By Amanda Boone

There are moments in life that leave an indelible mark on your soul. For me, one came in the middle of the night when I took a full breath for the first time in over a decade. At first, I was confused — then I realized what it was. I woke my husband, who sprang to action, fearing the worst after years of living in survival mode. But this time, it was good news. Everything was about to change.

Living with cystic fibrosis (CF) has always been a rollercoaster. In my younger years, I managed relatively well despite frequent hospitalizations and treatments. I built a career, moving from leasing agent to property manager, while quietly fighting to stay healthy.

Over time, that fight intensified. I was diagnosed with CF-related diabetes and began experiencing frequent lung bleeds. After a severe flu in 2014, my health declined rapidly. I entered a cycle of infections, hospitalizations, and surgeries, eventually forcing me to step away from work.

Seeking better care, I moved from Texas to Colorado for treatment at National Jewish Health. There, I was diagnosed with mycobacterium abscessus, a serious infection requiring long-term IV antibiotics. My health continued to deteriorate, and I began preparing for a double lung transplant.

Then, in October 2019, everything changed.

A breakthrough therapy, Trikafta, had just been approved. I wasn’t sure it would work for me, but within four days of starting it, I woke up, took a deep breath, and saw my lung function jump by ten percent. I immediately started crying — I knew my life was changing.

Soon after, I was told I no longer needed a transplant. For the first time in years, I felt hope. While I’m not cured, the progression of my disease has been halted. This therapy is my lifeline.

In the summer of 2023, just as I was beginning to rebuild my life, that lifeline was threatened. My medication came under review by Colorado’s Prescription Drug Affordability Board (PDAB), which was considering a price cap, called an Upper Payment Limit (UPL). This arbitrary cap would not reduce what I paid — insurance companies are not required to pass on any rebate to the patient — but because of the way drug pricing is structured nationally, it would make it infeasible for manufacturers to offer this revolutionary therapy in the state. If no agreement was reached with the manufacturer of my drug, patients like me could lose access altogether.

I stepped into advocacy, joining others in the CF community to form a grassroots non-profit called CF United. We shared our stories, met with lawmakers, and fought to protect access to our treatment. It was a grueling and traumatizing process. In December 2023, we won: the board determined the drug was “not unaffordable,” and a price cap was not imposed.

But the victory came with uncertainty. The board can bring these drugs back for review at any time. For patients like me, that means living with the constant fear that the medication keeping us alive could be taken away. Would I have to uproot my family and leave the state?

Rare disease patients should not have to spend their limited time fighting to keep access to the treatments that sustain them. Other states with PDABs (Oregon and Washington) have recognized this and created protections for rare disease therapies. Colorado can — and should — do the same.

That’s why SB26-140 has been introduced this legislative session, to exempt rare disease drugs from the PDAB’s purview.

This bill would create safeguards to ensure patients with rare, life-threatening conditions are not harmed further by cost-control policies. It offers a path forward where affordability and access can coexist — without putting lives at risk. The PDAB was originally designed to lower drug costs but has not been able to achieve that in the past five years. At a time when our state is facing budget deficits and cuts to Medicaid, healthcare is already bearing the brunt — and along with it, the most vulnerable among us. We cannot afford to compound that harm by putting access to lifesaving treatments at risk.

I am one of the lucky ones. Many people with rare diseases are still waiting for their breakthrough. For cystic fibrosis alone, about 10% of patients don’t yet benefit from therapies like Trikafta. They desperately need fewer restrictions on the development and production of these medicines, not more.

We can’t stop fighting until every person with a rare disease has access to a lifesaving therapy — and the protection to keep it. Because without those protections, we risk discouraging the very innovation that makes these breakthroughs possible in the first place.

Amanda Boone is an individual with cystic fibrosis and the co-founder of CF United, a grassroots organization ensuring affordable access to therapies for CF and rare disease patients.