Dear Dr. Kudisch:
On behalf of the more than 650 people with cystic fibrosis that rely on the Texas Medicaid program for some or all of their coverage, we write to urge the Drug Utilization Review (DUR) Board to add additional coverage for inhaled tobramycin and aztreonam lysine for inhalation (AZLI), critical components of CF care. We thank the state for covering these inhaled antibiotic therapies for people with CF aged six and older and request expanded coverage for those under the age of six. Expert clinicians providing specialized care to people with CF in Texas have expressed concerns that some patients are unable to access these medically necessary therapies if they are under six years of age. Inhaled tobramycin and AZLI serve to eradicate Pseudomonas aeruginosa and prevent or delay chronic pseudomonal infection of the airways. It is imperative that all people with CF can access these therapies to prevent damage due to bacterial infection.
Cystic fibrosis is a progressive, genetic disease that affects the lungs, pancreas, and other organs. There are close to 40,000 children and adults living with cystic fibrosis in the United States, and CF can affect people of every racial and ethnic group. CF causes the body to produce thick, sticky mucus that clogs the lungs and digestive system, which can lead to life-threatening infections. As a complex, multi-system condition, CF requires targeted, specialized treatment and medications.
Medicaid is a crucial source of coverage for 45% of children in Texas and provides access to vital care and treatments — including inhaled tobramycin and AZLI — to help people with CF maintain their health and well-being.
About inhaled antibiotic therapies
The buildup of mucus in the lungs makes people with cystic fibrosis more likely to develop bacterial infections. The most common bacterial infection is Pseudomonas aeruginosa with 25% of people with CF culturing positive for this bacterium in 2023. These infections can last for short periods of time or for many years, and to manage these infections, people with CF take antibiotics as a part of their regular daily treatment. Both inhaled tobramycin and AZLI have been established as effective therapy for fighting P. aeruginosa and are recommended for use in people with mild, moderate, and severe lung disease. These treatments can be delivered at home, are less nephrotoxic, and would avoid the need for hospitalization and treatment with IV antibiotics.
Inhaled tobramycin is commonly used in people with CF under the age of six, despite the Food and Drug Administration’s indication being for people with CF aged six and older. Fifteen percent of children under six years of age inhaled tobramycin. The safety and efficacy of inhaled tobramycin is well demonstrated for this population; it has been studied in children as young as one year of age and has been found to reduce P. aeruginosa density and is not associated with any renal toxicity or ototoxicity. Further, oral antimicrobial therapy (fluroquinolone medications) is contraindicated in children and thus is not a treatment option.
Tobramycin can only be used for a 28-day course, and patients must wait another 28-day cycle before taking a subsequent course. During the 28-day interval without tobramycin, AZLI is the only other CF-specific inhaled antibiotic available to meet patients’ medical needs. It is important to note that tobramycin and aztreonam treat P. aeruginosa through different mechanisms of action. According to the 2023 CF Foundation patient registry report, 2.3% of children between the ages of three to five and 1% of children under three are prescribed AZLI.
The clinical practice guidelines for preschoolers with CF recommend use of alternating tobramycin and AZLI therapy for the management of infants and children with new and chronic P. aeruginosa infections.
Coverage of inhaled antibiotics
Currently, Texas Medicaid does not cover inhaled tobramycin or AZLI for children under six years of age. For this population, the only alternative option to fight a P. aeruginosa infection is hospitalization with IV antibiotics — in 2023 the median duration of a hospital stay for IV antibiotic treatment in individuals less than 18 years of age was 10 days. This is a costly and burdensome alternative to the child with CF, their family, and their insurance provider. Not eradicating P. aeruginosa can result in chronic infection leading to declines in lung health and recurrent future hospitalizations at substantial cost to the healthcare system. We urge the DUR Board to expand coverage of AZLI (Cayston®) and at least one formulation of nebulized tobramycin (such as generic tobramycin, Kitabis® Pak, TOBI®, or Bethkis®). TOBI® Podhaler with capsules is not an option for young populations as it requires an inhalation technique on behalf of the person with CF that is not possible for infants.
The Cystic Fibrosis Foundation appreciates your commitment to ensuring access to vital treatments for people with CF in Texas. We would be happy to connect you with our clinician partners at local CF care centers to further discuss inhaled tobramycin and its use in CF care.