MMIT Reality Check on Cystic Fibrosis (4Q2023)
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 21% 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 88% of the lives under Medicare Part B policies have access to at least one of the drugs without utilization management restrictions.
For about 93% of the covered lives, payer pharmacy benefit formularies do not require step therapy (ST). Of the lives that require ST, 24% require multiple steps. Around 77% of payer-controlled pharmacy benefit covered lives require prior authorization, with 15% of those lives covered by policies that are restrictive as compared with a product’s FDA-approved label.
Trends:
FDA Expands Patient Population for Kalydeco
In May 2023, the FDA expanded the patient population for Vertex Pharmaceuticals Inc.’s Kalydeco (ivacaftor) to include the treatment of children ages 1 month to less than 4 months old who have at least one mutation in their cystic fibrosis transmembrane conductance regulator gene that is responsive to Kalydeco based on clinical and/or in vitro assay data. The drug already is approved for patients at least 4 months old. The agency first approved the agent on Jan. 31, 2012, and it is available in five different unit-dose packets of granules and as a 150 mg tablet. Dosing for the new population is one 5.8 mg packet of oral granules every 12 hours for children 1 month to less than 2 months who weigh at least 3 kg. For those aged 2 months to less than 4 months and weigh at least 3 kg, dosing is one 13.4 packet of oral granules every 12 hours. Drugs.com lists the price of 56 packets of 25 mg, 50 mg and 75 mg granules, as well as 56 150 mg tablets, as more than $26,405.
FDA Broadens Patient Population for Trikafta
In April 2023, the FDA broadened the patient population for Vertex Pharmaceuticals Inc.’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include the treatment of children ages 2 through 5 with cystic fibrosis who 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. It previously was approved for use in people at least 6 years old. Dosing for the new patient population in people less than 14 kg is elexacaftor 80 mg/tezacaftor 40 mg/ivacaftor 60 mg oral granules in the morning and then ivacaftor 59.5 mg in the evening. For people weighing at least 14 kg, dosing is elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg oral granules in the morning and then ivacaftor 75 mg in the evening. Drugs.com lists the price of 84 tablets of two different strengths — elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg co-packaged with ivacaftor 75 mg, as well as elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor75 mg co-packaged with ivacaftor 150 mg — as more than $26,405.
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.
Key Findings:
Market Events Drive Changes
In May 2023, the FDA expanded the patient population for Vertex Pharmaceuticals Inc.’s Kalydeco (ivacaftor) to include the treatment of children ages 1 month to less than 4 months old who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to Kalydeco based on clinical and/or in vitro assay data. In April 2023, the agency broadened the patient population for Vertex’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include the treatment of children ages 2 through 5 with cystic fibrosis who 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. In September 2022, the FDA expanded the label for Vertex’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 CFTR gene.
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 a step through a preferred generic or brand CF agent, bronchodilators or pancreatic enzymes supplements.
Key Players in Market:
