(Colorado Newsline) My parents didn’t plan on having a child born with a rare disease — no one does.

The rarity of these conditions often means limited research, scarce treatment options, and constantly searching for anything that could help manage the disease.  The past 33 years since my diagnosis has been filled with countless tests, hundreds of doctors visits, and filling multiple prescriptions every month. But my family was luckier than many people in the U.S. affected because my rare disease, cystic fibrosis, has had several groundbreaking treatments discovered, changing my life forever. 

What’s more, because my family lives in Colorado, we have access to great hospitals and medical research facilities and a governor who has made helping people with rare diseases one of his priorities.

But that luck looks to be running out, because Colorado’s Prescription Drug Affordability Review Board is using bad math and making risky assumptions that could put my access to my lifesaving treatment at risk.

When Gov. Jared Polis was first elected in 2018, I was ecstatic that Colorado had elected someone who made a point of joining the Rare Disease Caucus when he served in Congress. Surely that meant that things could only get better. In addition to his thoughtful handling of the COVID pandemic, I was thrilled to see him sign the PDAB bill into law and create a Rare Disease Advisory Council here last year.

But now as the PDAB is transitioning from ideas to implementation of its goal — to make prescription drugs more affordable — orphan drugs to treat rare diseases like I have, have unintentionally gotten caught in the middle, despite the law creating the PDAB explicitly stating that orphan drug status should be considered as part of the evaluation process.

PDABs like the one here in Colorado have gained traction as a tool for states to assess the cost-effectiveness and affordability of medications and help drive down costs for patients. While the intention behind these boards is to ensure that drugs are accessible, applying this approach to orphan drugs would have unintended consequences for those of who are impacted by rare diseases.

Rare diseases affect a small percentage of the population, often fewer than 200,000 individuals in the United States. The small patient population means that research and development costs are spread thinly across a limited number of individuals. The costs associated with conducting extensive trials and navigating the review process pose significant financial risk to companies, particularly when the patient population is small. 

If the PDAB subjects orphan drugs to price caps, we risk suppressing innovation and depriving rare disease patients of potential breakthrough therapies. Unlike more prevalent conditions, such as diabetes or heart disease, rare diseases often lack standardized treatment protocols or off-label options. For many, the available treatments are very limited (in some cases there is just one) and losing access would leave our family stranded in a sea of uncertainty without any viable solutions. 

One of the primary concerns with PDABs is that they prioritize cost-effectiveness over other factors. While cost considerations are essential, the value of orphan drugs cannot be solely determined by their price tags. For those of us impacted by rare diseases, these medications are our lifeline, offering a chance at a better quality of life or even survival. Prior to being put on my most recent drug regimen, I struggled catching my breath and had a massive lung bleed, which made it necessary for me to have a pulmonary embolization.  Today, I enjoy going to Orange Theory, running and hiking with my family, and have the ability to do so for decades to come — which is far more than I had ever dared to dream.

That’s why I’m a firm believer that the value of a treatment should encompass its impact on patients’ lives, the level of unmet medical need, and the potential to improve long-term outcomes.

Polis has been a champion for our community throughout his years of public service. We need him and the PDAB to stay true to their goals. If the aim is to ensure that patients can access lifesaving medications, then Colorado should not consider making moves that could jeopardize that access for the thousands of people with rare diseases who live in Colorado.  

Colorado Newsline is part of States Newsroom, a network of news bureaus supported by grants and a coalition of donors as a 501c(3) public charity. Colorado Newsline maintains editorial independence. Contact Editor Quentin Young for questions: info@coloradonewsline.com. Follow Colorado Newsline on Facebook and Twitter.