10/04/2022
Photo by ThisisEngineering RAEng on Unsplash
The US Food and Drug Administration released a final guidance “Submitting Documents Using Real-World Data and Real-World Evidence to FDA for Drug and Biological Products” in September 2022. It describes what needs to be included in the cover letter of a document submission that contains real world evidence or real world data so the Agency can more easily track them.
Background
This guidance is published in line with the 21st Century Cures Act (Cures Act). Part of the Cures Act is related to the use of real-world evidence (RWE) to help support regulatory decision making. According to the FDA, RWE is “data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than traditional clinical trials” and the “clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of real word data (RWD).” RWD are “data relating to patient health status and/or the delivery of health care that are routinely collected from a variety of sources” which comes from medical billing claims, electronic health records (EHR), clinical registries, patient-generated data including from in-home use, and other sources. RWE provides valuable information on the safety and efficacy of a drug, biologic, or device when it is used as intended, outside the constraints of a clinical trial. The Cures Act requires the Agency to track submissions that include RWD or RWE.
To make it clear to the Agency that the document submission contains RWD or RWE, the cover letter should contain the:
Purpose(s) for the RWD or RWE:
To support safety and/or effectiveness
To support labeling changes
To support or satisfy a postmarketing requirement (PMR) or postmarketing commitment (PMC)
Type of study design that use(s) RWD (e.g., randomized controlled)
Type of RWD source(s) used for RWE (e.g., electronic medical record)
An appendix in the draft guidance provides an example of a submission cover letter that includes RWD/RWE information in a checklist format.
For more information on the FDA’s framework for RWE, visit our blog “FDA Framework for Use of Real-World Evidence.”
In September 2021, the FDA published a draft guidance “Real-World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision Making for Drug and Biological Products.” Read our blog HERE for more information.
In October 2021, the FDA published a draft guidance “Data Standards for Drug and Biological Product Submissions Containing Real-World Data.” Read our blog HERE for more information.
In December 2021, the FDA published a draft guidance “Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products.” Read our blog HERE for more information.
If you are preparing a regulatory submission and need assistance with knowing how to apply these methods, Clinical Pathways can provide training. Contact us at info@clinicalpathwaysresearch.com.
- The Clinical Pathways Team
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