The girl loves to move — cycling and snowshoeing and swimming — but when seizures grip her body, the movement stops.
Her heart rate soars above 170 beats per minute and her blood oxygen level plunges below 70%. She gasps for air. She hunches forward.
And when the seizure cluster finally ends, sometimes many minutes after it began, she looks over to her mother, kneeling beside her, and whispers, “Mommy. Scary. Scary.”
Heather Kluck, who lives in Colorado Springs, calls her 12-year-old daughter, Avery, “my miracle child.” When Avery was diagnosed as an infant with a rare genetic condition called Aicardi syndrome, doctors told Heather and her husband that Avery may not survive her first year and would likely never walk or talk. She defied those predictions, though she has significant developmental delays.
But the miracle is fragile, and the seizures, which have been getting worse as she nears her teen years, could end it. To help quiet the seizures, doctors have recommended that Avery start on a drug called Sabril, a powerful anticonvulsant that can cost as much as $10,000 per month.
Because of that extraordinary price, Sabril is on the radar of a Colorado board seeking to control the cost of prescription drugs. The Prescription Drug Affordability Board, or PDAB, reviews whether drugs are unaffordable and has the ability to set caps on what patients and insurers have to pay for them.
To Kluck, though, this could be a curse to patients. If the caps cause pharmaceutical companies to pull their drugs from Colorado — something at least one company has already threatened — or causes pharmacists to stop stocking them, then patients like Avery might end up harmed.
And, so, Avery has not yet started taking the drug that may save her life. Kluck worries: What if she begins and then we can’t get it anymore? What would happen if Avery had to quickly stop a drug that is notably dangerous to wean off?
“We’re to a point now where her seizures are getting more violent, and this is our last resort,” Kluck said. “And now I’m finding out she may not have access to it.”
☀️ READ MORE
Surprising opponents
Colorado lawmakers created the PDAB three years ago to address a persistent problem: A big chunk of Coloradans aren’t taking their medicines because they can’t afford to.
The most recent edition of the Colorado Health Institute’s Colorado Health Access Survey found that nearly 11% of people in 2023 did not fill a prescription because of cost. That number has remained more or less unchanged since the survey began in 2009.
“The original goal of the PDAB is to increase access to drugs,” said Hope Stonner, a policy coordinator at the Colorado Consumer Health Initiative, which supports the PDAB. “Folks aren’t taking drugs because of cost.”
There are a handful of states that have created PDABs to address rising drug costs, but Colorado’s may be the furthest along. Last year, the board began conducting reviews of specific drugs to determine whether they are unaffordable — a task made all the more difficult because some patients may pay very little for a drug and others, especially those who are uninsured, may pay a huge amount for the same drug.
If the board does declare a drug unaffordable, it can then move to set a so-called upper payment limit, which would cap how much patients and insurers have to pay for a drug — and effectively limit how much a manufacturer or a pharmacist can charge. No PDAB in the U.S. has ever done this, but Colorado’s is currently debating whether to become the first to set a price cap, on the arthritis drug Enbrel.
Pharmaceutical companies are opposed to the PDAB. Enbrel’s maker, Amgen, sued Colorado this year to strike down the PDAB’s authority.
But people who suffer from rare diseases — or, like Kluck, care for those who do — surprisingly have emerged as some of the most passionate opponents of the PDAB’s work. They are not opposed to the idea of the PDAB, they say, and they are not defending the price of their drugs. Instead, they say the PDAB has brought stress into lives already full of it.
Their concerns center on the potential unintended consequences of the PDAB’s actions. Would their drugs become unavailable? Would manufacturer assistance programs that many patients rely on to pay for their drugs dry up?
“It’s an experiment, and it’s really gross that they’re doing it on people who are really sick,” said Jennifer Reinhardt, a Littleton mother whose daughter lives with cystic fibrosis and relies on the drug Trikafta.
The stress of uncertainty
Last year, the PDAB considered whether to declare Trikafta unaffordable, and members of the cystic fibrosis community organized in force to tell the PDAB not to.
They told of how Trikafta, a breakthrough therapy, had improved lives. They worried they may have to move out of state if a PDAB-imposed price cap meant the drug became unavailable here.
