NATIONAL HARBOR, Maryland — The growing threat of antimicrobial resistance, coupled with the dwindling pipeline of available, effective antibiotics to combat infections, was examined in a session presented at the 2021 World Orphan Drug Congress.

Antimicrobial resistance (AMR) occurs when bacteria, fungi, and other infectious organisms evolve in response to exposure to antibiotic treatment. Over time, these changes render the organisms no longer susceptible to treatment with typical first-line antibiotics. Sometimes, these organisms may become resistant to multiple drug regimens, earning the label “multidrug-resistant.”

The AMR statistics are sobering. More than 2.8 million Americans develop AMR infections per year, and 35,000 deaths occur due to these drug-resistant organisms. Globally, AMR accounts for a death toll of approximately 700,000 people annually.

Amanda Jezek, senior vice president of public policy and government relations for the Infectious Diseases Society of America, said at the session that these numbers may underrepresent the enormity of the problem and that more surveillance is required for accurate approximations. In particular, she mentioned the need for more research analyzing the health disparities surrounding AMR, especially in minority groups that may not have easy access to quality care.

Michael Boyle, MD, president and CEO of the Cystic Fibrosis Foundation, regards AMR as a “top public health concern.” He cautions that individuals with underlying disease mechanisms are more susceptible to developing AMR infections. This has been apparent during the coronavirus disease 2019 (COVID-19) pandemic, as patients with COVID-19 who are placed on ventilators are more predisposed to developing AMR infections.

The risks that AMR pose to individuals with rare diseases are dangerous and potentially life-threatening. In particular, Dr. Boyle explained how individuals with cystic fibrosis are prone to developing chronic upper respiratory tract infections due to the thick mucus secreted as part of the disease process. Drug-resistant bacteria are more likely to become trapped within this mucus, causing an AMR infection against which doctors may not have the necessary pharmaceutical weapons to fight.

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One might ask why there is such a shortage of new anti-infective drugs in the pipeline. The answer is simple — money. Companies that develop and manufacture anti-infective drugs face an operational challenge with the current antibiotic reimbursement model. Doctors must also consider antibiotic use sparingly, only when necessary, so they do not contribute to the growing AMR problem.

Therefore, the money obtained from limited drug use and low reimbursement rates (if they are used) may not sustain the expense of manufacturing. As such, pharmaceutical companies are not monetarily incentivized to participate in research and development for new anti-infective drugs, leading to a dwindling pipeline for this drug classification, according to Dr. Boyle.

What is a viable solution to this problem? Dr. Boyle and Jezek stressed the importance of government policy makers reshaping reimbursement reform and market incentives for antibiotic development.

One such development is the reintroduction this summer of the Pasteur Act, which would drive vital investments by addressing economic incentives. This can be done by unlinking reimbursement from antibiotic sales, with the government paying a flat subscription fee for antibiotics through programs like Medicare. This would mean a more predictable return on investment for drug manufacturers, while simultaneously allowing doctors to make wise decisions to utilize these newly manufactured drugs sparingly to prevent further AMR snowballing.

This policy change could have far-reaching impacts on a foreseeable future public health crisis. Jezek pleaded with policy makers and session attendees to “act now,” as “patients are already losing their lives” due to drug resistance to every possible treatment available today. Dr. Boyle concluded, “the stakes are high,” so “it is a worthwhile cause.”


Dwindling pipeline and growing resistance: examining the impact of antimicrobial resistance on rare disease patients. Presented at: World Orphan Drug Congress USA 2021; August 25, 2021; National Harbor, MD.