“The Colorado Prescription Drug Affordability Board [PDAB] recently determined that Trikafta is not unaffordable for people living with CF. We’re grateful the review centered around patients and their ability to afford medication, and that access is preserved.”
So tweeted the Cystic Fibrosis Foundation (CFF) on December 11, via a statement that was very carefully worded and, indeed, noteworthy for what it does not say.
With a list price in excess of $300,000, Trikafta is a miracle drug that is at least three times as expensive as it ought to be. So far, Vertex Pharmaceuticals has gotten away with charging that kind of money. The CF Foundation, like the thousands of patients who benefit from Trikafta, are caught in the middle of any conversation that challenges such an astronomical price.
Nowhere does the CF Foundation defend what Vertex is charging for Trikafta. The Foundation merely praised the Colorado regulators for deeming the drug – as the CFF put it – “not unaffordable.”
Colorado is not exactly the center of the CF universe – less than 500 Coloradans were on Trikafta last year – but the stakes were high nevertheless. Only ten states (Colorado, Maine, Maryland, Minnesota, New Hampshire, Oregon, and Washington) have PDABs. Some – Colorado included – have the authority to set an “upper payment limit” (UPL) for drugs deemed unaffordable. The Colorado PDAB is the first such body to review Trikafta – and Trikafta was the first drug reviewed for affordability by the Colorado PDAB.
So, Big Pharma was watching. The CF community was watching. And those who parrot the Big Pharma talking points were talking.
This means you, Gunnar Esiason – son of celebrity NFL quarterback Boomer Esaison and self-appointed spokesperson for his fellow CF patients. His November 3 essay in the trade publication Stat derides PDABs as “bureaucratic agencies” created to “impose European-style price controls on prescription medications.”
Why was the Colorado PDAB vote on Trikafta such a big deal? It’s all about Medicaid, the federal health insurance program that is the nation’s dominant payor when it comes to prescription drugs.
Medicaid gets a minimum of 23.1 percent off the best price available to other payors. Thus, according to Esaison, a new and lower price for Trikafta, had the Colorado PDAB ultimately set one, could “cascade” into the Medicaid market. He warned that Vertex could simply decide to stop selling Trikafta altogether in Colorado to avoid such a cascade.
It’s a cascade alright – of baloney. It appears that Gunnar Esiason cannot picture a world in which Vertex lives up to its self-proclaimed commitment to cystic fibrosis patients by charging everyone a fair price for a breakthrough drug like Trikafta.
According to the Institute for Clinical and Economic Review (ICER), a fair price for Trikafta would be somewhere around $80,000 to $100,000 a year. Note that ICER – an independent nonprofit -- exists because, as Esaison notes, the U.S. has not followed the lead of other countries around the world and allowed the government to determine fair prices for expensive drugs. The National Institute for Health and Care Excellence (NICE) – which IS part of the government, in the United Kingdom – recently caused an uproar by issuing a draft assessment that agreed with ICER: Trikafta (known across the pond as Kaftrio) is way too expensive.
All of which brings me around to full disclosure time.
As New Hampshire’s state-appointed Consumer Advocate – my day job is to represent the interests of residential customers of electric, gas, and water utilities -- I know excessive monopoly prices when I see them. And I am a cystic fibrosis dad, which is why I was outraged when I discovered what Vertex was charging for Trikafta.
When I get mad – especially about anything involving cystic fibrosis – I take to social media. That’s how ICER discovered me, and invited me to join the experts who spoke at the public meeting ICER convened to deliberate on its Trikafta report. Today I serve on one of the three independent appraisal committees that ICER convenes to conduct such deliberations. It is an honor to help ICER assure that its assessments are grounded in the real-world needs of patients and their families.
Meanwhile my daughter – the reason I care about all of this stuff – seems to have inherited the outspokenness gene from her dad. She, too, is now connected to ICER (as a patient engagement intern) as she approaches her graduation from college next May. And good grief did she ever make her old man proud when she stepped forward to rebut my friend Gunnar and to testify before the Colorado PDAB just before it ruled Trikafta “not unaffordable.”
I mention my daughter here not just in the interest of full disclosure but also to prove that almost all of the public accounts of the Colorado PDAB meeting are wrong. Reading those accounts, one would think that the CF community was united in its defense of Vertex and its ridiculously high price for Trikafta.
In reality, Vertex buys off its potential critics. It does that not just by donating money outright to organizations like the Boomer Esaison Foundation and the Cystic Fibrosis Research Institute. It does that through its co-pay assistance program, the purpose of which is to assure that no matter how huge the price of the drug is the out-of-pocket costs to patients are small or nonexistant.
It is the availability of such assistance – and the fact that, so far, both Medicaid and private insurance plans have been covering their part of Trikafta’s costs – that formed the basis of the Colorado PDAB’s determination that Trikafta is affordable for patients. I earnestly hope the nation’s nine other PDABs keep that in mind should Vertex’s prices ever come before them.
As a publicly traded company, Vertex has been a high-flying stock of late. On December 13 alone, the price shot from $357 a share to $405, thanks to some promising results from a drug trial unrelated to CF. Trikafta and its related modulator drugs have made a fortune for Vertex, but the bonanza is largely an accidental one. Vertex acquired the rights to the modulators in 2001, when it purchased Aurora Biosciences for reasons unrelated to cystic fibrosis. (If you don’t believe me, read Chapter 37 of Bijal Trivedi’s remarkable 2020 book Breath from Salt, which tells the CF story.)
It's also worth noting that Aurora and Vertex developed those modulators with the help of a $225 million investment from the CF Foundation, which later cashed out its resulting royalty rights to the tune of $3.8 billion.
If that’s a paradigm example of a Faustian bargain, my family is among its beneficiaries. As my daughter forthrightly explained to the Colorado PDAB, she is among that small cohort of CF people who, because of unusual CF mutations, is ineligible for the Vertex modulator drugs. Her miracle, funded in part by that $3.8 billion, lies ahead. And when it arrives it will be expensive, which is why both she and I care about the price of breakthrough CF medications.
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