MMIT Reality Check on Cystic Fibrosis

Payer Coverage:

A review of market access for cystic fibrosis treatments shows that under the pharmacy benefit, about 65% of the lives under commercial formularies are covered with utilization management restrictions. Around 32% of the lives under Medicare formularies are not covered for at least one of the drugs.

cystic-fibrosis-pharmacy

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.

cystic-fibrosis-medical

For about 93% of the covered lives, payer pharmacy benefit formularies do not require step therapy (ST). Of the lives that require ST, 22% require multiple steps. Around 77% of payer-controlled pharmacy benefit covered lives require prior authorization, with 24% of those lives covered by policies that are restrictive as compared with a product’s FDA-approved label.

cystic-fibrosis-pa

Trends:

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.

FDA Expands Trikafta Label

In December 2020, the FDA expanded the label of Vertex Pharmaceuticals Inc.’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include people with cystic fibrosis (CF) at least 12 years old with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that are responsive to Trikafta based on in vitro data. The agency also expanded the label of Symdeko (tezacaftor/ivacaftor and ivacaftor) to include additional mutations in people with CF at least 6 years old, as well as Kalydeco’s (ivacaftor) label to expand treatment to people at least 4 months old. The changes will make more than 600 new people eligible for treatment. Drugs.com lists the price of 56 tablets of Kalydeco and 84 tablets of Trikafta as $24,957.06, while 56 Symdeko tablets are listed at $23,395.10.

FDA Expands Kalydeco Label

In September 2020, the FDA expanded the label of Vertex Pharmaceuticals Inc.’s Kalydeco (ivacaftor) for use in children ages 4 months to less than 6 months old with cystic fibrosis 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. Drugs.com lists the price of 56 packets of oral granules at all three doses as $24,957.06.

Key Findings:

Market Events Drive Changes

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 (CF) 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. In December 2020, the agency expanded the label of Trikafta to include people with CF at least 12 years old with certain mutations in the CFTR gene that are responsive to Trikafta based on in vitro data. The agency also expanded the label of Vertex’s Symdeko (tezacaftor/ivacaftor and ivacaftor) to include additional mutations in people with CF at least 6 years old, as well as Kalydeco’s (ivacaftor) label to expand treatment to people at least 4 months old.

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. Contracting is prevalent among the antibiotics; competition will increase as more antibiotics enter the CF market.

Pharmacy, Medical Benefit Implications

Good formulary coverage exists for most products in this class. The majority of prior authorization is defined by diagnosis; few policies will require a step through a preferred generic agent. Coverage is primarily under the pharmacy benefit, but nebulizer solutions may also be covered under the medical benefit.

Key Players in Market:

cystic-fibrosis-players

0 Comments

AIS Health Staff

Related Posts

https://www.mmitnetwork.com/wp-content/uploads/2021/11/wordpress-featured-image-reality-check-chronic-lymphocytic-leukemia.jpg
November 5

MMIT Reality Check on Chronic Lymphocytic Leukemia

Read More
https://www.mmitnetwork.com/wp-content/uploads/2021/10/wordpress-featured-image-reality-check-hepatocellular-carcinoma.jpg
October 29

MMIT Reality Check on Hepatocellular Carcinoma

Read More
https://www.mmitnetwork.com/wp-content/uploads/2021/10/WordPress-Featured-Image-reality-checl-psoriasis.jpg
October 22

MMIT Reality Check on Psoriasis

Read More

GAIN THERAPEUTIC AREA-SPECIFIC INTEL TO DRIVE ACCESS FOR YOUR BRAND

Sign up for publications to get unmatched business intelligence delivered to your inbox.

subscribe today