Dear Chair Burgess and Representatives Ferguson, Carter, Smucker, Moore and Yakym,
On behalf of the Cystic Fibrosis Foundation, we thank the House Budget Committee and Health Care Task Force for the opportunity to provide input on improving U.S. health outcomes while lowering health care spending. We would particularly like to comment on how legislation designed to combat the antimicrobial resistance (AMR) crisis, such as the Pioneering Antimicrobial Subscriptions to End Upsurging Resistance (PASTEUR) Act, is both relevant and critical to several areas of interest described in this Request for Information: (1) Congress’s efforts to promote and incorporate innovation into programs like Medicare to reduce health care spending and improve patient outcomes, and (2) examples of evidence-based, cost-effective preventive health measures or interventions that can reduce long term health costs.
About AMR and cystic fibrosis
It has become clear that AMR poses a rapidly-growing threat to the United States health infrastructure and patient health outcomes. According to the 2019 CDC Antibiotic Resistance Threats in the United States report, AMR causes 2.8 million infections and claims over 35,000 lives in the United States each year. The COVID-19 pandemic only worsened this crisis: in 2020, the U.S. experienced a 15% increase in hospital-onset AMR infections and deaths, and experts do not expect a return to pre-pandemic levels without concerted action. Furthermore, AMR has a substantial economic impact. According to a 2022 report from the National Academies of Sciences, Engineering, and Medicine, the direct medical costs in the U.S. of treating only six of the most common drug-resistant pathogens is $4.6 billion per year. Notably, this figure does not include associated and downstream costs, including the cost to the health system after discharge, lost wages, diminished worker productivity, short- and long-term disability, mortality, and cost burden on family and caregivers.
The AMR crisis is particularly dangerous for people with cystic fibrosis, who are extremely vulnerable to pathogenic colonization due to the thick, sticky mucus in their lungs. These infections often require hospitalization and treatment with antimicrobials and can lead to losses in lung function and death. As the funder of more cystic fibrosis research than any other organization in the world, the Cystic Fibrosis Foundation has committed over $140 milllion to date to antimicrobial research and development for antimicrobial through our Infection Research Initiative. However, many people with CF still battle difficult-to-treat infections for which there are no effective antimicrobial drugs available. This dire lack of treatment options stems, in part, from the fact that antimicrobial products are uniquely unsuited to the way that the pharmaceutical market operates. Antimicrobial products are meant to be used for a limited period of time; prescribed sparingly, so as to limit the development of AMR; and, even with appropriate antimicrobial stewardship, become less effective over time, due to the gradual evolution of drug-resistant pathogens. These factors make it extremely difficult for developers of antimicrobial products to earn a return on investment — and, in some cases, to even remain in business.
Because of this, the CF Foundation remains steadfast in our commitment to both supporting the development of novel antimicrobial products and advocating for policy solutions to this systemic market failure. Without an innovative, cost-effective strategy for tackling the AMR crisis, the staggering impact of AMR on people with CF and the broader United States health infrastructure, economy, and population will continue to worsen. The PASTEUR Act presents an opportunity to strengthen the incredibly weak antimicrobial research and development pipeline, stabilize the broken market for antimicrobial products, and ensure clinicians have the novel products they need to treat patients.
Efforts to promote and incorporate innovation into programs like Medicare to reduce health care spending and improve patient outcomes
Looking beyond the CF community, infection and AMR are a significant threat to Medicare beneficiaries. Several demographic and epidemiological characteristics of the Medicare population, including age and the prevalence of chronic health conditions, contribute to high rates of AMR infections in the Medicare population. Aging and age-related changes are associated with the weakening of the immune system and increased susceptibility to infection, and 87% of traditional Medicare beneficiaries are over the age of 65. Analysis of chronic conditions amongst Medicare beneficiaries in 2018 further revealed that the prevalence of cancer and arthritis was 8% and 33%, respectively. Common treatments for these conditions — including chemotherapeutics, steroids, and surgery — can put individuals at particularly high risk for infection through immunosuppression, extended hospitalization and increased exposure to pathogens, and other mechanisms.