Along the way, Trikafta’s manufacturer, Vertex, seemed to validate their fears when it wrote in a letter to the PDAB: “Given the national market architecture for drug pricing, as a consequence of the PDAB’s rules, manufacturers subject to an (upper payment limit) may have no practical choice but to withdraw from Colorado.”
Ultimately, the PDAB decided not to declare Trikafta unaffordable, citing Vertex’s patient-assistance programs. But the feeling of trauma lingered for patients.
“It was so torturous for our family,” Reinhardt said.
Others, such as Kluck, say that just the potential for a PDAB review causes them stress.
Avery’s new anti-seizure medication, Sabril, is currently ranked 114th on the PDAB’s list of 604 drugs eligible for review. Given that the PDAB has only completed three affordability reviews in its three years and may set upper payment limits on at most 18 drugs in a year, it could be years before it gets to Sabril, if ever. (Due to her disability, Avery is covered by Medicaid, meaning her family would not have to pay for Sabril now and a price cap would not directly benefit them.)
For a family dealing with a serious disease, that is still too much uncertainty, Kluck said. Every seizure Avery has is life-threatening. Kluck, as Avery’s full-time nurse and caregiver, is almost always by her daughter’s side — her entire life organized around protecting her child’s health.
“We shouldn’t be playing with people’s lives,” she said.
Patients championed a bill at the state Capitol this year that would have exempted so-called orphan drugs — designated by the Food and Drug Administration as treating rare diseases — from a PDAB review. That bill stalled amid concerns it would wipe out a huge swath of drugs eligible for review, but supporters have launched a petition hoping to revive it.
A compromise bill, Senate Bill 203, which would require the PDAB to consult with rare disease advocates before selecting drugs for review, appears headed for passage. But Kluck and other patients worry it doesn’t provide enough protection.
The bill had its final committee hearing on Monday, a day earlier than originally scheduled as the legislature scrambles to fit everything into its calendar in the closing days of the session. Kluck said she didn’t learn about the hearing change until after it happened — and Avery had a doctor’s appointment, so she couldn’t have attended on short notice anyway. No one showed up to testify on the bill.
“That’s a bully tactic.”
The state’s Division of Insurance, which oversees the PDAB, declined to make board members available for comment. Division staff also declined to comment, citing the pending lawsuit.
During a confirmation hearing earlier this year at the state legislature, Amy Gutierrez, a pharmacist who serves on the PDAB, defended the board’s work.
“We need to do a better job at educating the public that what we’re trying to do is to help them,” she said. “It’s not to create a barrier.”
Gutierrez accused pharmaceutical companies of fearmongering, something that Stonner, the CCHI policy coordinator, echoed.
“Frankly, that’s a bully tactic, and I think that shows the need for the board in the first place,” she said.
Stonner said the board, which is made up of doctors, pharmacists and health care administrators, has been moving thoughtfully and carefully. It is common to hear even opponents praise board members’ earnestness.
She said the board is also highly responsive to patient feedback. It conducts patient surveys and holds patient stakeholder meetings before it even begins to conduct an affordability review, with more opportunity for comment after that.
“This idea of drugs becoming unavailable raises some questions when you’re sitting at the table and understanding the thoroughness,” she said.
But patients have also raised concerns that the mechanics of the supply chain, not the whims of pharma companies, could lead to unintended consequences. An upper payment limit applies only to what the final purchaser — patients, insurers or both — pay for a drug. So what happens if pharmacists have to pay more to get a drug than they can charge a patient to buy it, thus forcing them to sell the drug at a loss?
When Gutierrez, who is the chief pharmacy officer for UCHealth, asked that question during a meeting last week, board staff provided no guarantees, instead promising a thorough review to help the board make the best decision.
“When you all decide on the upper payment limit, you will have a lot of this information in front of you,” Lila Cummings, the PDAB’s director, told Gutierrez.
To Kluck, that kind of answer brings no comfort. She said she understands that prescription drugs are too expensive — one of the major contributors to rising insurance costs. She understands that people deserve to be able to afford their medication.
She doesn’t understand how her family got caught in the middle of it.
“Why take the risk of putting people’s lives at stake if you don’t need to?” she said. “That’s what we’re all not understanding.”