MMIT Reality Check on Cystic Fibrosis (4Q2022)
Payer Coverage:
A review of market access for cystic fibrosis treatments shows that under the pharmacy benefit, about 63% of the lives under commercial formularies are covered with utilization management restrictions. Around 20% of the lives under Medicare formularies are not covered for at least one of the drugs.
Under the medical benefit, about 9% of the lives under commercial policies are covered with utilization management restrictions. Almost 89% of the lives under Medicare Part B policies have access to at least one of the drugs without utilization management restrictions.
For about 94% of the covered lives, payer pharmacy benefit formularies do not require step therapy (ST). Of the lives that require ST, 25% require multiple steps. Around 76% of payer-controlled pharmacy benefit covered lives require prior authorization, with 16% of those lives covered by policies that are restrictive as compared with a product’s FDA-approved label.
Trends:
FDA Expands Orkambi Label
In September 2022, the FDA expanded the label for Vertex Pharmaceuticals Inc.’s Orkambi (lumacaftor/ivacaftor) to include the treatment of cystic fibrosis in people ages 12 to <24 months who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The agency first approved the drug on July 2, 2015. The medication is available in tablet and oral granule formulations. Dosing for the newest indication in people between the ages of 2 and 5 who weigh less than 14 kg is one 100 mg lumacaftor packet and ivacaftor 125 mg granules every 12 hours; for those at least 14 kg, dosing is one 150 mg lumacaftor packet and ivacaftor 188 mg granules. In people ages 6 through 11, dosing is two 100 mg tablets of lumacaftor and two 125 mg tablets of ivacaftor every 12 hours. Drugs.com lists the price of 56 units of 100 mg/125 mg and 150 mg/188 mg oral granules as more than $23,116. It also lists the same price for 112 100 mg/125 mg and 200 mg/125 mg tablets.
Researchers Examine CF, UC/Crohn’s Adherence, Say Specialty Pharmacies ‘Could Help Reduce Medical Burden’
Two studies of specialty-drug treated conditions examined the impact of adherence on hospitalizations and medical costs. Findings of the studies — one on cystic fibrosis (CF) and the other on ulcerative colitis (UC)/Crohn’s disease — from AllianceRx Walgreens Prime (which changed its name to AllianceRx Walgreens Pharmacy in late June) demonstrate the importance of specialty pharmacy interventions in helping keep patients adherent to therapy.
FDA Gives Trikafta Another Indication
In June 2021, the FDA gave another indication to Vertex Pharmaceuticals Inc.’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis who are 6 to 11 years old and have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene or a mutation in the CFTR gene that is responsive to Trikafta based on in vitro data. The agency also approved a new dosage strength of the tablet: elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg and ivacaftor 75 mg. The FDA first approved the drug on Oct. 21, 2019.
Key Findings:
Market Events Drive Changes
In September 2022, the FDA expanded the label for Vertex Pharmaceuticals Inc.’s Orkambi (lumacaftor/ivacaftor) to include the treatment of cystic fibrosis (CF) in people ages 12 to <24 months who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. In June 2021, the FDA gave another indication to Vertex’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with CF who are 6 to 11 years old and have at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive to Trikafta based on in vitro data.
Competitive Market Landscape
CFTR modulators are the first type of CF therapy to treat the root cause of the disease rather than the symptoms. Although costly, these therapies have made a large impact on market value. New-to-market products will compete with multiple brand inhaled aminoglycoside and monobactam antibiotics, CFTR modulators and inhaled mucolytics.
Pharmacy, Medical Benefit Implications
Good formulary coverage exists for most products in this class. Coverage is primarily under the pharmacy benefit, but nebulizer solutions may also be covered under the medical benefit. Most plans cover the products with a prior authorization once diagnosis and CFTR mutation requirements are met. Some policies require step through preferred generic or brand CF agent, bronchodilators or pancreatic enzymes supplements.
Key Players in Market:
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