In the State of the Union address, President Barack Obama recognized the need to “stay ahead of drug-resistant bacteria” and that developing therapies to fight these threats is an opportunity for American innovation and discovery.
The threat of drug-resistant bacteria is real, and the need for antibiotic development clear.
Today, patients lack treatment for increasingly common and life-threatening infections, such as those caused by carbapenem-resistant Enterobacteriaceae, or CRE, which is a family of bacteria that includes Escherichia coli (E. coli). The Centers for Disease Control and Prevention recently classified CRE as an urgent threat, calling it “nightmare bacteria” because it is resistant to our strongest antibiotics and is rapidly spreading across the United States.
Ideally, the pipeline of antibiotics would be primed with a wide variety of potential treatments meant to address today's resistant bacteria and to arm us for tomorrow's threats. In reality, however, few novel antibiotics1 have reached the market over the past three decades, limiting the options to treat patients who have infections caused by resistant bacteria. The decrease can be attributed in part to the significant difficulties faced by antibiotic developers: At best, just 1 in 5 drugs that make it into initial testing in humans—or Phase 1 clinical trials—will ultimately be approved for use. It is particularly difficult to study drugs for highly resistant bacterial infections because of the small number of patients in which they occur. Those are daunting odds for any company considering the time, effort, and often huge expense that can accompany the development of any new medicine, and particularly challenging for small companies. Pew's research on antibiotic development to be released next month shows that the vast majority of products in development today are being studied by small companies, not the large pharmaceutical firms that once dominated this field.
Pew is working with members of Congress on the Antibiotic Development to Advance Patient Treatment, or ADAPT, Act, bipartisan legislation directed at making sure that the pipeline is filled with an adequate number of antibiotics to address patients' unmet needs and that these drugs have a clear path toward approval.
The ADAPT Act would direct the Food and Drug Administration to approve new antibiotics that would be studied for use in fewer patients than required in conventional studies. This would help lower industry's development costs and make clinical trials more feasible for drug developers. The result would be antibiotics intended for use in patients with few or no other options.
Importantly, the information doctors receive with the antibiotics approved under ADAPT would explicitly state that the drugs were approved only for a limited population. While this is a step in the right direction, the drug indication included in the packaging needs to be distinctive and informative enough to guide prescribers' decisions. Pew is urging Congress to require a prominent visual cue on ADAPT antibiotics to make it immediately clear that the drugs were tested for a specific, limited population and should be used only in patients with few or no other options. Because antibiotic use drives resistance, prudent use of the critically needed antibiotics developed under the ADAPT pathway would help preserve their effectiveness.
ADAPT builds on the success of the Generating Antibiotic Incentives Now, or GAIN, Act, which was signed by President Obama in 2012. GAIN extends the period during which antibiotics that treat serious or life-threatening infections can be sold without generic competition. This additional period of “exclusivity” increases the potential for profits from new antibiotics by giving the companies that developed them more time to recoup their investment costs. GAIN also speeds the development and review of qualified antibiotics. At least 16 new antibiotics have benefited under this important law.
ADAPT is an important step toward filling the antibiotic pipeline and making sure the drugs being developed have the greatest chance of reaching the market.
It's an investment in the future of health care that simply cannot wait.
Nicole Mahoney is a senior officer with the antibiotics and innovation project at The Pew Charitable Trusts.
1John Powers, M.D., “Scientific and Regulatory Issues in Studying Experimental Antibiotics; Doing Better With More Efficient Studies,” presented at the Pew-IDSA-PhRMA conference Reviving the Pipeline of Life-Saving Antibiotics: Exploring Solutions to Spur Innovation, Washington, Sept. 22, 2011.