New cystic fibrosis drugs may significantly increase pharmacy benefit costs

April 15, 2015

New cystic fibrosis treatments, that target the gene mutations causing the disease, will significantly increase healthcare costs, according to a study presented at the Academy of Managed Care Pharmacy (AMCP) 27th Annual Meeting and Expo, in San Diego.

New cystic fibrosis treatments, that target the gene mutations causing the disease, will significantly increase healthcare costs, according to a study presented at the Academy of Managed Care Pharmacy (AMCP) 27th Annual Meeting and Expo, in San Diego.

Cystic fibrosis is a genetic disease affecting multiple organ systems, most importantly the lungs and airways. An estimated 30,000 children and adults in the United States have the disease, which can lead to life-threatening lung infections and the inability to absorb nutrients from food. Some individuals may need a lung transplant because of progressive damage to the lungs.

In 2012, the first treatment to target a specific gene mutation, ivacaftor (Kalydeco), was FDA approved. Ivacaftor treats a type of gene mutation found in about 4% of people with cystic fibrosis and costs $300,000 per year. A new combination drug-ivacaftor plus lumacaftor-is pending FDA approval for treatment of people aged 12 years and older who have the gene mutation found in nearly 50% of people with cystic fibrosis. The price of the new drug is not yet public, but is widely expected to be similar to ivacaftor’s price.

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According to pharmacy benefit manager Prime Therapeutics LLC (Prime) analysis of more than 13 million commercially insured members, the mean total medical and pharmacy cost for individuals using ivacaftor is more than $367,000 per year. If a person with cystic fibrosis continued to take ivacaftor for 28 years (40 years is the approximate life expectancy) they would accumulate $8 million in drug costs alone. For those patients with cystic fibrosis who received a lung transplant, total medical plus pharmacy costs in the year of transplant averaged nearly $577,000, while annual costs averaged $143,000 per year following transplant. Members who had not received a lung transplant and were not being treated with ivacaftor had total medical and pharmacy costs that averaged about $76,000 per year.

The study found that 3.7% of its members with cystic fibrosis who were aged 6 years and older were taking ivacaftor. The new combination drug would treat people aged 12 years or older. Further analysis found the prevalence of cystic fibrosis among Prime’s commercial members aged 12 eyars and older was 9.6 per 100,000. Based on these results, an estimated 4.5 per 100,000 members would meet criteria for the new combination drug when it becomes available. Prime researchers estimated that 90% of these members would receive the new drug, at an anticipated cost of $300,000 per year (similar to ivacaftor). This would result in an increase of about $1 per member per month in pharmacy costs, or approximately $150 million per year in new costs.

Dr Gleason“Ivacaftor and the expected new drug ivacaftor plus lumacaftor are breakthrough drugs that provide individuals with improved quality of life,” said Patrick Gleason, PharmD, director of health outcomes at Prime “With the expected 10-fold increase in individuals with cystic fibrosis expected to be eligible for ivacaftor plus lumacaftor drug therapy at a talked about price of $300,000 a year per individual treated, the costs will likely result in health insurance premium increases as the drug treatment is not expected to substantially offset medical expenditures in comparison to its cost.”

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