Real-World Evidence Regulatory Guidance

FDA has published a guidance, Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products, to discuss the applicability of the IND regulations to various clinical study designs that use real-world data (RWD). The document also clarifies the agency’s expectations concerning clinical studies using RWD submitted to FDA in support of a regulatory decision on the effectiveness and safety of a drug, as part of an NDA or BLA. The guidance focuses primarily on clinical study designs that are non-interventional.

FDA says issuing the guidance partially fulfills a Federal Food, Drug, and Cosmetic Act (FFDCA) mandate to issue a guidance about using real-world evidence to help support approval of a new indication for a drug already approved under Section 505(c) of the FFDCA or to help support post-approval study requirements.