CF drug Kalydeco took up 86% of Scotland’s rare disease fund

Spending on Vertex’s cystic fibrosis drug Kalydeco accounted for the vast majority of Scotland’s Rare Conditions Medicines Fund (RCMF) last year.

An investigation by the Scotland’s Sunday Herald newspaper found that £18.6 million ($26.5m) was spent on Kalydeco (ivacaftor) in the 2013/14 financial year – representing 86% of the special fund.

The newspaper article raises once again concerns about the high price of drugs for rare diseases. Kalydeco costs around £180,000 a year per patient per year, making it one of the world’s most expensive drugs.

The drug was first approved in 2013 to treat a subset of CF patients with the G551D mutation, and (now extended to patients with eight rare non-G551D gating mutations), making just a few dozen patients in Scotland eligible for treatment.

Kalydeco was paid for through the RMCF after being rejected by cost effectiveness watchdog the Scottish Medicines Consortium.

The £18.6 million is understood to cover two year’s worth of the drug, but clearly raises questions about the sustainability of the fund.

The Sunday Herald asked the Edinburgh government for a list of the 10 drugs that had taken up most of the budget and a breakdown of cost. The request was refused by ministers until the Scottish Information Commissioner ordered the data to be released.

The information showed that Alexion’s Soliris (eculizumab) was second in the list, with £660,000 spent on it, followed by Celgene’s Imnovid (pomalidomide) in third place with £455,000 of spending.

The RCMF has already been replaced with the New Medicines Fund, which has a budget twice as big, £80 million for the 2015/2016 financial year which ends on 31 March.

The newspaper sought out a comment from David Miller, a Sociology professor at Bath University, who accused Vertex of being a ‘profiteer’ from the NHS, and called for more transparency in decision making.

A spokesperson for Vertex said: “The price of Ivacaftor is based on the benefits the medicine brings by treating the underlying cause of the disease, the very small number of people eligible for the medicine and, importantly, our commitment to and significant investment in continued research to develop new medicines to help even more people with CF.”

The news comes just days after NICE, the cost effectiveness watchdog for England and Wales, rejected Vertex’s Orkambi, (a combination of ivacaftor and new agent lumicaftor). Orkambi is licensed to treat people aged over 12 years who have the F508del mutation, a more common subtype of the disease, meaning more patients are eligible for treatment.

Read the Sunday Herald story here

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