Hope is a powerful elixir.
My family was dealt an unexpected blow nearly 26 years ago when our son Gunnar was diagnosed with cystic fibrosis, a debilitating genetic disease for which there is no cure. At the time, the diagnosis was akin to a death sentence because there were no approved treatments and most patients died before they turned 30.
The outlook is brighter for patients diagnosed today. Years of painstaking research has produced three FDA-approved treatments that address the underlying cause of the disease in a small group of patients. More groundbreaking medicines are on the way. Researchers are on the cusp of other medical breakthroughs that could extend life for years — possibly decades.
New medicines and the promise of future discoveries give patients and their families real relief to the debilitating effects of cystic fibrosis. This maturing research pipeline and the promise of additional new treatments on the horizon also give people suffering from this disease hope that tomorrow will be even better than today. And that faith empowers people to fight this life-threatening disease at a time when they feel the most helpless.
These innovative treatments and the potential for future breakthroughs are why I am so concerned with efforts to limit patients’ access to these new medicines. The Institute for Clinical and Economic Review, a self-appointed watchdog with no officially recognized credentials, is marketing problematic “research” to pressure payers to impose dangerous access restrictions on these life-changing treatments. Biopharmaceutical companies make convenient scapegoats, but industry critics are pushing risky policies that make it harder for families in need to access these treatments.
ICER staged a hearing this week in St. Louis to discuss their “findings.” This exercise was set to produce plenty of predictable hand-wringing that largely ignores the economic realities of the health care market. The problem with ICER’s review is that the organization knew the outcome before they started looking at the data and filtered the evidence to arrive at that verdict. They refused to acknowledge the economic benefits of these groundbreaking treatments and ignored the pleas of patients and patient advocates like me who begged them to consider the broader impact of medicine that, for the first time, addresses the underlying causes of this devastating disease.
For 14 years, I had the privilege of playing quarterback in the NFL, having led the Cincinnati Bengals to a Super Bowl and been recognized as NFL MVP during the 1988 season. My career was a dream come true. But not even a successful football career or access to the best doctors prepared us for what lay ahead with Gunnar’s diagnosis. We felt powerless, realizing how little research and awareness there was at that time. My wife, Cheryl, and I took up this cause and created the Boomer Esiason Foundation to improve the lives of those suffering from CF.
Today patients living with CF have a projected life expectancy of only 40 years. To stay “healthy,” patients undergo therapy and take dozens of medicines daily. And their definition of “healthy” is not typical — CF patients spend roughly two hours a day managing their symptoms. The daily demands of managing treatments can take a toll on patients and caregivers alike. Patients often experience severe complications from CF that require hospitalizations for days or weeks at a time. These hospital stays and other complications are disruptive to one’s education, work, social life, and financial security.
For years, treatment was aimed at controlling these symptoms. But decades of dedicated investments led to major medical breakthroughs in the fight against CF, treating the underlying cause of the disease, not just the symptoms. These innovations are why it’s so important to continue research on — and to expand access to — these life-saving treatments.
Although many people currently benefit from the existing drug therapies available, thousands of families like mine are still waiting for medical treatment for their child’s specific type of CF. Roughly 30,000 individuals in the United States are living with CF — about half without medicine to treat the cause of their disease, including my son. Important medical research shouldn’t be suppressed, because it’s critical to improving the lives of tens of thousands of CF patients nationwide both now and in the future. Patients of rare diseases cannot and should not be discriminated against because of a mathematical formula on costs.
This debate upsets me because the criticism ignores the vast time and money invested in developing these treatments and because the proposals advanced by the critics rarely account for the damaging impact on actual patients. This is a hypothetical exercise for groups like ICER, but patients like my son are the ones who suffer if policymakers embrace these questionable economic reviews.
Gunnar is a successful 27-year-old man, and I’m hopeful that we’ll share many more years together. If we discover a new medical treatment, this becomes less of a wish and more of a reality. I hope we remain focused on expanding — not restricting — access to life-saving treatments for those suffering from rare diseases, like CF.
The opinion article was originally published in the St. Louis Post-Dispatch.
Former Bengals quarterback Boomer Esiason is co-chairman of the Boomer Esiason Foundation.