Drug Companies, Health Groups Back IDSA Proposal

Limited Population Antibacterial Drug (LPAD) Legislation Would Expedite Development of Much-Needed Antibiotics

WASHINGTON, April 12, 2012 – Pharmaceutical manufacturers, medical societies, public health organizations and patient groups are lining up in support of a legislative initiative proposed by the Infectious Diseases Society of America (IDSA) that streamlines the development of antibiotics desperately needed to treat the most serious bacterial infections. The Limited Population Antibacterial Drug (LPAD) mechanism would benefit the increasing number of patients who face life-threatening infections yet have few if any treatment options, due to growing antibiotic resistance.  

Fourteen drug companies and many prominent public health groups have signed on to back the inclusion of LPAD in U.S. Food and Drug Administration (FDA) Prescription Drug User Fee Act (PDUFA) reauthorization, considered one of the must-pass bills in Congress this year. Much as the Orphan Drug program provides appropriate mechanisms for testing and developing drugs for rare diseases, LPAD would address an unmet medical need by speeding up development of antibiotics to treat patients who have serious infections for which therapeutic options are insufficient.

“We’re on the brink of a medical catastrophe – more and more patients are succumbing to drug-resistant infections for which we have no good treatment options, and yet the number of new antibacterial drugs being developed is plummeting,” said Thomas G. Slama, MD, president of IDSA. “We are engaged in ongoing discussion with congressional leaders to help them appreciate just how serious this problem is and that the wide support illustrates that LPAD is a viable part of the solution. We’re also encouraged that the FDA seems receptive to LPAD.”

The LPAD pathway would address resistant infections with few if any treatment options, such as those caused by certain strains of Acinetobacter, Pseudomonas and Klebsiella. These would be the type of drugs that might have helped David Ricci, 20, of Seattle, Washington, who lost part of his leg in a train accident and spent the next six months fighting one infection after another caused by highly drug-resistant bacteria – including Pseudomonas aeruginosa and Klebsiella pneumoniae – losing more and more of his leg each time the infection returned and had to be surgically removed. David nearly lost his life because the doctors had to give him last-resort antibiotics that damaged his internal organs. Although he’s now recovering, he continually worries that the infection will return and this time there will be no drugs to treat it. Read more about David’s story – and those of others who battled drug-resistant infections – here.

Dr. Slama notes that the few drug companies still investing in antibacterial research and development say they may focus on European, Asian and Latin American markets rather than the United States, due primarily to the lack of feasible and predictable regulatory approval pathways for these drugs. LPAD is designed to complement the economic incentives for antibiotic development Congress is considering for inclusion in PDUFA.

Standard FDA drug approval requires traditional, large-scale clinical trials, which are not feasible in the development of these antibiotics because of the limited numbers of patients in which such serious infections occur. Like the Orphan Drug program, the LPAD mechanism would allow for testing a drug’s safety and effectiveness in smaller, shorter and less expensive clinical trials. LPAD was formerly known as the Special Population Limited Medical Use (SPLMU) pathway, before the concept was narrowed to focus specifically on antibacterial drugs.

“Investment in the discovery and development of new antibiotics is not sufficient to meet the growing challenge of bacterial resistance,” said Kenneth Hillan, M.B., Ch.B., chief executive officer and chief medical officer of Achaogen, Inc., based in San Francisco, one of the companies that supports approval of LPAD. “A key reason that companies are less willing to invest in new antibiotics in spite of the increasing need is that the traditional development path is too expensive and unclear for drugs that target specific types of highly drug resistant bacterial infections. The proposed LPAD pathway would establish a clear and appropriate alternative for approval of new antibiotics aimed at the most critical unmet needs and a mechanism for ensuring that these critical new drugs are used appropriately.”

LPAD assures that approved drugs would be indicated for use in small, well-defined populations of patients for whom the drugs’ benefits have been shown to outweigh their risks. The mechanism will not be used to approve drugs to treat more common infections or those for which sufficient alternative options exist.

Drugs approved through LPAD would carry an LPAD logo and would come with an FDA notice to doctors and others that these products carry less-precise estimates of risk, meaning they should be used narrowly for the indicated population. Of importance, clinical decision-making will be left in physicians’ hands, but it is expected that LPAD drugs would rarely be used “off-label,” and, even then, only to treat life-threatening infections where insufficient treatment options exist. An added benefit is that the limited use would help slow the rate at which resistance to these drugs develops.

“This proposal would help reduce the size of studies that are needed, enabling sponsors to reduce the cost, speed the clinical development process and potentially accelerate new drug development,” said Prabhavathi Fernandes, Ph.D., president and chief executive officer of Cempra, a North Carolina-based company, which is dedicated to developing new antibiotics and supports LPAD. “LPAD will provide an important, innovative incentive that will help companies develop new antibiotics for the patients who most desperately need them.”

IDSA has long sounded the alarm regarding the burgeoning problem of antibiotic resistance. Unchecked, the crisis will lead to a future that resembles the days before miracle antibiotics existed: one in which people died of common infections and many medical interventions that we take for granted – from surgery to chemotherapy – will no longer be possible. Please visit IDSA’s website for more information, including:

• IDSA’s “10x’20 Initiative,” which aims to encourage the development of 10 new systemic antibiotics by 2020, visit http://www.idsociety.org/10x20/.  
• IDSA’s recommendations on how best to address antimicrobial resistance: “Combating Antimicrobial Resistance: Policy Recommendations to Save Lives.”
    

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