To determine the financial impact of AMR as it relates to the Medicare population, a study published in Clinical Infectious Diseases analyzed data from patients 65 years and older within the VA healthcare system and concluded that AMR infections in older adults may have led to an estimated $1.9 billion in health care costs in 2017. An internal analysis of more recent data from the Centers for Medicare and Medicaid Services (CMS) and published in 2021 found that Medicare beneficiaries account for the majority of cases of both new diagnoses of AMR infections and resulting deaths in United States hospitals, spending hundreds of thousands of additional days in hospitals each year due to AMR and costing taxpayers billions in extra heath care costs annually. These data indicate that efforts to address AMR will not only improve patient outcomes for Medicare beneficiaries but may also yield net savings to the federal government.
CMS has made several efforts to mitigate this costly issue and secure access to life-saving drugs for American seniors using modest, proof-of-concept “pull” incentives, which are designed to reward successful antimicrobial development by increasing or ensuring future revenue. One example is the recently-finalized alternative pathway for the New Technology Add-On Payment for drugs designated by the Food and Drug Administration (FDA) as Qualified Infectious Disease Products (QIDPs). This change, which increased payments to hospitals for QIDPs, “reflects [CMS]’s awareness of the public health imperative for novel antibiotics” and is meant to “reduce barriers to accessing the incentive while ensuring that payment systems reflect the value of new innovations.” In recognition of the added clinical complexity and cost of treating patients with drug-resistant infections, CMS also increased the severity level designation for diagnostic codes for AMR; this facilitated increased payments for inpatient cases involving AMR and was designed to ensure that physicians are incentivized to use the appropriate, if more costly, antimicrobial products to treat AMR infections.
However, while CMS’s efforts speak to the urgency and necessity of addressing AMR specifically within Medicare and facilitating access to novel antimicrobials for the Medicare population, the impact of even these narrow changes is limited by the current lack of novel antimicrobials, both existing and in development. It is therefore critical to enact broader, more effective versions of these “pull” incentives — such as the PASTEUR Act — that would provide sufficient financial incentive to spur the research and development of new antimicrobials, which may then be made available to Medicare beneficiaries under these new rules to improve patient outcomes.
Examples of evidence-based, cost-effective preventive health measures or interventions that can reduce long term health costs
Despite the magnitude of the AMR crisis, the antimicrobial ecosystem is remarkably weak. Fewer than 50 antibacterial therapeutics are currently in clinical development worldwide, only a handful of which are for the most threatening gram-negative pathogens. In recognition of the dire unmet need for innovative antimicrobial products, the United States Government Accountability Office formally recommended in a March 2020 report that the Department of Health and Human Services develop a strategy to further incentivize the development of new antimicrobial products for drug-resistant infections, including through the use of post-market financial incentives.
The PASTEUR Act offers an innovative post-market incentive structure to both facilitate the revitalization of the antimicrobial pipeline and make novel antimicrobials available to those in need. Under PASTEUR’s subscription model, the federal government can enter into contracts with developers of innovative antimicrobials to pay for a reliable supply of product. The subscription contract is all-inclusive, and the federal government only pays once; drug products that have been awarded PASTEUR funding become available to patients enrolled in federal health care programs, including Medicare, at no additional cost to the government. Payments are decoupled from the volume of antimicrobials used, thereby removing the incentive for companies to promote the widespread use that often results in the development of drug-resistant pathogens. Similar delinked approaches have been endorsed by the Presidential Advisory Council on Combating Antibiotic-Resistant Bacteria and the Duke University Margolis Center for Health Policy.
Critically, PASTEUR contracts are awarded exclusively on the basis of innovation and success. PASTEUR will only fund antimicrobials that have been approved by the FDA and meet established criteria for novelty and fulfilling unmet AMR needs — in other words, products with a significant impact on patients and public health. Economic modeling performed by the Center for Global Development suggests that a subscription-based approach to incentivizing antimicrobial development, such as that authorized by the PASTEUR Act, would generate a significant return on investment (ROI) in both the short- and long-term. From the U.S. domestic perspective, taking into consideration the value of averted death and disease plus associated hospital costs, the predicted ROI for an annual $1 billion investment in new, high-impact antimicrobials was calculated at 6:1 over ten years and 28:1 over thirty years. This represents a significant value for the United States and U.S. taxpayers.
The CF Foundation appreciates the opportunity to respond to the House Budget Committee and Health Care Task Force’s Request for Information on improving U.S. health outcomes while lowering healthcare spending. Tackling the AMR crisis through legislation like the PASTEUR Act will help reduce long-term health care spending while also improving patient outcomes. We look forward to continuing this dialogue and serving as a resource for the Committee and Task Force going forward.