FDA Issues Final Guidance for Industry, “Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease”

FDA Issues Final Guidance for Industry, “Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease”

Today the FDA published final guidance for industry entitled “Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease.” It describes the FDA’s current recommendations on how to group patients with different molecular alterations for eligibility in clinical trials and general approaches to evaluating the benefits and risks of targeted therapies within a clinically defined disease where some molecular alterations may occur at low frequencies.  This guidance finalizes the draft guidance issued in December 2017.
This guidance is intended to provide drug developers greater clarity on the FDA’s recommendations for researching and developing new targeted therapies, including enrolling patients with different underlying mutations, some of which may be rare, into clinical trials for targeted therapies when reasonable scientific evidence suggests the drug could be effective for patients with these genomic findings. Additionally, this guidance specifies the evidence needed to demonstrate effectiveness for a variety of molecular subsets within a particular disease, which could lead to more consistent development and approval of targeted therapies for patients who are likely to benefit from them.