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Cystic Fibrosis Foundation Drug Development Model Spawns More Than 30 Promising Therapies

The top priority in clinical research is protecting the safety of people who participate. Learn more about the numerous layers of review that help keep participants safe.

When my 8-year-old daughter with CF found out that the disease is potentially fatal, I was grateful for her optimistic views on life and death. But deep down, I was saddened with the thought of a cure being so far away that I wouldn't be around to see it. Today, my fears have dwindled. 

Ever wondered what it takes for a potential cystic fibrosis drug to become approved by the U.S. Food and Drug Administration? Here's an explanation of the four phases of clinical research.

Earlier this month, we learned that the new CF drug Orkambi was approved by the FDA. This was such an exciting, memorable and emotional day for everyone in the CF community. We heard from many who were thrilled about the potential for this drug to help people with CF and also heard from those who would not benefit from this breakthrough. Following are some reactions from those this impacts the most: people with CF and their loved ones. 

Dr. John P. Clancy shares new developments in drugs that restore airway surface liquid in the lungs of people with cystic fibrosis, making it easier to clear mucus.

Dr. James Chmiel shares new developments in drugs to reduce inflammation in the lungs and help prevent lung damage.