FDA Serious Bacterial Diseases

The FDA has issued revised draft guidance to assist sponsors in the clinical development of new antibacterial drugs.

The guidance specifically explains the agency’s current thinking about possible development programmes and clinical trial designs for antibacterial drugs to treat serious bacterial diseases in patients with an unmet medical need, including patients with a serious bacterial disease for which effective drugs are limited or do not exist.

Antibacterial drug resistance continues to be a public health concern and has led to an increasing number of patients with serious bacterial diseases who may not respond to drugs which are currently available.

However, conducting clinical trials to evaluate antibacterial drugs to treat those diseases can be challenging for a number of reasons. Speedy treatment is of the essence to save lives, but trial therapy effects may then be obscured by already-administered medication; the severity of the illness in patients may make it difficult or impossible to obtain informed consent or conduct other administrative procedures. Determining the exact cause of a bacterial disease can be uncertain and it can overlap with the potential need for concomitant antibacterial drug therapies which can make assessment of the efficacy of the investigational drug difficult.

Despite these challenges, however, there is still an urgent need for the development of new antibacterial drugs. Given that, the FDA feels that sponsors should be aware of the recognised need for flexibility in meeting effectiveness requirements of this research while still guaranteeing safety and effectiveness.

One example of what’s outlined in the new guidance is the types of antibacterial drugs which may be appropriate for a more flexible development programme. It also covers, in detail, a comprehensive number of other questions such as potentially using drugs which treat a single species of bacteria for a flexible development programme, enrolling patients who have infections at different body sites in the same clinical trial and the clinical trial design considerations in a streamlined development programme for an antibacterial drug for the treatment of patients with serious bacterial diseases and an unmet medical need. Other areas of the paper deal with special considerations for product labelling, greater drug toxicity and statistical approaches or randomisation strategies.