CDER Officials Discuss Expanding Use of RWD and RWE

Regulatory NewsRegulatory News | 15 August 2018 |  By 

Thanks to the 21st Century Cures Act, by the end of 2018, the US Food and Drug Administration (FDA) has to create a framework to evaluate the use of real world evidence (RWE) to support the approval of new indications for approved drugs or to satisfy post-approval study requirements. 

FDA defines RWE, according to a JAMA viewpoint published Monday, as “the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of RWD [real world data].”

Currently, FDA routinely uses RWD to provide evidence about drug safety, drawing on claims and pharmacy data from more than 100 million people in its Sentinel System.

Sentinel also has provided FDA with experience in using claims data, but claims data “will not capture many of the clinical end points used to support new indications for approved drugs. EHRs [electronic health records] can provide more granular clinical data, including laboratory results, imaging, and clinical assessments; however, EHR data are often unstructured and at times inconsistent due to entry variations across providers and health systems. This is not surprising because EHR data are not presently generated with research goals in mind,” the authors say.

But FDA is now focused on identifying additional areas in which RWD may be used to generate evidence of effectiveness.

“To explore how randomized trials can be conducted in real-world settings, the FDA is supporting the first randomized clinical trial in Sentinel, which to date has only been used to assess safety. The IMPACT-Afib trial will test an educational intervention to address the important public health problem of underuse of effective medications to reduce the risk of stroke in patients with atrial fibrillation, and it could serve as a proof of concept for future RWE trials,” the authors write.

“FDA also is funding a study to explore whether observational methods can be used to replicate the results of approximately 30 clinical trials designed to provide evidence about the effectiveness of a drug. This project will assist the FDA in understanding how observational methods can be applied to address questions involving drug effectiveness,” they add.

The CDER authors also make clear that if RWD and RWE are to be “effectively leveraged for public health purposes,” there will need to be more sharing among clinicians, patients, health care systems, pharmaceutical companies and regulators.

Real-World Evidence and Real-World Data for Evaluating Drug Safety and Effectiveness


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