The National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA) both play a critical role in advancing new therapies for cystic fibrosis that are both safe and effective. The CF Foundation builds on the work of the NIH and FDA by providing additional resources for each stage of the research process. This collaborative approach has led to the approval of more than a dozen CF therapies, including four that treat the underlying cause of the disease.

Advancing Innovation at the National Institutes of Health

More than 30 years ago, there was no effective treatment for CF, and little was known about the disease. In 1989, Francis S. Collins, MD, PhD, who is now the director of the NIH, co-identified the CF gene. In the more than three decades since, incredible progress has been made in the understanding of CF and development of therapies for people living with CF. These scientific advances, along with a comprehensive approach to care, are changing the face of the disease.

The NIH provides the foundational support for basic science research laboratories to survive, develop, and flourish. Discoveries at NIH have helped support our knowledge of the biology of CF and how the disease works, contributing to the fundamental research that fuels the development of future therapies for people with CF.

Today, the CF Foundation works closely with the NIH to support basic science and clinical research for CF. With the help of clinical trial volunteers, the CF Foundation and its research partners are accelerating the search for a cure. Through our policy work, we help ensure that NIH-funded research continues to deepen our knowledge of CF.

Advancing Therapies Through the Food and Drug Administration

A well-resourced FDA helps ensure new drugs are safe and effective and are swiftly reviewed to get them into the hands of people with CF as soon as possible. The CF Foundation’s work with policymakers helps ensure that the FDA proactively seeks meaningful input from patients, providers, and researchers when reviewing applications for rare disease treatments. Through our policy work, we aim to help the FDA accelerate drug review timelines while maintaining the highest standards of approval.