Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Cystic Fibrosis (CF).

Treatment Trikafta 50-25-27.5 and 75 milligrams (mg) or tablet therapy pack (TBPK).

The insurer denied coverage Trikafta for50-25-27.5 and 75 milligrams (mg) or tablet therapy pack (TBPK).

The denial is upheld.

This is a child with a diagnosis of Cystic Fibrosis (CF) who was hospitalized in the neonatal intensive care unit (NICU) for months following surgery for meconium ileus at birth. He has a chronic cough and is treated with daily pulmozyme, ursodeoxycholic acid, inhaled steroids, chest physical therapy and three times a week azithromycin. He underwent a bronchoscopy and was found to have a dynamic collapse of the mid and lower trachea. He has been on Orkambi and has been gaining weight.  His pediatric pulmonologist has requested switching his CF modifier treatment from Orkambi to Trikafta.

Per UpToDate article Cystic fibrosis: Clinical manifestations of pulmonary disease:

"INTRODUCTION - Cystic fibrosis (CF) is a multisystem disease affecting the lungs, digestive system, sweat glands, and reproductive tract. Patients with CF have abnormal transport of chloride and sodium across secretory epithelia, resulting in thickened, viscous secretions in the bronchi, biliary tract, pancreas, intestines, and reproductive system [1,2]. Although the disease is systemic, progressive lung disease continues to be the major cause of morbidity and mortality for most patients. Over a highly variable time course ranging from months to decades after birth, individuals eventually develop chronic infection of the respiratory tract with a characteristic array of bacterial flora, leading to progressive respiratory insufficiency and eventual respiratory failure

Per UpToDate article Cystic fibrosis: Overview of the treatment of lung disease:

"INTRODUCTION - Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are a class of drugs that act by improving production, intracellular processing, and/or function of the defective CFTR [cystic fibrosis transmembrane conductance regulator] protein. These drugs represent an important advance in management of cystic fibrosis (CF) because they target the production or function of the mutant CFTR protein rather than its downstream consequences [1]. Their indications and efficacy depend upon the CFTR gene mutations in an individual patient.

Trikafta is FDA (food and drug administration) approved for treatment of patients with CF with specific mutations that have been documented by double blind clinical trials to improve clinically and through in vitro testing to demonstrate improved function of the chloride channel.

Trikafta is an FDA approved modifier medication that has been rigorously tested and demonstrated to be effective in improving lung function, sweat chloride levels and quality of life in patients with CF over preschool ages. This medication can have some side effects such as headaches, elevation of liver enzymes, liver failure, skin rashes and occasional cases of cataracts. The patient does not currently qualify for this medication because he is only of preschool years.

Vertex Pharmaceutical is currently conducting double blind placebo-controlled studies to determine the appropriate dose and safety of this medication for children with CF under the age preschool ages. These studies are essential to ensure that this medication is safe for younger children and to determine the optimal dosage. It is not the standard of care to provide Trikafta for children under that age.

The health plan acted reasonably with sound medical judgment in the best interest of the patient.

The insurer's denial of coverage for Trikafta 50-25-37.5 and 75 milligrams (mg) or tablet therapy pack (TBPK) is upheld. Medical necessity is not substantiated.