Intelligent Solutions in Pharmacy Benefits
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What you may want to know about the drug is the following:
- Only 28% of CF patients (8,500 in the U.S) have the correct genotype for Orkambi to be effective. By comparison, Kalydeco (ivacaftor), another FDA approved CF drug, is effective in 4% of CF patients, (2,000 in the U.S), has been on the market for over a year, and costs about $300K per year per patient.
- The latest information we have is that the FDA Advisory Panel recommended Orkambi for approval, and $287K is the estimated annual wholesale cost. Once patients are started on this drug, they can be expected to continue for the duration of their lives.
- The two active ingredients, lumacaftor and ivacaftor, work together to alleviate the defect of the mutated gene in two steps. Lumacaftor corrects interaction with the CFTR protein and ivacaftor potentiates the effect to keep the CFTR open at the cell surface.
- In a study published in the New England Journal of Medicine, the impact of the drug was as follows: an increase in lung function of 2.6 to 4.0% and a decrease in the rate of pulmonary exacerbations by 30-39%.
- The issue for plans is that only those members being treated for CF with the specific CFTR gene mutation should be treated with this combination drug. The concern is that some prescribers will try this drug before confirming genotype and the drug will be ineffective at a cost of about $25K/month. This was seen when Kalydeco first came on the market.
- PBIRx Clinical agrees that this drug is a clear candidate for a Prior Authorization strategy, to confirm use only in appropriate members.
What you may want to know about the disease CF is the following:
- There are 30,000 estimated CF patients in the U.S.
- Cause of the disease is a mutated CFTR gene causing thick, sticky mucous which impairs breathing. This results in inflammation and recurrent pulmonary bacterial infections, and sometimes death.
- CF is a hereditary disease. When both of the two CFTR genes in a person have mutated, the person will have CF. If just one gene mutates, the person is a carrier for subsequent generations.
- CF affects mostly the lungs, but the pancreas, liver, intestines, and kidneys can also be impacted.
- While in 1955 the median age of survival with CF was 5 years, by 1985 it was 25 years, and by 2012 it became 41 years.
- Current treatments include: vitamins; painkillers; antibiotics; and Kalydeco (for 4% of CF patients) to maintain daily activities.
For more information about this new drug and how it may affect your pharmacy benefit plan, please give us a call at (888) 797-2479.