Dear Members of Colorado’s Prescription Drug Affordability Board:
On behalf of people living with cystic fibrosis in Colorado, the Cystic Fibrosis Foundation and Colorado’s CF Care Center Directors write to provide comments on the Prescription Drug Affordability Board’s (PDAB) prioritized list of drugs eligible for affordability review. We commend Colorado for taking steps to increase transparency around health care costs and support the Board’s goal of improving access to and affordability of care for Coloradans. We understand the need to address the rising cost of prescription drugs as part of ensuring sustainability of the state’s health care system. As the PDAB conducts affordability reviews and considers use of upper payment limits, we urge you to ensure that people with CF continue to have access to vital therapies, including elexacaftor/tezacaftor/ivacaftor (Trikafta®) and all CFTR modulators.
About cystic fibrosis and the Cystic Fibrosis Foundation
Cystic fibrosis is a life-shortening genetic disease that affects nearly 40,000 children and adults in the United States, including over 700 in Colorado. CF causes the body to produce thick, sticky mucus that clogs the lungs and digestive system, which can lead to lung damage, life-threatening infections, malnutrition, and other complications. Cystic fibrosis is both serious and progressive; lung damage caused by infection is often irreversible and can have a lasting impact on length and quality of life, resulting in extended hospitalizations, transplant, or premature death. As a complex, multi-system condition, CF requires targeted, specialized treatment and medications. There is no cure.
As the world’s leader in the search for a cure for CF and an organization dedicated to ensuring access to high-quality, specialized CF care, the Cystic Fibrosis Foundation supports the development of CF clinical practice guidelines and accredits more than 130 care centers nationally — including two in Colorado.
Modulators mark a significant advancement in cystic fibrosis treatment
For eligible patients, elexacaftor/tezacaftor/ivacaftor is the most significant therapeutic advancement in CF to date, marking a transformation in the nature of the disease. This oral therapy is the most commonly used in a class of drugs called CFTR modulators, which address the underlying cause of CF by restoring CFTR function in individuals with specific genetic variants. As these therapies only work for individuals with certain genetic profiles, they are not interchangeable and there are currently no generic alternatives.
Among the CFTR modulators, elexacaftor/tezacaftor/ivacaftor is considered highly effective and is associated with improvements in lung function, quality of life, and body mass index, as well as reductions in symptom exacerbations. Data from the CF Foundation patient registry shows dramatic declines in both pulmonary exacerbations (episodes of acute worsening of symptoms) and lung transplantation coinciding with the approval of elexacaftor/tezacaftor/ivacaftor: pulmonary exacerbations declined by 65 percent among adults from 2018-2022 and lung transplants declined by 80 percent from 2017-2022 (elexacaftor/tezacaftor/ivacaftor was approved in 2019). Longer-term data supports that these improvements are sustained over time. As a result, we have seen historic, population-wide improvements in quality of life for people with CF as well, as many are newly able to work, attend school, or start a family. For instance, pregnancies among women with CF increased dramatically since 2020; from just 279 in 2018 to 634 in 2022, allowing many to realize a previously unimagined goal of growing a family.
Restoring CFTR function preserves health and lung function, reduces costly hospitalizations, improves quality of life, delays the need for lung transplantation, and improves survival. Cystic fibrosis experts agree that elexacaftor/tezacaftor/ivacaftor is clinically appropriate as a first line therapy for all cystic fibrosis patients aged 2 years and older who have at least one copy of the F508del or other eligible mutations per the FDA label.
Benefits from early initiation of CFTR modulators
Short of a cure for cystic fibrosis, modulators have the potential to dramatically alter the course of this chronic, life-shortening disease, particularly for those who start treatment at a young age. People with CF who start modulator treatment at a young age may be able to restore CFTR protein function to normal levels, thus preventing organ damage, halting the progression of the disease and avoiding future damage. An early start on modulators could have long-term benefits in sustaining health by 1) reducing the rate of lung function decline through prevention of structural damage to the lungs; and 2) improving nutrient absorption and weight gain through preservation of exocrine pancreatic function and normalization of intestinal pH. For these people, we anticipate a life span that approximates that of the general population. Additionally, we anticipate that there is potential to reduce some costs associated with current symptom management and treatment, particularly for those with early modulator initiation.
Importance of continued access to CFTR modulators
As conversations about the cost and value of innovative therapies continue, patients must be at the center of the discussion. We appreciate the PDAB’s stated aims include ensuring that Coloradans continue to have access to prescription medications needed to maintain their health. This is imperative for people with CF; because CF is a progressive disease, patients who delay or forgo treatment — even for as little as a few days — face increased risk of lung exacerbations, costly hospitalizations, and potentially irreversible lung damage.
Elexacaftor/tezacaftor/ivacaftor and CFTR modulators have changed the course of this disease for thousands of people, allowing many to reach previously inconceivable milestones such as working, attending school, or starting a family. It is reasonable that the PDAB should want to take action to compel drug manufacturers to reduce costs, but any solutions to address rising prescription drug costs cannot come at the expense of access to care for these essential therapies for people with CF.
The Cystic Fibrosis Foundation and the undersigned CF physicians in Colorado stand ready to serve as a resource for the Prescription Drug Affordability Board as it explores solutions to improve access to care for Coloradans. As you assess the cost-effectiveness of modulators and host conversations around how these highly effective therapies should be priced, it is critical to consider the lifesaving impact these therapies have already had for so many and the long-term value we expect these medicines will continue to provide.
Thank you for the opportunity to comment on these important issues.