Colorado lawmakers deciding between access and affordability for ‘Orphan Drugs’

COLORADO SPRINGS, Colo. (KRDO) - The bipartisan Senate Bill 24-060 is making its way through the Colorado Legislature right now. It's the tip of the iceberg of an ongoing debate between patient access and affordability to rare, often life-saving prescription drugs referred to as "Orphan Drugs."

If passed, the bill will not allow the Prescription Drug Affordability Board (PDAB) to review and evaluate "Orphan Drugs" for affordability. Set up in 2021, the PDAB was supposed to save Coloradans money by evaluating the price of prescription drugs and setting an upper payment limit for them.

In 2021, Cystic Fibrosis patient and Colorado Springs resident Amanda Boone was watching the PDAB legislative process closely. That's because a life-saving drug she was taking, Trikafta, was set to be reviewed for patient affordability first. Boone explained that she currently pays nothing for the drug that's essentially reversed the debilitating symptoms of her rare and chronic condition.

"They were concerned Trikafta would leave the state, the makers of my drug would take it out of the state and they would not be able to dispense it," Boone said.

Earlier that year, Boone said she had gotten a call from her pharmacist notifying her that depending on the outcome of the PDAB's decision, he wouldn't be able to sell the drug anymore. If she doesn't take her dose of the drug on the hour, Boone risks declining into a worse state than she was before the drug, which was weeks from death.

Essentially, according to Boone, there is no guarantee that when the PDAB sets payment limits on certain drugs, those pharmaceutical manufacturers wouldn't pull out of distributing the medication to the state entirely because of the financial ramifications.

She and other members of the 600-adult person community of Cystic Fibrosis patients in Colorado lobbied against the board setting the price limit on the medication and won. She will testify to the State Senate tomorrow with her story during SB24-060's next hearing, lobbying for its success.

She says she hopes no other chronically ill person ever has to go through that strain and work she did to ensure she could continue to live in the state. Two years before that legislative battle, Amanda had moved her five-person family to Colorado because it was notorious for excellent care for Cystic Fibrosis.

She's an example of an especially vulnerable community in Colorado: patients who live with rare diseases and rely on Orphan Drugs, defined as drugs without viable alternatives. Those patients are vulnerable because of the high price tags that often come with the life-saving drugs given that designation, and if cut from access, risk serious health decline.

Colorado Consumer Health Initiative is on the other side of the argument, lobbying against the bill.

They've been working for the healthcare rights of Coloradans for years in the state, and see Senate Bill 24-060 as a way for pharmaceutical companies to continue with the monopoly they've held for years, continuing to squeeze patients for extra money.

"We are opposed to this bill specifically because the Orphan Drug designation is a misnomer and more than anything, this designation has allowed pharmaceutical manufacturers to monopolize drugs on the marketplace while also letting them set outrageous prices with minimal intervention," a spokesperson for the organization said in an email statement Wednesday.

According to a fact sheet sent over by the organization, the bill would exclude 67% of the drugs up for review by the PDAB because "they have some form of Orphan designation."

“Managing a rare illness is already a huge financial burden for patients. My family spends tens of thousands of dollars every year between a series of primary and specialized healthcare professionals and unexpected emergency room visits, just to keep my husband’s health somewhat controlled,” said Tamika Matthews, a member of the Colorado Consumer Health Initiative’s Community Advisory Board. “On top of those costs, the annual prices for the orphan drugs that could treat his condition are completely unattainable for us. We look forward to when my husband’s drugs are up for review. Prescription drugs don’t work if people can’t afford them."

A three-way standoff between the PDAB, pharmaceutical companies, and patients who rely on orphan drugs in Colorado will continue in the legislature on Thursday when the bill is evaluated by the State, Veterans and Military Affairs Committee.

Boone and other patients with rare illnesses or who rely on orphan drugs are set to testify to the Colorado Legislature tomorrow.