There are several FDA programs intended to facilitate and expedite development and review of new drugs to address unmet medical need in the treatment of serious or life-threatening bacterial infections. New expedited approval pathways for antibiotics may have opened the door for future drug approvals based on surrogate endpoints. There are some prevailing concerns whether the expedited drug approval pathways for antibiotics could potentially lower scientific standards and increase the number of drugs on the market which are ineffective or harmful.

For acute bacterial infection therapies, some key opinion leaders have pointed out that that trials based on surrogate endpoints may be less informative and the direct benefits of new antimicrobial drugs on patient symptoms and clinical outcomes are uncertain when assessments rely on surrogate endpoints. This uncertainty is compounded by the design of non-inferiority studies, which establishes efficacy indirectly by using historical evidence for efficacy in absence of appropriate therapy (eg, placebo response).

In this roundtable session, we hope to revisit types of endpoints and study designs for future antibiotic trials. Could a better endpoint and an optimal study design of a superiority trial be used to assess a new treatment for a drug-resistant bacterial infection? Other considerations may include emergence of antibiotic trial networks and the need for earlier larger incentives to meet the 2020 antibiotic goals.