Just over 24 years ago, my son Gunnar was diagnosed with cystic fibrosis, a debilitating and unrelenting disease for which there is no cure. At the time of his diagnosis, CF was akin to a death sentence – there were no FDA-approved treatments and we could only hope that we might see a cure in time to save Gunnar’s life.
I had the privilege of playing quarterback in the NFL for 14 years. Leading the Cincinnati Bengals to a Super Bowl and being recognized as NFL MVP during the 1988 season highlighted a career that was a dream come true. However, when Gunnar was diagnosed, no successful football career, personal wealth or access to the best doctors could have prepared us for what we encountered – a lack of research and awareness of a rare disease that cut many lives far too short. For me and my wife, Cheryl, it became the cause of our lives.
Fortunately, these past few decades of costly and tireless research have yielded major breakthroughs in the fight against CF. Today, there are two FDA-approved treatments available to a small group of patients that treat the underlying cause of the disease, with more on the way. Researchers are making new discoveries that stand to extend lives by years, if not decades. In the 1950s, kids with CF often didn’t live long enough to attend elementary school. However, thanks to cutting-edge research and public-private collaboration, nearly half of today’s CF population is over the age of 18.
While thousands of families are benefiting from these new discoveries, many continue to wait for drug options that will treat their son or daughter’s specific type of CF. We are amongst those families: prescription drug treatments are still unavailable for Gunnar. There are more than 30,000 people living with CF in the U.S., and medicines that treat the underlying cause of the disease are available for only a fraction of these patients – more than two-thirds are still waiting.
What will it take to turn the corner? Beyond research and hope, we must rely on regulatory bodies such as the U.S. Food & Drug Administration to play their part and prioritize the review of treatments that have successfully completed clinical trials.
For Gunnar, we have had multiple attempts with clinical trials, but little success. The speed at which new drugs are reviewed and approved will directly impact the chances that a new treatment – whether it be in a research laboratory or one that is entering the clinical trial process – is the one to save his life.
When it comes to new drug approvals, the FDA has made considerable progress. In 2015, the agency approved 51 new medicines, the highest number since 1950. Over the past five years, Congress has helped reduce barriers by expanding what the FDA can consider for accelerated approval and permitting the use of data sets for multiple drug review processes, among other modifications.
This is laudable in every way, but our work is far from over. Again, my son is beating the odds and surviving this disease. But the fact that there is no available treatment to address the underlying cause of his CF should be enough to demand a more aggressive review of medicines that could save lives.
Some have proposed major changes to the existing system, including a number of states considering right-to-try legislation that would permit limited access to medicines prior to FDA approval. I don’t fault patients and families for wanting to do everything they can to give their loved ones a fighting chance, but I’m confident that the existing system is workable if we have the right resources and mindset in place.
Today, Gunnar is a successful 26-year old young man. I’m hopeful that he’ll live for many more years to come. With the right treatment, this moves from possible to reality.