This guidance is intended to assist sponsors in the clinical development of new antibacterial drugs. Specifically, the guidance explains the FDA’s current thinking about possible streamlined development programs and clinical trial designs for antibacterial drugs to treat serious bacterial diseases in patients with an unmet medical need, including patients who have a serious bacterial disease for which effective antibacterial drugs are limited or lacking. Antibacterial drugs that are pathogen-focused can be developed for the treatment of serious bacterial diseases in patients who have an unmet medical need.

We note that section 3042 of the 21st Century Cures Act (Public Law 114-255), which establishes a limited population pathway for certain antibacterial and antifungal drugs (LPAD) that are intended to treat a serious or life-threatening infection in a limited population of patients with unmet needs, was enacted shortly before publication of this guidance. Some antibacterial drugs that are candidates for a streamlined development program may also be candidates for LPAD. We intend to issue separate guidance regarding LPAD. Sponsors are encouraged to
discuss proposed approaches with the Division of Anti-Infective Products.

This guidance does not contain discussion of the general issues of statistical analysis or clinical trial design. Those topics are addressed in the ICH guidances for industry E9 Statistical Principles for Clinical Trials and E10 Choice of Control Group and Related Issues in Clinical Trials, respectively.

Posted on the FDA website on 1 August 2017