- A rare disease fund set up by the Scottish government to help pay for uncovered drugs spent 86% of its budget between 2013 and 2014 on Vertex's Kalydeco, according to a report from the Scottish Herald.
- The Rare Conditions Medicines Fund, now replaced with the New Medicines Fund, reportedly dedicated £18.6 million of its £21.7 million in funding toward purchasing two years worth of the cystic fibrosis drug. Although the exact price paid by the National Health Service (NHS) in Scotland for the drug is unclear, the drug can cost over $300,000 per patient per year elsewhere.
- In the U.S., Kalydeco is only approved to treat a small subset of patients with specific types of cystic fibrosis mutations. But it can substantially improve breathing and reduce infection by targeting the underlying genetic mutations
The Rare Conditions Medicines Fund was set up to pay for rare disease drugs which the Scottish Medicines Consortium had rejected on cost-effectiveness grounds. It is now organized as the new Medicines Fund, and its budget has swelled to £80 million for 2015-2016.
Although Kalydeco can offer substantial benefit to CF patients, it is only approved to treat a relatively small population. The U.S. FDA estimates the drug's current indications cover about 3,400 people in the U.S., Canada, Europe, and Australia. Overall, about 75,000 people in those regions have cystic fibrosis.
Kalydeco's high price has led to criticism and, as the Scottish Herald report illustrates, it can have an outsize impact on government drug budgets.
Compared to the 86% share in 2013-2014, spending on Kalydeco now consumes a smaller proportion of the Scottish fund's budget, according to the report.