New Breakthrough Cystic Fibrosis Drug Promising for Patients, Challenging for Benefit Plans

New Breakthrough Cystic Fibrosis Drug Promising for Patients, Challenging for Benefit Plans


The FDA has announced the approval of Trikafta (elexacaftor/ivacaftor/tezacaftor), the first triple combination therapy to treat the most common form of cystic fibrosis. This new drug has the cystic fibrosis (CF) community excited because it is expected to treat 90% of the CF population. For comparison, another recently developed drug to treat CF, Kalydeco, can only treat about 6% of the CF population because it targets a different genetic marker.

Cystic fibrosis is a rare genetic disease that produces thick mucus in the lungs, digestive tract, and other parts of the body. This thick mucus causes difficulty breathing, digesting food and ultimately leads to many infections and secondary diseases such as diabetes. Trikafta treats the most common genetic mutation that causes CF and helps the defective gene protein work more effectively.

While it is a rare disease and cystic fibrosis patients typically have shorter life spans, the new drug has great potential for extending lives.  In the U.S., it is expected that Trikafta will help nearly 27,000 people with CF and it will have a price tag of around $26,000 per month.

One key strategy for managing that $312,000 annual cost per patient is to ensure your pharmacy benefit has a pharmacogenomic program. This works similar to a prior authorization program, but requires confirmation of the genetic marker in a patient to ensure Trikafta is right for them. Without this in place, your pharmacy plan could be paying for this drug for patients that will not benefit from it. In fact, due to the rarity of this disease and the genetic component, we would expect to see Trikafta on most PBM’s pharmacogenomic prior authorization program and specialty drug lists.


Topics: Trends, Insights & Analysis, Prescription Drug News & Regulations - Employers

Posted by

Michelle Gerlovin, PharmD.

Senior Client Services Director
Michelle is a clinical pharmacist with a passion for improving outcomes while controlling healthcare costs. She has worked in various pharmacy practice and pharmacy benefit management settings. Michelle's experience includes pharmaceutical rebate analysis, drug utilization management, and strategic pharmacy benefit support for government clients.