Patients, members of Colorado House debate placing price caps on 'orphan drugs'
Placing upper payment limits on orphan drugs will either increase or decrease their access and affordability, depending on who you ask.
Orphan drugs are defined as medications used to treat conditions that affect 200,000 people or fewer in the U.S. These orphan diseases, as they're known, encompass both widely recognized illnesses such as muscular dystrophy, cystic fibrosis and ALS, as well as lesser-known conditions like Duncan's Syndrome and Madelung's disease. Because these medications are not widely used among the American population, they are not considered profitable by pharmaceutical companies and can often cost thousands of dollars for a single prescription.
In 2021, the legislature established the Prescription Drug Affordability Review Board, or PDAB. The Board is responsible for reviewing the most expensive drugs on the market and establishing upper payment limits. Although PDAB currently has the authority to review orphan drugs, it sparked controversy last year when it considered placing a price cap on Trikafta, a drug commonly used for cystic fibrosis treatment.
Although the board ultimately decided against the price cap, the mere consideration raised significant concerns for many of the nearly 700 Coloradans living with cystic fibrosis, as several of them testified during a Senate Health and Human Services Committee meeting to discuss Senate Bill 060.
The bill, which has bipartisan sponsorship from Senators Barbara Kirkmeyer, R- Brighton, and Joann Ginal, D- Fort Collins, would repeal PDAB's authority to perform affordability reviews and set upper payment limits for orphan drugs.
"For those folks who need these drugs, that 10% of the people who have a rare disease in the United States that have the opportunity for a drug that gives them a life, that's what we're here for," she said, noting that 90% of those living with rare diseases have no treatment available. "Patients and advocates have expressed over and over their concerns about the PDAB involvement in their disease drugs."
Kirkmeyer said it often takes up to 15 years and costs billions of dollars to develop a new drug, and she believes implementing price limits on drugs hampers research as pharmaceutical companies may no longer find it financially viable. Her co-sponsor Senator Ginal reiterated that orphan drugs are not developed for economic reasons and noted that of the eight states that have, or are, in the process of passing PDAB bills, three of them have created review exemptions for orphan drugs. She also mentioned several emails she and some of her fellow legislators have received in opposition to the bill, which she said contained false statements.
"It's concerning to me. It should be concerning to all of us because this really is misinformation," she said of the emails, which she believed to be sent through a third party as any responses her aide sent bounced right back.
Ginal said the emails contained information claiming that the bill would "destroy" PDAB and prevent it from reviewing the prices of commonly used drugs, both of which are untrue.
"This is not about minimizing PDAB," she said. "What it is trying to do is be able to keep orphan drugs available to that small group of people that are lucky to have some of these drugs."
Some family members of individuals living with rare diseases said they support the bill, asserting that removing the option of upper payment limits for orphan drugs preserves the incentive for research into potential new drugs to treat orphan diseases. According to Kelly Wiberg, whose daughter has cystic fibrosis, there are many copay programs and grants available to help alleviate the financial burden associated with these drugs.
Jen Reinhardt, whose daughter Maya also has cystic fibrosis, said her family considered moving to a state with orphan drug cap exemptions during PDAB's Trikafta deliberation. "We shouldn't, as a society, as the United States, say 'hey, your disease is too expensive, so we're gonna let you die We're gonna sacrifice these 400 people in CO in the name of affordability,'" she said. "We need someone to stand up and protect people with rare diseases. A genetic therapy for a sole condition isn't the same as something that treats millions of people where the pharmaceutical companies are gauging people. Until we have access protections, please stop doing this to us, it's not helpful."
Amanda Boone, who moved from Texas to receive treatment for her cystic fibrosis at Denver's National Jewish Health, said she was spending two weeks at a time in the hospital hooked up to oxygen before Trikafta.
"This medicine saved my life," she said. "I'm not putting it lightly; it did save my life. I am no longer on oxygen, I am healthy, and I gained 30 pounds. I don't live in the hospital anymore; I haven't been there in over a year."
Boone said she had friends in Canada and the United Kingdom who died from cystic fibrosis while awaiting government negotiations with drug manufacturers. She said if Colorado were to engage in similar price deliberations, she and other CF patients might not be adequately protected.
"Excluding orphan drugs from the PDAB review process will continue to deny rare disease patients access to these life-changing drugs that don't break the bank," said Tamika Matthews of the Colorado Consumer Health Initiative, whose husband has sickle cell disease. "Patients with rare diseases are the ones who are most harmed by uncontrolled costs."
Other patients and advocates argued that placing price caps on orphan drugs would actually increase accessibility. Rose Keller, a college student who has cystic fibrosis, said she began taking Trikafta last month. "My continued access to the drug is made more secure, not less, by the PDAB's ability to review orphan drugs," she said.
Keller said that because Vertex, the manufacturer of Trikafta, holds a patent on the drug, the company will not face any market competition until at least 2037.
"Without safeguards like the PDAB affordability review, who is to stop them from raising Trikafta's list price even more?" she asked. "As a young adult living with a rare disease, I worry that I will not be able to afford my medications when I go off my parents' health insurance in just a few years.
"In the long term, allowing pharmaceutical companies to price gauge beyond any semblance of affordability causes harm to patients, CF and otherwise. Ultimately, patients will be forced to delay or go entirely without treatment if these pricing trends continue. I am not comfortable burdening my community with rising healthcare premiums just to satiate Vertex's near avarice," she continued.
Kaitlyn Tollefson with Young Invincibles shared her experience living with early-onset prurigo nodularis, an immune disorder characterized by painful skin nodules. She said there is only one clinical treatment available for the disease, and it would cost $12,000 per dose without insurance.
"While the creation of rare disease treatment is necessary, if the solution is unaffordable to most people, then it is useless," she said. "It is crucial to make a price cap on orphan drugs; these medications are difficult to access now and will be entirely impossible to access if the cap is removed."
Austin Blumenfeld of Centennial State Prosperity said he was surprised to learn that the drug recommended for his psoriasis, Humira, is considered an orphan drug despite it being used for widely diagnosed conditions like arthritis, Chron's disease, and ulcerative colitis. He also said that 67% of the approximately 600 drugs eligible for review by PDAB are considered orphan drugs.
"This is a national strategy for pharmaceutical companies to make it impossible for PDABs across the country to bring down the cost of prescription medications like these," he said. "Give our PDAB a chance to work before changing law or creating exemptions."
