It’s no secret that antimicrobial resistance (AMR) is one of the most serious public health problems we face today: global in scope, more urgent with each passing day, and daunting when one considers the challenges that must be overcome to effectively address the crisis. Already, the Review on Antimicrobial Resistance, a report funded by the United Kingdom, estimates that at least 700,000 people die each year from drug-resistant infections, and that number will only grow if drug resistant pathogens continue to proliferate faster than our supply of new innovative products to treat them. The report estimates that by 2050, 10 million lives will be lost each year at a cumulative cost to the global economy of $100 trillion – unless corrective steps are taken.
Fortunately, there’s reason for hope. A number of new promising biological targets have been identified in recent years, and multiple new funding opportunities have also surfaced to provide support for AMR product development. For example, the CDC, BARDA, the Wellcome Trust, and many others are all committing resources through new initiatives, like the CARB-X Accelerator, to help advance novel, life-saving AMR products.
These promising new technologies and funding opportunities was the subject of a lively panel today at the 2017 BIO Investor Forum in San Francisco, California. Moderated by Dr. Gregory Frank, BIO’s Director of Infectious Disease Policy, the panelists included:
- Shelley Chu, MD, PhD, Partner, Abingworth;
- Ciara Kennedy, PhD, President and CEO, Amplyx Pharmaceuticals, Inc.;
- David Puerta, PhD, Chief Operating Officer and Vice President of Discovery, Forge Therapeutics;
- Ted Schroeder, President and CEO, Zavante Therapeutics; and
- Heather Shane, Executive Director, CLSI CARB-X, California Life Sciences Institute
Several panelists noted that GAIN Act, which provides an additional five years of regulatory exclusivity and priority FDA review for innovative antimicrobials that target qualifying pathogens (“Qualifying Infectious Disease Products”, or QIDPs). Zavante’s Ted Schroeder noted that without the provision, his company’s AMR program would not be feasible. Zavante is seeking to win marketing approval in the United States for an antibiotic that has long been available in other countries – not an easy or inexpensive process when one considers the rigorous safety and efficacy data that must be gathered to secure FDA approval.
“[t]he GAIN Act is the only reason we’re engaged in that… we’re developing intravenous fosfomycin, an extraordinarily broad spectrum antibiotic with particular activity against important multi-drug resistant bacteria… without some intervention on the part of Congress, this never would have seen the light of day in the United States,” Schroeder said.
Ciara Kennedy of Amplyx Pharmaceuticals also echoed the GAIN Act’s value:
“I think the GAIN Act really reinvigorated antimicrobial research across the antifungal and antibacterial space. People got back into the game because of the GAIN Act. Our indications are orphan indications, so for the first time, I can say to people, ‘we have orphan designation, we have QIDP [status], we’re a pediatric indication: we have the potential for a 12 and a half years regulatory exclusivity in the U.S.,’ and that’s unprecedented. That is a de-risking event in and of itself for investors” due to the uncertainty involved in relying on patents alone to recoup one’s investment.
Unlike regulatory exclusivity, which begins once a product is approved, patent exclusivity is a 20 year window which usually begins at the very outset of developing a new drug candidate, a process which frequently takes 10-15 years or even longer).
“When I can say with certainty that there’s 12 and a half years exclusivity because of GAIN, it just changes the conversation,” Kennedy added.
The need for more creative reimbursement models was also a major theme of the panel. Zavante’s Schroeder argued that delinking antibiotic reimbursement from Medicare’s “diagnostic related grouping” (DRG) is “the holy grail” in terms of changes that would make a positive impact. Under a DRG, a hospital is reimbursed a fixed amount based upon a patient’s diagnosis – meaning that prescribing a new, perhaps more expensive but clinically appropriate antibiotic could impact the hospital’s bottom line – creating pressure to prescribe cheaper therapies that may not do as good of a job fighting a patient’s infection (or worse, contribute to the development of drug resistant pathogens).
“It continues to drive healthcare providers to make the wrong choices, because they’re worried about how they’re going to get paid, and when an antibiotic is caught up in the overall DRG, that calculation… leads to sub-optimal decision-making. It’s very clear that the best therapy that you can give to a patient that has a deep-seated infection is aggressive therapy right from the beginning… the reimbursement scheme today doesn’t really drive naturally to that.”
Heather Shane of the California Life Sciences Institute agreed: “The reimbursement model right now, I agree, is at odds with the benefit of public health, because as you said, it drives the wrong decisions and also incentivizes the companies to promote use – sales and use – that’s how they get reimbursed – instead of clinical efficacy, cures, and reducing the likelihood of antimicrobial resistance.”
Changes to the reimbursement system for AMR products are clearly necessary in order to produce the system changes that will win the battle against drug resistant pathogens. Such changes were among the proposals put forth by BIO this summer in a white paper on models to better incentivize the antimicrobial pipeline, available here.