USFDA’s Real-World Data: Assessing e-Health Records & Med Claims Data To Support Regulatory Decision

On 28-September, 2021 USFDA released the guidance “Real-World Data: Assessing Electronic Health Records and Medical Claims Data To Support Regulatory Decision-Making for Drug and Biological Products”.

FDA created a framework for a program to evaluate the potential use of real-world evidence (RWE) to help support the approval of a new indication for a drug already approved under section 505(c) of the FD&C Act or to help to support or satisfy postapproval study requirements (RWE Program).

FDA is issuing this guidance as part of its RWE Program and to satisfy, in part, the mandate under section 505F of the FD&C Act to issue guidance about the use of RWE in regulatory decision-making. The RWE Program will cover clinical studies that use real-world data (RWD) sources, such as information from routine clinical practice, to derive RWE.

For the purposes of this guidance, FDA defines RWD and RWE as follows:

• RWD is data relating to patient health status or the delivery of health care routinely collected from a variety of sources.

• RWE is the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from the analysis of RWD.

This guidance is intended to provide sponsors, researchers, and other interested stakeholders with considerations when proposing to use electronic health records (EHRs) or medical claims data in clinical studies to support a regulatory decision on effectiveness or safety.

Also, FDA released NEW Guidance on Drugs for non-TB pulmonary disease.

The purpose of this guidance is to assist sponsors in the clinical development of drugs for the treatment of nontuberculous mycobacterial pulmonary disease (NTM-PD) caused by Mycobacterium avium complex (MAC).

Specifically, this guidance addresses the FDA’s current thinking regarding clinical trial design issues, choice of study population, and endpoints for the treatment of naïve and refractory NTM-PD caused by MAC.

The design of clinical trials of new drugs for the treatment of NTM-PD was discussed during an FDA public workshop.