Caleb Nolan would probably object to be called little. He's an active 7-year-old who plays football, baseball, soccer and basketball. He also was born with a rare form of cystic fibrosis that requires him to undergo rigorous treatments and take 23 pills every day.
Recently, Caleb started taking a new drug called Kalydeco that, according to his doctor, has greatly improved the quality of his life. Kalydeco is effective in treating only 4 percent of CF patients. But a new treatment that combines Kalydeco with another drug is one of a handful of new therapies the FDA gave the ok to under the "Breakthrough Therapy" approval pathway we created in the FDA reform bill last year. The new treatment is expected to work for 65 percent of cystic fibrosis patients.
Under the old way of doing business, dramatic treatments would take an average of 15 years to reach patients. Now, it can approved within a matter of a few years.
What we heard from Colorado's doctors, patients, and bioscience community was that the FDA needed to prioritize the approval of drugs and other treatments that showed early dramatic results for serious or life-threatening illnesses. Thanks to the new "Breakthrough Therapy" designation, the FDA can move more quickly to ensure these drugs are safe and approve them for the patients for whom they will work.
Hanging out with Caleb and his mom Melissa at Children's Hospital Colorado was a great way to end a long week in Washington, a place where it's important to remember that little victories can mean big results.
And while we agreed on our mutual love of the Rockies, the Nuggets and the Broncos, we did have a major disagreement: He, like my three daughters, was very opposed to my idea that kids should have to go to school year-round.
Michael F. Bennet
To unsubscribe from this newsletter visit www.bennet.senate.gov/unsubscribe"