Congress will consider a new drug approval pathway to encourage development of critically needed, lifesaving antibiotics as part of an overall strategy for addressing the public health crisis of antibiotic resistance. The Infectious Diseases Society of America (IDSA) proposed the new pathway, called the “Special Population Limited Medical Use (SPLMU)”* mechanism, to provide an important new approval option for companies interested in developing drugs to treat patients with serious infections where few or no treatment options exist.
IDSA submitted the proposal to the House Energy and Commerce Committee Subcommittee on Health during a March 8 hearing on the U.S. Food and Drug Administration (FDA) Prescription Drug User Fee Act (PDUFA) reauthorization legislation. PDUFA is considered one of the few must-pass bills this year in Congress. During the hearing, lawmakers will consider whether to incorporate within PDUFA pending legislation known as the Generating Antibiotic Incentives Now (GAIN Act), which proposes incentives to address the dry antibiotics’ research and development (R&D) pipeline.
“Antimicrobial resistance is unequivocally one of the world’s greatest public health threats, and we’ve been sounding the alarm about it – and the lack of new antibiotics in development – for years,” said Brad Spellberg, MD, co-chair of IDSA’s Antimicrobial Availability Task Force. “This new mechanism provides the opportunity to address this public health crisis while there is still time to fix it.”
Economic, scientific, and regulatory obstacles have led to a climate that makes antibiotic R&D in the U.S. more difficult, and which has had dire results: In 1990, there were nearly 20 pharmaceutical companies with large antibiotic R&D programs. Today, there are only two or three.
The SPLMU mechanism streamlines the approval pathway and enables pharmaceutical companies to study SPLMU drugs in far fewer patients than currently is required, more rapidly, and at significantly less cost – because they are intended for more targeted use within a special population of patients who lack other treatment options. SPLMU designation reserves a drug for use in specific populations in which the benefits outweigh the risks, and encourages prudent use of the drug to slow the rate of resistance.
“The proposed SPLMU drug approval mechanism will bring critically needed innovation to the anti-infective pipeline, encouraging antibiotic development and lowering the hurdles that need to be cleared to get these critically needed drugs to patients who desperately need them,” said Robert Guidos, vice president of public policy and government relations at IDSA. “In addition, we’ve designed it to foster antimicrobial stewardship, so these drugs will be used only when appropriate, extending their lifesaving power.”
Without immediate action to address antimicrobial resistance and restore the drug development pipeline, we will soon face a future that resembles the days before these miracle drugs were developed: one in which people died of common infections, and where many medical interventions that we take for granted – including care for premature infants, surgery, chemotherapy and organ transplantation – become impossible.
Eleven-year-old Addie Rerecich, of Tucson, Ariz., can testify that this is already a too-close reality. In 2011, Addie went to the doctor with pain in her hip and found out her body was wracked with life-threatening strains of Staphylococcus aureus, Escherichia coli (E. coli), and other resistant infections. The bacteria wreaked havoc on Addie’s body, causing sepsis, pneumonia and eventually leading to a pulmonary embolism and a life-saving lung transplant. In addition, during the five months she was in the hospital, Addie was put on colistin, a highly-toxic last-resort drug that can cause organ failure, because it was the only antibiotic available that was effective against her infections. Read more about Addie’s story, and many others, here: http://www.idsociety.org/Patient_Stories/
In addition to proposing the SPLMU mechanism, the IDSA testimony also recommends other public policy strategies – within the context of the GAIN Act and PDUFA – that focus on ways to reduce the economic disincentives that have contributed to a market failure in antibiotic R&D. These include various push and pull economic incentives and support for new diagnostics development and antimicrobial stewardship programs. To see the details of the full statement, see “Promoting Anti-Infective Development and Antimicrobial Stewardship through PDUFA Reauthorization.”
Last month, a group of 50 organizations representing patients, health care providers, health systems, veterans, women’s health, children’s health, seniors, and other key stakeholders sent a letter to House and Senate leaders urging them to address the serious and growing problems of antimicrobial resistance and the dry antibiotic R&D pipeline as part of PDUFA.
* Editor's Note (04/05/2012): The proposed new drug approval pathway is now known as the Limited Population Antibacterial Drug (LPAD) approval mechanism.
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