Gilead and Novartis Seek to Expand What FDA Considers as Real-World Data and Real-World Evidence

Just over two months ago, the US Food and Drug Administration (FDA) released draft guidance explaining to drugmakers what constitutes real-world data (RWD) and real-world evidence (RWE) and how to submit such data to the agency.

The draft guidance spells out what the agency considers to be an RWD source (i.e. electronic health record data, medical claims or billing data, etc.) and what relevant submissions may include RWE (i.e. single arm trials that use RWE as an external control, observational studies that generate RWE to support an efficacy supplement or observational studies using RWE to fulfill a postmarketing requirement, etc.).

But biopharma companies Gilead and Novartis are calling on FDA to expand on its sources of RWD and the scope of RWE, even as some physicians and experts have cautioned against using RWD and RWE for regulatory decisions or as a replacement for clinical trials.

Gilead, for instance, says FDA should include lab data, data from expanded access protocols and data derived from the delivery of health care as sources of RWD.

The California-based company called on FDA to provide examples of data that the agency would not consider to be suitable sources of RWD and RWE. Retrospective analyses of RWD collected outside a clinical trial should also be defined as a method for generating RWE, Gilead says.

“Study designs using observational data are complex and should be explained in detail, particularly matching and follow-up procedures,” Gilead says, and also calls on FDA to include examples of observational studies, such as cohort studies, case control studies and cross-sectional studies.

Novartis, meanwhile, requests that FDA include pharmacy claims for consideration as RWE for regulatory purposes.

“We believe the guidance should expand the scope of RWE for regulatory purposes,” the Basel-based company says. “Patient reported outcomes (PROs) and quality of life (QALY) are often collected and become an important part of the RWE for regulatory and reimbursement purposes.”

FDA should also clarify in the final guidance how RWE can be applied in single arm trials without an external/historical control arm, the comment notes.

Industry groups BIO and PhRMA also seek further clarifications from FDA.

For instance, both PhRMA and BIO call on FDA to harmonize the submission process for RWD and RWE across FDA’s Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research and Center for Devices and Radiological Health.

And BIO also calls on FDA to “periodically publish aggregate, deidentified data on the number of RWE submissions made, context-of-use, study design, data type, and therapeutic area. Similar metrics have been made available to track the implementation of the Breakthrough Therapies Program and have been found generally helpful by the research community.”

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