Dear Chairs and Ranking Members of the Energy and Commerce, Full Committee and Health Subcommittee,
On behalf of the Cystic Fibrosis Foundation, we thank the House Energy and Commerce Health Subcommittee for its dedication to strengthening the healthcare system for all Americans, including the nearly 40,000 children and adults living with cystic fibrosis in the United States. CF is a complex, multi-system genetic disease that can affect people of every racial and ethnic group. It causes the body to produce thick, sticky mucus that clogs the lungs and digestive system and can result in life-threatening infections; if left untreated, these infections and other exacerbations caused by CF can result in irreversible lung damage and early death, usually due to respiratory failure.
The CF Foundation is a national organization dedicated to ensuring that people with CF live long, fulfilling lives, with the ultimate goal of curing CF. We achieve this by investing in research and development of new CF therapies, advocating for access to care for people with CF, and funding an accredited network of specialized CF care centers. In response to this markup of legislative proposals aimed at strengthening America’s healthcare system, the CF Foundation offers support for several pieces of legislation tackling orphan drug exclusivity and telehealth — topics that are critical to the CF community. In addition to the bills slated for markup, the CF Foundation urges the Committee to recognize the critical issue of antimicrobial resistance, particularly for patients with chronic diseases, and take up and pass H.R. 2940, the Pioneering Antimicrobial Subscriptions To End Upsurging Resistance (PASTEUR) Act.
Protecting Orphan Drug Exclusivity
The CF Foundation supports H.R. 7383, the Retaining Access and Restoring Exclusivity (RARE) Act, which would restore the FDA’s long-standing system for awarding orphan drug exclusivity (ODE) based on “use or indication” within a disease or condition. To incentivize the development of drugs for rare and orphan diseases, the Orphan Drug Act established a term of market exclusivity for drugs intended to treat those populations. ODE protects companies from parties seeking approval for the “same drug for the same disease or condition” for seven years. Importantly, the FDA has historically interpreted this as protecting exclusivity for the “same use or indication” within a disease or condition. However, the Catalyst Pharms., Inc. v. Becerra court decision would require the FDA to grant ODE based on “disease or condition,” not “approved use or indication” within the disease or condition. Under the Catalyst decision, once an orphan drug is approved for a single use or indication, the FDA cannot approve another company’s application for the same drug for any additional use or indication within that disease (e.g., pediatric populations).
In the case of CF therapeutics, sponsors often pursue label expansions to add additional indications, such as new genotypes or age groups, to a drug’s label. Each label expansion receives an additional, but separate orphan drug exclusivity period. Without the clarification provided by the RARE Act, these additional label expansions could block generic drugs from coming to market for the populations included in previous labels, even when those earlier exclusivity periods expire. This means that patients may wait longer for more affordable options. The CF Foundation therefore asks Congress to support the RARE Act and restore FDA’s long-standing policy on orphan drug exclusivity.
Ensuring Access to Telehealth Services
The CF Foundation also supports H.R. 7623, the Telehealth Modernization Act, which updates coverage restrictions in Medicare and ensures continued access to telehealth services for beneficiaries with cystic fibrosis. With the telehealth flexibilities initiated during the public health emergency set to expire at the end of 2024, this legislation is essential for patients who have come to rely on telehealth for necessary care.
Prior to the COVID-19 pandemic, Medicare rules largely limited use of a patient’s home as the originating site to those living in rural areas or with a specific condition. The drastic increase in telehealth usage during the public health emergency demonstrated the burden of geographic restrictions and shown it is appropriate and safe for patients to receive care from their homes. While telehealth visits are not suitable for all health care services and are not a substitute for in-person care, there are several aspects of a regular CF visit that can be conducted through telehealth. For instance, clinicians can easily review medical history, current medications and symptoms, and adjust a patient’s care plan. CF patients and care teams can also review data from home spirometers to track trends in lung function. This legislation permanently removes these antiquated location requirements, permitting people with CF to access their established care team from their homes.
Beyond eliminating originating site restrictions, the temporary flexibilities have increased the types of providers who are eligible to provide telehealth services. The CF clinical care team includes physicians, nurses, dietitians, social workers, and respiratory therapists — each of whom plays a unique role in managing CF care. Having access to all members of the care team helps patients better maintain and manage their care, leading to more consistent and better outcomes. This legislation enables the HHS Secretary to maintain and expand providers eligible to deliver telehealth services, therefore increasing access to care and improving care continuity for patients with CF.
The pandemic demonstrated the importance of access to telehealth services and provided evidence that certain barriers to access are no longer necessary or beneficial to patients or providers. The CF Foundation thanks you for your leadership on this important issue for people with cystic fibrosis and we support your efforts to extend and expand access to critical telehealth services post the public health emergency.
Combatting the Threat of Antimicrobial Resistance
AMR poses a rapidly growing threat to both CF patients and the entire United States population. 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. The economic toll of the AMR crisis is also staggering: 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 threat is particularly dangerous for people with CF, who are extremely vulnerable to pathogenic colonization and life-threatening infections 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. For these reasons, the CF Foundation urges this subcommittee to take up the H.R. 2940, the Pioneering Antimicrobial Subscriptions To End Upsurging Resistance (PASTEUR) Act in your next legislative hearing.
It is critical that the United States invest in innovative approaches to combatting the AMR crisis, especially those that would incentivize the development of new antimicrobial products. 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 GAO 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.
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. 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. 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. Furthermore, the subscription contract is all-inclusive, and the federal government only pays once. Economic modeling performed by the Center for Global Development suggests that this sort of subscription-based approach to incentivizing antimicrobial development would generate a significant return on investment 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.
Without innovative, cost-effective strategies for tackling the AMR crisis, the tremendous impact of AMR on people with CF and the broader United States population will continue to worsen. Because 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 that patients have access to the novel treatment options they need, the CF Foundation urges the Energy and Commerce Committee to prioritize this urgent health issue and support passage of this vital legislation.
The CF Foundation appreciates the opportunity to support and provide feedback on the subcommittee’s critical efforts to strengthen America’s healthcare system. We look forward to continuing this dialogue and serving as a resource for the committee going forward.