for drugs and biological products already approved or to help support or satisfy drug post-approval study requirements. This framework is also intended for application to biological products licensed.

What studies could be covered by the RWE program?

The FDA will consider the following trial designs to have the potential to generate RWE.

  • Hybrid design clinical trials. In a hybrid design clinical trial, certain elements could rely on the collection and analysis of RWD extracted from medical claims, EHRs, or laboratory and pharmacy databases. Hybrid trials could use RWD for one clinical outcome, (e.g. hospitalization, death), while other elements were more traditional (e.g. specified entry criteria, monitoring and collection of additional study endpoints by dedicated study personnel).
  • Pragmatic clinical trial. The pragmatic clinical trials can include some elements that more closely resemble routine clinical practice. The pragmatic trials often rely on RWD and have the potential to generate RWE.
  • Observational Study. For purposes of this framework, observational studies are non-interventional clinical study designs. FDA considers a retrospective observational study to be one in which the study identifies the population and determines the exposure/treatment from historical data (i.e. data generated prior to the initiation of the study). The variables and outcomes of interest are determined at the time the study is designed. In a prospective observational study, the population of interest is identified at the start of the study, and exposure/treatment and outcome data are collected from that point forward.

 

Observational clinical studies might be another way to generate RWE that is relevant to effectiveness determinations. Therefore, the RWE program will also consider the evaluation of observational clinical studies using RWD to support product effectiveness determinations.

 

When generating RWE, an attention should be paid to evaluating that RWE in the context of regulatory decision-making depends not only on the evaluation of the methodologies used to generate the evidence but also on the reliability and relevance of the underlying RWD.

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