FDA Considering Use of Off-Label Data for Drug Approvals

The U.S. Food and Drug Administration (FDA) signaled its willingness this month to use off-label information in approving new indications for drugs in the future. Currently, the FDA requires a controlled research study before the agency will approve drugs for specific uses. Doctors may then prescribe these drugs for off-label uses not approved by the FDA, but the data on these uses is considered insufficient to meet the agency’s regulatory standards.

Rajeshwari Sridhara, PhD, of the FDA’s Center for Drug Evaluation and Research, made the announcement at a conference on the use of “real-world data” collected outside of clinical trials. Dr. Sridhara suggested that the agency would need to have the ability to audit off-label data before it would consider using them for drug approvals, adding that the agency would likely consider the rarity and severity of a disease when making these judgments.

“I don’t think we are there yet, but that doesn’t mean we will never accept [it],” said Dr. Sridhara.

Source: STAT News, July 10, 2018.