The FDA published two draft guidances for comment this week: “Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease” and “Standards Development and the Use of Standards in Regulatory Submissions Reviewed in the Center for Biologics Evaluation and Research.”
The guidance on targeted therapies is intended to provide guidance on the type and quantity of evidence that can demonstrate efficacy across molecular subsets within a disease, particularly where molecular alterations occur at frequencies low enough that it is not feasible to conduct a clinical trial limited to that alteration. Specifically, the guidance describes (1) the FDA’s current recommendations on how to group patients with different molecular alterations in clinical trials; and (2) 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. The comment period for this draft guidance closes February 16, 2018.
The guidance on standards development provides recommendations on the use of standards in product development and FDA’s policy on accepting standards used in regulatory submissions. The comment period for this draft guidance closes March 19, 2018.