GAIN Act, FDA stance only first steps to refilling antibiotic pipeline in U.S.
There is a consensus among the U.S. government, industry and the medical community that the failure of antibiotic development to keep pace with the evolution of bacterial pathogens constitutes a public health crisis, and that new regulatory policies and economic incentives are needed to create a continuous stream of new antibacterial products.
The Generating Antibiotics Incentives Now (GAIN) Act, which came into effect on Oct. 1, is a building block for creating some of the changes that will be needed to get antibiotic drug development back on track. And political changes have allowed FDA to begin shifting its risk-benefit calculus.
But much more is needed. Infectious disease specialists and biopharma drug hunters say FDA's positive attitude now must be turned into endpoints and trial designs that match clinical realities.
The agency has started down this path, crafting guidance that will make it easier to develop drugs for intra-abdominal infections, and telling companies that it has reconsidered bans on prior treatment with antibiotics in lung infection trials.
More work is needed to make endpoints in settings like pneumonia acquired in hospitals or associated with ventilator use consistent with clinical practice.
There is also an urgent need to create a regulatory pathway that will facilitate the development of new drugs to combat emerging, rare pathogens, especially those that are resistant to multiple antibiotics.
On both scores, Europe is ahead of the U.S.
But in both Europe and the U.S., new policies also are required to improve the economic incentives for bringing new antibiotic drugs to market by making it possible to lower development costs and enable attractive pricing.
The first indicator of whether FDA's new attitude is translating into practice could come on Nov. 29, when the Anti-Infectives Drugs Advisory Committee will review an NDA from Theravance Inc. for use of Vibativ telavancin in nosocomial pneumonia.
Priority to antibiotics
The GAIN Act, which was incorporated in the FDA Safety and Innovation Act that reauthorized prescription drug user fees, provides added exclusivity for antibiotics and earmarks antibiotics for Priority Review.
It also mandates the creation of a pathogen-focused antibacterial drug development pathway.
Moreover, GAIN may remove some of the impediments to financing antibiotic drug development. The ability to develop drugs for emerging multi-resistant pathogens based on the bugs they kill - regardless of the site of infection - should stimulate the creation of new drugs as FDA and sponsors agree on minimum efficacy datasets that can be obtained practically and rapidly.
But the most important effect of the new law is its signal that Congress has reversed its attitudes toward antibiotic drug development.
With GAIN, political pressure to limit the risk of adverse effects from antibiotics has been replaced by congressional calls to reinvigorate product development.
Attitudes at FDA mirror the evolution in the political environment.
"The whole tone of the conversation has gone from 'no' to 'go,'" said John Rex, VP and head of infection, global medicines development,