- Kalydeco (ivacaftar), an oral, twice-daily medication developed by Vertex Pharmaceuticals, is one of the biggest game-changers for patients with cystic fibrosis, literally expanding lung capacity and decreasing need for hospitalization in patients with certain genetic mutations. However, at $307,000 per year, some payers, including the National Institute of Care and Excellence (NICE) in the UK are denying treatment to patients.
- Kalydeco is routinely available to around 360 patients with the G551D mutation of CF.
- Ed Owen, CEO of the Cystic Fibrosis Trust, told the Pharma Times that not covering the medication is "indefensible." He characterized Kalydeco as a drug that could not only increase quality of life, but also provide a survival benefit that would normalize life expectancy in patients able to get Kalydeco.
The funding crisis in the UK's health system has been well publicized. However, advocates are asking NICE to extend coverage to an additional 44 people.
Based on an analysis presented by Biopharma Dive in July 2014, only about 4% of the 70,000 people in the world are eligible for treatment with Kalydeco, a very personalized form of treatment. Yet for these patients, Kalydeco changes everything.
In reality, only a small number of people will ever be treated with Kalydeco, so costs can be contained—and perhaps negotiated. This is what the Cystic Fibrosis Foundation and eligible CF patients are counting on.