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New BIO White Paper: Incorporating Real-World Evidence Within the Label of an FDA-Approved Drug

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Guest Author
June 25, 2019

In today’s increasingly connected world, health care researchers have access to previously unimaginable troves of data about the effects of healthcare interventions, including medicines. New technologies are allowing researchers to use so-called “big data” to investigate ways to better diagnose disease, predict which patients will benefit from which therapies, and observe the effects of medicines on a broader population than is possible during clinical trials. Such observations can often suggest potential new benefits, indications and patient populations for medicines that were not apparent or studied during the initial clinical trials conducted to approve a drug. While the FDA has long used such data in its regulatory oversight, the era of big data presents new challenges and opportunities to ensure that real-world evidence (RWE) and the underlying real-world data (RWD) are optimally utilized in a scientifically rigorous way for the benefit of patients.

As policy makers and regulators consider how best to incorporate the use of RWE and RWD for changes to the label of an approved product, BIO has prepared a white paper to offer the perspectives of BIO member companies. The paper, Incorporating Real-World Evidence Within the Label of an FDA-Approved Drug: Perspectives from BIO Membership, may be downloaded here.

BIO believes that RWE studies can augment insights gained from traditional randomized clinical trials about the effectiveness of therapeutics. This application of RWE builds on the long-standing FDA use of RWD in safety assessments, including Sentinel. While RWE has potential uses in a variety of regulatory contexts, BIO identified label expansion as a priority issue that can benefit from greater regulatory clarity. This topic is timely; activities to further RWE use, including the issuance of draft guidance and a regulatory framework, are explicitly listed as FDA deliverables under the 21st Century Cures Act and Prescription Drug User Fee Act (PDUFA) VI. We hope this document is useful in communicating the perspectives of industry, and to inform policy makers regarding future use of RWE.