Cystic Fibrosis Drug Offers Glimpse of Future for Precision Medicine

Ivacaftor, the first drug that targeted the underlying defect of cystic fibrosis (CF), which affects about 30,000 people in the United States, improved outcomes and reduced hospitalizations, according to an analysis in the May issue of Health Affairs examining precision medicine.

CF is an incurable, life-threatening genetic disease that is caused by a defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which regulates the movement of ions and water in and out of cells. The mutation causes secretions to accumulate in the lungs and digestive tract, resulting in severe respiratory and digestive problems and potentially contributing to complications like diabetes or infections.

The authors used claims data to examine the role and potential of precision medicine, and said the drug “effectively treats a subpopulation of people with cystic fibrosis based on their unique genetic makeup.” This can help providers, policy makers, and patients assess the value of treatments relative to their cost and use, they said, especially as eligibility for the drug expands by patients’ age and genetic mutation.

The drug was first approved by the FDA in 2012 and the FDA expanded the label to include additional variants in 2014 and, again, in 2017. The label expansions meant that 14% of the CF population were eligible to take the drug, which has a list price of $311,000.

Click to continue reading on The American Journal of Managed Care.