Draft guidance released by the US Food and Drug Administration (FDA) aims to support the development of treatments that focus on underlying genetic mutations that cause or contribute to disease.
The draft guidance offers help for companies developing certain targeted therapies on the basis of targeting a molecular subtype that is common across different phenotypes, rather than solely on an individual disease. The purpose of this guidance is to describe:
(1) the FDA’s current recommendations on how to group patients with different molecular alterations for eligibility in clinical trials; and
(2) approaches to evaluating the benefits and risks of targeted therapies within a clinically defined disease where some molecular alterations may occur at low frequencies.
The FDA states that “for the purpose of this guidance low-frequency molecular alterations are those that occur at frequencies low enough that enrolling a sufficient number of patients to conduct a clinical trial limited to the specific molecular alteration of interest is not feasible or practical”.
The draft guidance includes information regarding how to identify patients for inclusion in clinical trials, what findings can be generalisable, benefit and risk determination and labelling, and refining the target population after initial approval.
To view the FDA guidance on developing targeted therapies in low-frequency molecular subsets of a disease, please click here.