Some of the memories from my 20s are harsh enough to send a shiver down my spine.
At a Fourth of July barbecue, I coughed up so much blood I had to make a mad dash to the inpatient unit at Columbia University Medical Center.
There was the antibiotic that was so toxic I couldn’t feel my face, hands and feet for days.
The worst of them all was when I was bedridden and wasn’t able to make the trip from New York to Maryland for my Grandy’s funeral.
Cystic fibrosis, the rare genetic condition that fills my lungs with thick, sticky mucus, stole most of my early 20s from me. Starting a career was out of the question when getting through one day at a time was an accomplishment unto itself.
There was doubt that I would make it beyond 30 years old – about the median age of death for people with cystic fibrosis.
Instead of enjoying the independence that comes with early adulthood, my survival was completely dependent on my parents. My cystic fibrosis was progressing so rapidly that I was averaging three or four invasive medical procedures per year. Those interventions piled countless lost hours on top of my already rigorous daily standard of care. All told, my care routine was about two hours when I was “healthy,” but ballooned to five or six hours whenever my symptoms flared.
I never could have imagined what my life would look like now.
Today, I am a graduate student at Dartmouth College. I live hundreds of miles from my family home and when I skated with my grad school’s club hockey team this past season, the rush of fresh air in and out of my lungs felt like a victory.
I am thriving today because a medication called Trikafta saved my life.
In 2018, I enrolled in a phase III clinical trial that eventually led to Trikafta’s Food and Drug Administration approval in October 2019. Within a mere two weeks after starting the drug, my lung function increased for the first time ever. From there, I went procedure-free for a full calendar year, a feat I had not achieved since the beginning of high school. Amazingly, I have since been able to cut 35 percent to 40 percent of medications out of my daily routine.
The medication that has turned my life around has, however, come under the suspicious eye of a watchdog group called the Institute for Clinical and Economic Review. ICER uses a discriminatory cost-effectiveness model called Quality-Adjusted Life Years to determine the value of different medications. QALY, which ICER calls the “gold standard” to determine cost-effectiveness, is a utility analysis that evaluates the price of a particular drug against its efficacy in improving a person’s life, or according to the model, the utility value recovered by the drug. It is discriminatory because it does not value years of life equally for people who are terminally ill compared with people who are healthy.
The core calculation is quite simple, but hinges on several inputted assumptions like a patient’s expected mortality. QALY multiplies the cost of a drug with utility gained (the state of a person’s health) over the expected remaining years of life for a patient with chronic disease. The output is the “QALYs gained” by the patient.
Quality of life is on the short list of inputs included in ICER’s models. By its own admission, though, those models fall short of accurately measuring quality of life gains. In that regard ICER is right. There is no model on this planet that could quantify the insidious panic I felt when I was spitting up blood on the way to the hospital a decade ago. Just as there is no way to measure the heart pounding adrenaline I felt when I proposed to my fiancée this past February.
The very best parts of life do not have a number attached to them.
But according to QALY, the worst do. Those of us with critical illness achieve the highest relative value when we die – when our care costs and utility are both 0 – and that’s exactly where I was headed before I started on Trikafta.
Last week, ICER published a cost-effectiveness analysis for Trikafta. The organization concluded that Trikafta “substantially improves patient outcomes,” but is priced too high relative to its efficacy. The former feels like common sense for those of us living with CF. The latter is an ominous statement directed towards insurers and politicians left open to interpretation.
Yes, the list price for Trikfata is high, but I ask you, what is more cost-effective for society? Is it the five-year period of my life when I was a health care super-utilizer, back when I submitted myself to nearly two dozen invasive medical procedures, countless off-label medication prescriptions, clinic visits every month and more readmissions than I care to count? Or is it now, a time when I am a grad student who is planning a wedding and looking forward to a long career?
Despite the fact that the Affordable Care Act says that cost-effectiveness measures like QALY cannot be used to determine Medicare coverage, insurers and state Medicaid agencies have cited ICER analyses in the past, which ICER acknowledged in a statement to the CF community. Curiously, however, it clarified its conclusion by saying, “Even though our report’s findings suggest that the price of Trikafta is set too high in relation to its clinical benefits, we certainly do not call for non-coverage as the answer.” Then why proceed with the QALY model? ICER says in its report that other QALY models are used across the globe to justify not covering medications for people with CF.
The real-life implications of this drug are so profound that a condition that once killed most patients before they turned 30 has seemingly been transformed overnight. Our futures are suddenly intact. Before Trikafta, we had been chasing a miracle for decades. It was finally delivered at the cost of countless lives lost. Do not let ICER take our miracle from us.
Gunnar Esiason, who is living with cystic fibrosis, is an MBA/MPH Candidate at Dartmouth College and an MBA Fellow at the Tuck School of Business Center for Health Care, and serves on the board of directors at the Boomer Esiason Foundation, his family’s nonprofit organization that has raised near $150 million in the fight against cystic fibrosis.
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