RWE Submissions: FDA Drafts Guidance

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.

To Read the Complete Article at RAPS, Click Here

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutics areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.