My daughter Rose is still waiting for her miracle, and I am not going to allow a think tank, or the son of a famous former NFL quarterback, or a pharmaceutical company keep it from her.
The miracle in question is an effective treatment for her cystic fibrosis – the life-shortening inherited disease with which she struggles heroically. Rose is off to Bowdoin College this fall, and has Olympic aspirations in her chosen sport (dressage), even though her CF is with her constantly and usually requires at least one extended hospitalization a year.
The think tank in question is the Pioneer Institute in Boston. The quarterback’s kid is Gunnar Esiason, also a CF patient. The Big Pharma firm is Vertex – which developed the latest CF miracle drug, Trikafta, approved by the FDA last fall.
Trikafta is the latest in the so-called “modulator” drugs produced by Vertex. Collectively, they provide help to as many as 90 percent of the nation’s CF patients. Unfortunately, Rose is not one of them.
Trikafta’s approval was the subject of delirious celebration at the North American CF conference in Nashville last fall. I know because I was there, caught up in the joy, even though Rose’s breakthrough is yet to come.
Nobody in Nashville was talking about the price of Trikafta. It’s $312,000 a year, per patient.
But the nonprofit Institute of Clinical and Economic Review is certainly talking about it, having released a report of over 300 pages in April demonstrating why Trikafta and the other Vertex modulators are too expensive.
Vertex is listed as a partner of the Boomer Esiason Foundation, whose director of patient outreach, Gunnar Esiason, has teamed up with the Pioneer Institute in an effort to discredit ICER.
The title of a recent webinar, at which Gunnar Esiason spoke alongside a representative from the Pioneer Institute and someone from a group called the Senate Conservatives Fund, says it all: “Protecting Cystic Fibrosis Patients from Discrimination by ICER Through COVID-19 & Beyond.”
The idea that it is discrimination to hold Vertex accountable for the obscene price of Trikafta has to do with a key metric employed by ICER in its analysis. At issue is the Quality-Adjusted Life Year.
A QUALY equals one year spent in perfect health. According to the ICER analysis, each of the Vertex modulator drugs carries a cost per QALY gained of more than $1 million – which, according to ICER, is “substantially higher than cost-effectiveness thresholds in the US and the rest of the world.”
According to Esiason and other critics, this is discrimination because it undervalues health benefits provided to people in less-than-perfect health, including people with CF who have suffered permanent damage to their lungs.
This claim is spurious for at least three reasons.
First, ICER can’t discriminate for the simple reason that it’s just a research and analysis organization. Its reports are purely advisory; ICER has no authority to set the price of Trikafta or any other drug.
Second, the QALY is not the only metric relied upon by ICER. Also analyzed is the evLYG (Equal Value Life-Year Gained), which makes no adjustment for quality of life. Notably, ICER found that Trikafta would still be too expensive if it cured cystic fibrosis outright. (It doesn’t, but in that scenario a QALY and an evLYG have equal value.)
Third, the purpose of the ICER report is not to keep anyone from accessing the Vertex drugs and thus subject them, somehow, to discrimination. The report actually has the opposite purpose – to pressure Vertex into charging a reasonable price and thus assure its wider availability.
The campaign to discredit ICER has enlisted patient/family advocates from the cystic fibrosis community. But I am one such advocate who is not fooled. It’s because of my day job. I am New Hampshire’s ratepayer advocate, which means I argue with public utilities about their rates for a living.
Companies that develop breakthrough medications like Trikafta are far riskier investments than electric or water companies with monopoly franchises. I get that, which is why I acknowledge that Vertex’s shareholders deserve a healthy rate of return. But the company should be transparent and accountable.
You might wonder why I connect all of this to my daughter Rose, given that she is not eligible to take the Vertex modulator drugs and likely never will be. Here’s my answer.
Her breakthrough will come, because the Cystic Fibrosis Foundation has committed at least half a billion dollars to deliver such a miracle to that last 10 percent. But if the price of Trikafta is more than $300,000 a year, a therapy for an even smaller cohort is destined to be even more expensive.
Unless, that is, we do something to hold drug companies accountable for the prices they charge. Which is exactly what ICER is striving to do. Many different words can be used to describe this effort, but discrimination is not one of them.
D. Maurice Kreis heads New Hampshire’s Office of the Consumer Advocate and is the dad of an 18-year-old with cystic fibrosis.
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