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Former FDA Commissioners Seek to Expand Use of RWE in Regulatory, Payment Decisions

Posted 21 August 2019 | By Michael Mezher 

Former FDA Commissioners Seek to Expand Use of RWE in Regulatory, Payment Decisions

A new report from the Bipartisan Policy Center makes recommendations to expand the use of real-world evidence (RWE) in regulatory and payment decision-making.
The report comes less than a year after the US Food and Drug Administration (FDA) released its Framework for FDA’s Real-World Evidence Program.
“Real-world evidence generated from the application of appropriate research methods and analysis of data derived from clinical, claims, and patient-facing software and systems can play a significant role in improving and modernizing both the drug evaluation and approval process and new value-based payment arrangements in the United States,” the report, authored by former Senate Majority Leader William Frist and former FDA Commissioners Robert Califf, Mark McClellan and Andrew von Eschenbach, says.
While the report claims “significant progress” has been made towards the use of RWE, especially with regulatory decision-making, it highlights areas for improvement and barriers to progress.
Specifically, the report lays out 12 policy recommendations to address what its authors see as the biggest barriers to, and opportunities for, advancing the use of RWE.
Among the recommendations are calls to fully fund FDA’s FY2020 budget request, which includes $60 million to develop a new “medical data enterprise” for human and animal drugs, biologics and medical devices.
The report also calls on FDA to continue to provide and expand upon its guidance “regarding the circumstances under which sponsors and the FDA may rely on real-world evidence to help support the approval of a new indication of an existing drug, as well as to help support or satisfy post-approval study requirements.”
According to the report, one of the biggest barriers to the use of RWE is limited access to data, as potential data sources are spread across a multitude of sources, such as physicians’ offices, hospitals, diagnostics labs and insurers. Even when electronic data can be accessed, interoperability poses a significant and costly challenge.
As such, the report calls for action to improve access to data sources and interoperability, including recommendations for the Office of the National Coordinator for Health Information Technology (ONC) to adopt previously proposed provisions for electronic health information export and application programming interface (API) support to make data contained in US Core Data for Interoperability (USCDI) for patients easier to access and exchange.
The report also calls for FDA, ONC and the Centers for Medicare and Medicaid Services (CMS) to collaborate on HL7 Fast Healthcare Interoperability Resources (FHIR) implementation guides and for CMS to expand access to Medicare beneficiary data.
Other recommendations include steps to improve the reliability and relevance of real-world data; leveraging patient generated data; expanding the use of artificial intelligence; and addressing legal and regulatory barriers cited as hurdles to value-based payment models.
Expanding the Use of Real-World Evidence in Regulatory and Value-Based Payment Decision-Making for Drugs and Biologics


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