For a miracle drug, how much should you have to pay?
In 2019, a medicine called Trikafta hit the market for patients with cystic fibrosis, a disease that damages the lungs and other organs, and the results have been nothing short of extraordinary.
People taking the drug have seen their hospitalizations reduced by more than 70%. Lung transplants have plummeted. According to the drug’s manufacturer, Boston-based Vertex, Trikafta is projected to be able to give cystic fibrosis patients a near-normal life expectancy for the first time in history.
The price of those benefits, though, is also extraordinary.
According to state data, Trikafta costs an average of $234,439 per patient per year in Colorado. Cystic fibrosis is a rare disease, but even with only a few hundred patients taking the drug in Colorado, the total amount spent on Trikafta in the state in 2022 topped $108 million. The state government spent more than $46 million of that because roughly half of cystic fibrosis patients taking the drug are covered by Medicaid.
But, in a remarkable hearing Friday, a ground-breaking state board declined to label Trikafta unaffordable. The decision, among other things, represents a startling turn for one of Gov. Jared Polis’ and Capitol Democrats’ major policy initiatives to reduce the costs of health care.
“This is an extraordinarily expensive drug, and that’s why we’re doing an affordability review of it,” Dr. Gail Mizner, a Snowmass Village physician who is the chair of the state’s Prescription Drug Affordability Board, or PDAB, said Friday. “But it is also an extremely beneficial drug that is making an enormous difference in the lives of Coloradans living with cystic fibrosis. And at this point, it does not seem to be unaffordable.”
To understand why this was such a significant decision, it helps to understand the procedural wheels that have been quietly churning for months.
When lawmakers created the board in 2021, they endowed it with an unprecedented power: The board can, after careful review, decide if a drug is unaffordable. If the board declares it to be so, it can then set what is called an “upper payment limit.” In other words, the board can cap the price of the drug in Colorado.
This has never happened before — Friday’s hearing marked the first time the board had taken an affordability vote. An upper payment limit on Trikafta would have been the first on any drug in the country, had the board approved it.
But the debate over Trikafta exposed the difficulty in pinpointing what the effective cost of a medicine is to a patient. It also created unusual battle lines, pitting consumer groups advocating for reducing costs on behalf of patients against some of those very patients who in theory would benefit from a price reduction but were worried that they would lose access to the drug altogether.
During Friday’s hearing, multiple people from the cystic fibrosis community, speaking against declaring Trikafta unaffordable, talked of the trauma that the board’s meetings had wrought in their lives. They were concerned that, if a price cap was set, Vertex would decide to stop selling Trikafta in Colorado.
“The fear that you saw from all of us was real,” said Amanda Boone, a Colorado woman living with cystic fibrosis who founded an advocacy group when the board began reviewing Trikafta. “We were worried that we would have to move out of the state, so then health reform would have been on our backs.”
Added Siri Vaeth, the executive director of the California-based Cystic Fibrosis Research Institute: “We are not here to defend the price of Trikafta. We are here to defend our access to the drug.”
Making the analysis even more difficult for board members is that most patients currently appear to shoulder very little of Trikafta’s enormous price.
According to data compiled by state regulators, the average out-of-pocket cost for patients in Colorado taking Trikafta was $8,907 — or about 4% of the total per-patient spending.
Approximately half of patients on Trikafta — excluding those on Medicaid — paid between $0 and $50. This is the result of discount and rebate programs currently available to patients from Vertex.
So how do you decide whether a drug is unaffordable? Unaffordable to whom?
“The state budget and Medicaid budget are limited,” Bethany Pray, with the Colorado Center on Law and Policy, told the board. “Spending more on drugs means we have to spend less elsewhere. … A so-called miracle drug can, nonetheless, be unaffordable.”
But the board members concluded they needed to look at affordability only to patients. And they had heard no testimony from Colorado patients on Trikafta who couldn’t afford the drug — even though board members said they were worried that could change if its maker drops the assistance programs.
“While they have the discount programs that may make it more affordable, we don’t have a guarantee on those discount programs staying in place,” said Cathy Harshbarger, a board member who is the CEO of Melissa Memorial Hospital in Holyoke.
The board ultimately voted 5-0 not to declare Trikafta unaffordable. Board members said they may revisit the analysis, though, if the assistance programs go away. Vertex this year decided to curtail some assistance programs, and the board members noted that the average out-of-pocket costs for patients in 2022 had nearly doubled compared to 2021.
“I would certainly hope if things change down the road, they would let us know,” Mizner, the board chair, said of members of the cystic fibrosis community. “If they’re suddenly finding Trikafta unaffordable, I want to know about that.”
Afterward, consumer groups said the board’s decision, rather than being a setback, was a win for the system. The board had done a detailed analysis of Trikafta’s price. It had considered the dozens of factors that state law and rules required it to. And it had made a well-reasoned decision.
“The PDAB has done extensive stakeholdering to come to this decision today,” said Hope Stonner, the policy manager for the Colorado Consumer Health Initiative, “and that shows the process is working.”