Dear Dr. O’Toole:
On behalf of the nearly 40,000 children and adults living with cystic fibrosis — including over 1,000 in Utah, Idaho, and Nevada — we write in response to Select Health’s changes to the coverage criteria for elexacaftor/tezacaftor/ivacaftor (ETI/Trikafta®). We urge Select Health to cover elexacaftor/tezacaftor/ivacaftor for all people with CF who meet eligibility requirements per the Food and Drug Administration (FDA) label. We urge the removal of the requirement to try and fail other modulator therapies. Additionally, we request the removal of clinically inappropriate prior authorization criteria, specifically around lung function parameters. Access to this treatment should not be restricted to patients with FEV1 between 40–90% because it is effective in patients with lower FEV1 to preserve and extend life without transplantation and patients with higher to prevent initial progression of disease. We request you continue to make this transformative therapy available according to its FDA approved indications.
About cystic fibrosis
Cystic fibrosis is a life-shortening genetic disease resulting from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that causes the body to produce thick, sticky mucus that clogs the lungs and digestive system. There is no cure for CF today. As a complex, multi-system condition, CF requires targeted, specialized treatment and medications. 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 and 35 affiliate programs nationally. Given the progressive nature of cystic fibrosis, it is imperative that people with CF have timely access to health care to minimize disease progression and prevent any health declines.
About Elexacaftor/Tezacaftor/Ivacaftor
For eligible patients, elexacaftor/tezacaftor/ivacaftor is the most significant therapeutic advancement in CF to date. This oral therapy addresses the underlying cause of cystic fibrosis — CFTR protein defects — in individuals with specific mutations in the CFTR gene. Among the CFTR modulators, elexacaftor/tezacaftor/ivacaftor is considered highly effective and is associated with improvements in lung function and body mass index and decreased exacerbations which in turn leads to stability in lung function. Longer-term data supports that these improvements are sustained over time. 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 U.S. Food and Drug Administration (FDA) label. It is imperative that elexacaftor/tezacaftor/ivacaftor is initiated as soon as patients and their physicians determine it is medically appropriate as it may prevent or slow future CF complications.
Concerns with criteria requiring failure on other modulators
We ask Select Health to continue to cover elexacaftor/tezacaftor/ ivacaftor for all people with CF who meet eligibility requirements per the FDA label. Clinical trial results provide strong evidence that elexacaftor/tezacaftor/ivacaftor is highly effective in treating CF, greatly exceeding results from other modulators for most mutations. Elexacaftor/tezacaftor/ivacaftor offers significant improvements even to individuals already stable on tezacaftor/ivacaftor (Symdeko®), with data showing an additional 10-point increase in lung function as measured by FEV1 for patients eligible for treatment by either. Further, people taking elexacaftor/tezacaftor/ivacaftor recorded a 13.8-point increase in FEV1 and a 64% reduction in pulmonary exacerbations, far exceeding the 2.8-point FEV1 change and the 30% reduction in exacerbations experienced by those on lumacaftor/ivacaftor (Orkambi®). The evidence supports that treatment-naïve patients and those who are currently on another modulator experience significant benefit clinically from elexacaftor/tezacaftor/ivacaftor. Considering these data, step therapy is clinically inappropriate; delays in treatment may further disease progression and irreversible organ damage caused by CF.
Restricting access to patients with FEV1 between 40-90%
Coverage criteria that restrict access to this therapy according to specific FEV1 range is misguided and clinically inappropriate. For patients with FEV1 values above 90%, elexacaftor/tezacaftor/ivacaftor represents the opportunity to preserve health lung function through reduction in CF pulmonary exacerbations, and avoid the permanent, irreversible damage that characterizes CF and leads to early death. Preserving lung function extends survival when the chance of high quality of life is highest. Restricting access requires these individuals to suffer a decline in lung function and quality of life before starting this modulating therapy. Further, this requires individuals near the 90% threshold — who may fluctuate above and below this value — to consistently get sicker before getting back on drug.
Alternatively, patients with FEV1 of 40% or lower could be characterized as those who may experience the most significant clinical benefit. For all patients with cystic fibrosis, but especially those with diminished FEV1 values, even modest increases in lung function can yield great benefits in health and quality of life. Data also supports use within this population. A clinical trial studying elexacaftor/tezacaftor/ivacaftor’s safety and efficacy included patients with an FEV1 below 40% at baseline. The safety and efficacy in this subgroup were comparable to that observed in the overall population, furthering the evidence that elexacaftor/tezacaftor/ivacaftor is safe and effective for this group of individuals. Other studies have found that for those with advanced lung disease, elexacaftor/tezacaftor/ivacaftor is clinically effective and is associated with rapid improvement that can often delay or negate the need for a lung transplant.
People with cystic fibrosis require timely, uninterrupted access to specialized care and treatments to manage the disease. We urge Select Health to remove clinically inappropriate criteria from their prior authorization forms and continue allowing eligible people with CF to access Trikafta, regardless of their documented use of other CFTR modulators or their FEV1, in line with the FDA labeling.
We stand ready to answer any questions about this or other aspects of CF care and request that we schedule a time to meet to further discuss the unique needs of people with CF.
Thank you for all you do for people with CF.