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FDA Bets on Real-World Evidence


The proposed framework could provide patients and providers answers quicker, according to FDA Commissioner, Scott Gottlieb, M.D.

In a push to advance data quality, the U.S. Food and Drug Administration (FDA) today proposed a new framework to advance the use of real-world evidence across drug and biologic development efforts.

The framework includes strategies for filling in the gaps in other sources of real-world data, an effort that could include the use of mobile technologies, electronic patient-reported outcome tools, wearables and biosensors.

It also outlines real-world evidence-related efforts to explore the potential for using this data to support approval of new indications for approved drugs or to support or satisfy post-approval study requirements.

>> READ: FDA Announces New Device Guidance, Enhanced Patient Input Program

“This framework is aimed at leveraging information gathered from patients and the medical community to inform and shape the FDA’s decisions across our drug and biologic development efforts,” FDA Commissioner Scott Gottlieb, M.D., said in a statement announcing the move.

The FDA will evaluate the role of observational studies in contributing to the evidence for demonstrating drug and biologic product efficacy. Additionally, the regulator will need to develop new guidance on designing clinical trials that include pragmatic design elements as a tool for generating evidence of effectiveness for regulatory decisions.

Real-world data generated from electronic health records, medical claims, product and disease registries, laboratory test results and technology paired with mobile devices can offer new opportunities to create evidence and better understand clinical outcomes, Gottlieb said.

Such data will ultimately be used to develop stronger real-world evidence to guide FDA decision making.

“… The collective evaluation of these data sources has the potential to inform clinical decision making by patients and providers, develop new hypothesis for further testing of new products to drive continued innovation and inform us about the performance of medical products,” Gottlieb said.

Real-world evidence provides a potential source of information that can complement, augment and expand the way medical products are used.

Such information can also aid FDA processes. The use of real-world data and evidence derived from the FDA’s Sentinel system eliminated the need for post-marketing studies on nine potential safety issues involving five products. Sentinel has also been used to understand patterns of opioid use.

“These tools will gradually allow us to shift some studies and data collection to the point of care, making the collection of data and the development of actionable evidence more efficient,” Gottlieb said.

For example, new drug applications are being reviewed in the oncology setting, and real-world evidence is helping to inform evaluations.

“By using (real-world data) and (real-world evidence), we may be able to provide patients and providers with important answers sooner — identifying a broader range of safety signals and following up on them more efficiently,” Gottlieb said.

The FDA launched the MyStudies app for capturing real-world patient data. Real-world evidence and the tool could improve the quality of the regulator’s decision making and safety monitoring regarding medications.

While healthcare insiders like the deal, there are still some concerns about how the data will be used.

“As we incorporate these types of novel data for evidence generation, considerations for individual consent and privacy need to be held in the same regard as data accuracy and consistency," Evidation’s CEO, Deborah Kilpatrick, Ph.D., said in a statement.

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