USA – Hematologic Malignancies: Regulatory Considerations for Use of Minimal Residual Disease in Development of Drug and Biological Products for Treatment

This guidance is intended to help sponsors planning to use minimal residual disease (MRD) as a biomarker in clinical trials conducted under an investigational new drug application (IND) or to support marketing approval of drugs and biological products2 20 for treating specific hematologic malignancies.

The use of MRD as a biomarker in drug development is distinct from FDA’s requirement for
investigating, clearing, or approving an in vitro diagnostic device for clinical use in measuring MRD. Manufacturers interested in developing a specific MRD assay for clinical use should consult the Office of In Vitro Diagnostics and Radiological Health in the Center for Devices and Radiological Health (CDRH).

In general, FDA’s guidance documents do not establish legally enforceable responsibilities.
Instead, guidances describe the Agency’s current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in Agency guidances means that something is suggested or recommended, but not required…