- Prime Therapeutics has estimated that a new combo treatment from Vertex for cystic fibrosis (CF), with Kalydeco (ivacaftar)as a backbone, would cost the healthcare system $4.5 billion.
- The FDA is reviewing a drug combination treatment for specific genetic variants of CF. The treatment, which includes Kalydeco, a $307,000 per year drug, could come as soon as July.
- The new combination treatment would greatly expand the pool of eligible CF patients beyond the small pool of patients who are eligible for Kalydeco alone.
When Kalydeco was approved by the FDA in January 2012 it was a welcome breakthrough for patients with CF, but it came with a hefty pricetag of more than $300,000. However, for the relatively few patients who could actually be treated with Kalydeco, the drug has made a huge difference. It dramatically improves breathing and reduces infections.
Kalydeco is approved for 10 cystic fibrosis transmembrane conductase inhibitor (CF-TR) mutations. But only about 4%—or roughly 2,400 people in the U.S.—are eligible for treatment with the drug. In contrast, the new combination treatment from Vertex would treat a gene mutation found in more than 80% of CF patients—but the treatment is only for patients aged 12 and older. This would push the treatment population in the U.S. up to 8,500 patients.
According to pharmacy benefits manager, Prime Therapeutics, the cost increase will be staggering and from a comparative pharmacoeconomic perspective, almost untenable. For example, Prime has estimated that even if other medical costs are eliminated, pharmacy costs could increase by $150 million per year based on eligible patients taking the new combo, and that overall 28-year treatment costs could exceed $8 million per person. Compared to the cost of treating CF using conventional treatment methods, which are roughly $76,000 per year as long as the patient does not receive a lung transplant, these costs are overwhelming to most PBMs.
However, Vertex maintains that it is providing a game-changing therapeutic option for patients with CF that not only extends their life expectancy, but also allows them to function in society in a more productive way. As advances in gene-based medicines continue, costs for specialty medications undoubtedly lead to sticker shock, but as hard as it is, it's not an awful problem to have.
CORRECTION: A previous version of this article misstated the number of mutations that Kalydeco can treat, as well as the percentage of CF patients who may benefit from the new combination treatment. Those errors have been fixed and this post has been updated.