CF Foundation Expresses Support of the Give Kids a Chance Act of 2024 and the Telehealth Modernization Act

CF Foundation Expresses Support of the Give Kids a Chance Act of 2024 and the Telehealth Modernization Act

In a letter to the House of Representatives Energy & Commerce Committee, the Cystic Fibrosis Foundation signaled support for the two bills, which recognize the complexity of modern rare disease drug development and protect telehealth flexibilities by removing certain barriers to access that are no longer necessary or beneficial to patients or providers.

Sept. 18, 2024 | 8 min read

Dear Chair McMorris Rodgers and Ranking Member Pallone:

On behalf of the Cystic Fibrosis Foundation, we write to express our support for the Give Kids a Chance Act of 2024 (H.R.3433) and the Telehealth Modernization Act (H.R. 7623). We appreciate that the Committee recognizes the importance and complexity of modern rare disease drug development and applaud the Committee’s dedication to incentivizing the development of these products. We further support the Committee’s work to protect telehealth flexibilities by removing certain barriers to access that are no longer necessary or beneficial to patients or providers.

About Cystic Fibrosis and the Cystic Fibrosis Foundation
Cystic fibrosis (CF) is a rare genetic disease that affects nearly 40,000 adults and children of every racial and ethnic group in the United States. In people with CF, mutations in the CFTR gene result in a buildup of thick mucus in multiple organ systems, leading to lung damage, life-threatening infections, and other complications. 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.

Reauthorizing the Rare Pediatric Disease Priority Review Voucher Program
Through our extensive involvement with the development of CF therapeutics, the CF Foundation is profoundly aware of the urgent need to develop and enable access to treatments that are safe and efficacious for both adults and children living with rare diseases. To ensure the continuation of a critical incentive for rare pediatric disease drug development, the CF Foundation supports the reauthorization of the rare pediatric disease priority review voucher (PRV) program as proposed in both the Creating Hope Reauthorization Act of 2024 (H.R. 7384) and the Give Kids a Chance Act of 2024. Due to limited patient populations, rare disease drug development is often challenging, both in terms of clinical trial design and return on investment once a product reaches the commercial market. These factors can serve as barriers for sponsors interested in entering the space, especially for ultra-rare diseases and narrow subpopulations of rare diseases. For that reason, the federal government has established multiple programs to increase the viability of rare disease drug development. This includes the rare pediatric disease PRV program, which is one of the mechanisms by which the FDA incentivizes the development of drugs specifically for rare pediatric diseases; under certain circumstances and at the FDA’s discretion, sponsors who have developed drugs for rare pediatric diseases may be awarded PRVs, which can be redeemed later to receive priority review of an application of the PRV holder’s choice. In the context of CF, PRVs have been awarded for the development of transformative CFTR modulator therapies, and multiple CF gene therapies in the clinical development pipeline have received rare pediatric disease designation, making them potentially eligible to receive a PRV upon approval.

Bolstering sponsor interest in developing treatments for rare pediatric diseases through various incentivization mechanisms is critical to meeting the needs of the millions of children in the United States living with rare diseases, including CF. By extending the rare pediatric disease PRV program through 2030, the Give Kids a Chance Act of 2024 preserves a valuable tool for tackling one of the greatest challenges of rare pediatric disease drug development; for that reason, the CF Foundation asks Congress to support this legislation.

Protecting Orphan Drug Exclusivity
The CF Foundation has previously supported the limitations on exclusive approval or licensure of orphan drugs proposed in the Retaining Access and Restoring Exclusivity Act (RARE Act, H.R. 7383). This language, also included in the Give Kids a Chance Act of 2024 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 Give Kids a Chance and RARE Acts, 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. For that reason, the CF Foundation once more asks Congress to join us in supporting the Give Kids a Chance Act of 2024 and restoring the 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 CF Foundation thanks the Committee for its leadership as it works to tackle the challenges of modern therapeutic development and specifically incentivize the development of drugs for rare diseases, including CF. We stand ready to work with you to ensure the reauthorization of the rare pediatric disease priority review voucher program and codify the FDA’s long-standing system for awarding orphan drug exclusivity. Additionally, we support efforts to extend and expand access to critical telehealth services post the public health emergency.

Download to Read More
Share this article
Topics
Public Policy

The Foundation regularly submits statements, letters, and comments to legislators for consideration.

View all activity
You might also be interested in...
Stay up to date with The Foundation

Sign up for our newsletter to get all of the latest news from The Foundation right in your inbox.

Subscribe