To make drug development and evaluation more efficient, the U.S. Food and Drug Administration (FDA) issued draft guidance on the use of minimal residual disease (MRD) in the regulatory evaluation of treatments for hematologic malignancies. The agency’s guidance is based on feedback from workshops with sponsors, researchers, and regulators, as well as an analysis of marketing applications that showed inconsistent quality of MRD data.
“We’re focused on making the process of generating pre-clinical and clinical evidence required for making risk-based regulatory decisions more modern, more scientifically rigorous, and more efficient,” FDA Commissioner Scott Gottlieb, MD, said in a statement announcing the new draft guidance. “[This document will] provide drug developers greater clarity and direction as they pursue the next generation of therapies and treatments for patients.”
The new guidance, which is open for public comment, advised sponsors to design trials that address two main questions: “Is MRD as assessed (sample, timing, threshold, etc.) a clinically valid biomarker for the context of use (disease, disease status, type of therapy, etc.)?” and “Is the MRD assay used (or to be used) in the clinical trial analytically valid for the range of results that are important to the trial?”
The guidance document also includes disease-specific considerations for the use of MRD in treatment evaluations.
Source: FDA news release, October 15, 2018.