Appeal type: Medical necessity
Decision: Overturned

Diagnosis:  Cystic Fibrosis.
Treatment:  Trikafta.

The insurer denied Trikafta.  The denial is overturned.

The patient is an adult female who has a history of Cystic Fibrosis (CF). At issue is Trikafta, which was denied coverage by the health plan, due to the patient not having the deltaF508 mutation or others considered responsive to Trikafta. According to a letter from her physician, the patient has a diagnosis of Cystic Fibrosis and is well known to her Cystic Fibrosis program at the hospital. She has an extensive requirement for oral and IV (intravenous) antibiotics, doctor visits, and multiple prescription medications with two separate episodes of pneumonia in the past year. Physical assessment revealed continued wheeze, cough, and hemoptysis with recent documented decline in 
pulmonary function and FEV1 (forced expiratory volume 1 second) 61% (percent) predicted. The patient has had recurrent bouts of hemoptysis and required treatment with intravenous antibiotics. The patient carries two rare CF (cystic fibrosis) genes G542X/3849+10KB, whose rarity has delayed their ability to find an effective, approved treatment. She is currently receiving treatment with ivacaftor (Kalydeco), a previously approved CF (cystic fibrosis) modulator. The patient did not tolerate tezacaftor/ivacaftor (Symdeko) which was subsequently also approved for her mutation. Even though the patient is currently taking ivacaftor, she has had a significant decline in health with recurrent hemoptysis, decline in lung function, and pneumonia.

It is the physician's opinion that the compounds in Trikafta will augment the patient's health. She cites three patients in her practice with identical mutations who have done extremely well with transition to Trikafta. The physician further indicates that the patient has attempted and failed all approved therapeutic options for the management of her disease. She indicates that Trikafta has been shown to be effective for the vast majority of Cystic Fibrosis mutations and could be effective for the patient based on data surrounding the drug. Due to the fact that there are over 2,000 mutations that cause CF (cystic fibrosis), it is not feasible to conduct trials in every mutation. Effective management for the patient is hindered by the rarity of her mutations. The patient carries two mutations G542X/3849+10KB which are Class I- production mutation and Class V- quantity mutation. Ivacaftor has proven effective for the Class V mutation 3849+10KB. However, the patient has failed this medication as evidenced by worsening symptomology and pulmonary function. Other appropriate cystic fibrosis therapies for this patient have been maximized, such as mucolytics, mucous hydrators, bronchodilators, airway clearance, and inhaled antibiotics. Trikafta in conjunction with standard CF (cystic fibrosis) therapies has the potential to improve lung function (or slow the rate of decline), reduce the frequency of hospital stays and IV (intravenous) antibiotics, improve quality of life and provide a longer life span for the patient, the mother of two young children. 

The requested Trikafta is medically necessary.

The patient has a well-documented history of CF (cystic fibrosis) but does not have one of the mutations listed as eligible for Trikafta per health plan. Because she has a rare mutation for which extensive testing is not available, her physician has made the fair request that she be trialed on Trikafta after having failed to improve on standard therapy. As a result of her mutations, CFTR (cystic fibrosis transmembrane conductance regulator) protein may have diminished number and function at the cell membrane, which can be augmented by drugs that either function as correctors (Lumacaftor and Tezacaftor) that stabilize the CFTR (cystic fibrosis transmembrane conductance regulator) protein and help protect it from degradation, or potentiators (Ivacaftor), chloride-channel activity at the cell membrane. Lumacaftor, Tezacaftor and Ivacaftor are the three components of Trikafta, which may provide additional benefit that her prior experience with one or more components did not.