Draft Guidance: Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease

The FDA is announcing the availability of a draft guidance for industry entitled Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease. The purpose of this guidance is to describe 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 therapeutics within a clinically defined disease where some molecular alterations may occur at low frequencies. Submit either electronic or written comments on the draft guidance within 60 days of the publication of the Federal Register Notice to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance. Read more about new FDA efforts to support more efficient development of targeted therapies at https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm589248.htm.