Cystic fibrosis has drastically changed in a single generation. Once seen as a pediatric disease, people with CF are now living into adulthood thanks to groundbreaking treatments and highly specialized care.

This progress must continue.

As Congress shapes its health care agenda for 2025, it must prioritize patients and protect the advancements that have changed lives. Congress must safeguard access to Medicaid and ensure the National Institutes of Health (NIH) and U.S. Food and Drug Administration (FDA) are well-resourced.

Safeguard Medicaid — a Lifeline for People With CF

Life with cystic fibrosis demands a complex regimen of care and treatments. While advances in CF care are helping people live longer, healthier lives, cost and access are barriers to care for many people with the disease. While nearly all people with cystic fibrosis have at least one form of health insurance, that’s often not enough to cover everything they need to stay healthy. Medicaid fills gaps left by private insurance plans, helping half of children and one-third of adults with CF afford treatments, medication, and specialized care.  

We call on Congress to maintain access to Medicaid for the nearly 40% of patients with CF who rely on it by: 

• Oppose funding cuts: Proposals that limit Medicaid funding will create more barriers to care for people with CF. States may change eligibility requirements or benefits provided through the program to counter funding cuts on the federal level. Rigid funding structures do not account for innovative, novel therapies or technologies that come to market. Such changes would limit a state’s ability to keep pace with providing residents access to these life-saving treatments and specialized care.    
• Reject access barriers: Establishing work reporting requirements increases the administrative burden for patients, which may lead to delays in care and treatment. Because CF is a progressive disease, people who delay or forgo treatment may face an increased risk of lung exacerbations, irreversible lung damage, and costly hospitalizations.

Fully Fund the NIH and FDA to Keep Innovation Moving

There is still no cure for cystic fibrosis. While many people with CF are now living longer, healthier lives due to the advancements in drug discovery and development, many are still waiting for transformative therapies to address the underlying cause of their disease.

Through public-private partnerships with the FDA and the NIH, progress in CF has been swift. Robust funding is critical to continue that progress.

National Institutes of Health

The CF story of progress and transformation would not exist as it does today without the NIH and the research it supports. The original research that led to the discovery and characterization of the gene responsible for cystic fibrosis was funded in part by both the NIH and the Cystic Fibrosis Foundation. Together, we also support multiple academic centers, advance the development of new technologies, provide training and research support, and co-host workshops on topics critical to CF research and development. The advances in modern therapeutics development that are synonymous with cystic fibrosis have been built on the foundation of decades of basic research, translational science, and clinical trials supported or carried out by the NIH.

This progress must continue. Robust funding for the NIH is critical to advancing cystic fibrosis research, drug discovery and development. In the 2023 fiscal year, the NIH spent $80 million on CF research.

Fully funding the NIH promotes deeper understanding of chronic illnesses like CF, making future drug discovery and development possible.

U.S. Food and Drug Administration

The FDA has evaluated and approved 16 groundbreaking CF therapies, including five to treat the underlying cause of CF, ensuring patient access to safe and effective therapies.

In 2024, the FDA created the Rare Disease Innovation Hub to facilitate and streamline interactions between the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) to drive rare disease drug development and make it easier for the rare disease community, including the CF Foundation, to collaborate with and provide expertise to the FDA.   

With dozens of new therapies currently in clinical trials, robust funding ensures the FDA maintains staff and resources to thoroughly and efficiently evaluate new drugs for CF and other rare and life-threatening diseases in an era of increasingly rapid and complex scientific and technological advances.