RWE Submissions: FDA Drafts Guidance

Regulatory NewsRegulatory News | 08 May 2019 |  By 

With an eye toward better tracking the use of real-world data (RWD) to generate real-world evidence (RWE), the US Food and Drug Administration (FDA) on Wednesday drafted guidance to help sponsors provide information to the agency on their use of RWD and RWE in a uniform format.

The uniform format is meant to help the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) track certain types of submissions using RWE under an investigational new drug application (IND), new drug application (NDA) or biologics license application (BLA).

Relevant submissions may include RWE used to support study objectives, such as: “IND submissions for randomized clinical trials that use RWD to capture clinical outcomes or safety data, including pragmatic and large simple trials; New protocols for single arm trials that use RWE as an external control; Observational studies that generate RWE intended to help to support an efficacy supplement; Clinical trials or observational studies using RWE to fulfill a postmarketing requirement to further evaluate safety or effectiveness and support a regulatory decision.”

But FDA also said in the 5-page draft that it does not intend to track RWE submissions that are not tied to a specific product or are not being used to support a regulatory decision regarding safety and/or effectiveness.

Such submissions that do not have to be identified as containing RWE include: “Natural history studies for development of a clinical outcome assessment or biomarker; Feasibility studies using RWE; Studies using RWD to perform exploratory analyses and generate hypotheses.”

In the cover letter accompanying a submission, FDA explains how the sponsor or applicant should identify the submission as containing RWE by including the following information: Purpose of using RWE as part of a regulatory submission (e.g. as part of a new product approval, to provide evidence to support a labeling change or as part of a postmarket commitment); study design using RWE (e.g. randomized or single-arm trial); RWD source(s) used to generate RWE.

Such RWD source(s) can include: “Data derived from EHRs; Medical claims and/or billing data; Product and/or disease registry data; Other data sources that can inform on health status (e.g., data collected from mobile technologies, patient-generated data).”

FDA explained that it will use this information for internal tracking purposes only.

The draft guidance also includes an appendix with a sample presentation of the cover letter for submissions including RWE.

Submitting Documents Using Real-World Data and Real-World Evidence to FDA for Drugs and Biologics: Draft Guidance for Industry


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