Orphan Drug Access Still Eludes Some Cystic Fibrosis Patients

November 3, 2016

More than a year after a breakthrough drug for cystic fibrosis became available, fewer than half of patients who meet the Food and Drug Administration’s criteria can actually access the medication.

A study released at October’s 30th Annual North American Cystic Fibrosis Conference reported that, in 2015, only 42 percent of eligible patients received the new combination therapy, lumacaftor/ivacaftor. The combination drug treats patients who have not just one but two copies of a gene mutation that leads to the rare disease.

According to “Uptake of Lumacaftor-Ivacaftor Use among Eligible CF Patients in the US in 2015,” patients who did not receive treatment were more likely to be older than 30, have better lung function and have required fewer antibiotics that year. The treatment is approved for patients 12 and older.

Cystic fibrosis causes mucus to cover the lungs, making it difficult to breathe. The disease is life threatening and often fatal, though advances in treatment have prolonged life expectancies and improved quality of life.

Advanced cystic fibrosis treatments are known as “orphan drugs” because of the small patient population they treat.   For example, ivacaftor monotherapy, a twice-daily drug approved by the FDA in 2012, treats a cystic fibrosis mutation carried by only about 1,100 people in North America.

Ivacaftor’s price tag led some Medicaid systems, most notably Arkansas’, to control costs by complicating access to treatment. Arkansas patients reportedly had to first show 12 months of failure on an older cystic fibrosis treatment before qualifying for ivacaftor. In the face of legal action, the staterevised its eligibility requirements in 2015.

Unlike earlier medications, ivacaftor and lumacaftor/ivacaftor address the mutations that cause the disease – not just the disease’s symptoms. Other, more traditional treatments include steroids, anti-inflammatory drugs and antibiotics to treat infection. Some patients may also use a mechanical vest to clear mucus from the lungs.

Widespread access to treatment will likely require continued advocacy and outreach to policymakers. Though Medicaid coverage for advanced cystic fibrosis treatments has improved, many patients and physicians still face prior authorization processes of varying complexity to access the drug.

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