FDA Clarifies Guidance for Drugmakers on Using Real-World Data

Data collected from patients may be used as evidence on how drugs that have received emergency use authorizations from the FDA perform in health care settings, according to agency guidance published Wednesday.

The document, which finalizes draft recommendations first proposed in December 2021, applies specifically to real-world evidence intended to help support the approval of a new indication for a drug or biological product already approved by the Food and Drug Administration or to help support post-approval study requirements.

The guidance fulfills a requirement of the 21st Century Cures Act, which in part called for incorporating a wider variety of data sources to accelerate the approval of medical products and bring potentially lifesaving treatments to patients in a more timely manner.

The FDA has made improving guidance on the use real-world data within drug applications a major priority. Real-world data includes information related to a patient’s health status or the delivery of health care, such as data from electronic health records and medical claims.

The finalized guidance clarifies that third-party health providers can use existing regulatory pathways to submit patient-level data to FDA when a product’s manufacturer can’t submit this data to FDA through traditional channels. For example, the third party can choose to open either a pre-investigational new drug application or a Type V drug master file to submit relevant data to the agency.