Legal status The US
Food and Drug Administration (FDA) approved ivacaftor in January 2012. Soon after, the
European Medicines Agency (EMA) Canada, and several European countries did so as well. Lumacaftor/ivacaftor was approved by the FDA in July 2015, under
breakthrough therapy status and under a
priority review.
Economics It is one of the most expensive drugs, costing over per year, which has led to criticism of the high cost. The cost of ivacaftor is per year, roughly similar to the price of other medications for extremely rare diseases. In the first nine months of its second year on the market (2014), ivacaftor sales were $339M, representing 54% of Vertex's product sales revenue. During the same period, total drug development expenses were $458M, most of which was spent on cystic fibrosis-related research. An editorial in JAMA called the price of ivacaftor "exorbitant", citing the support by the
Cystic Fibrosis Foundation in its development and the contribution made by fundamental scientific research performed by the
National Institutes of Health and relied upon by Vertex in its cystic fibrosis drug discovery programs. The company responded in an email that "while publicly funded academic research provided important early understanding of the cause of cystic fibrosis, it took Vertex scientists 14 years of their own research, funded mostly by the company, before the drug won approval." The Cystic Fibrosis Foundation, a non-profit organization dedicated to improving healthcare for people with cystic fibrosis, provided $150 million of the funding for the development for ivacaftor in exchange for royalty rights in the event that the medication was successfully developed and commercialized. In 2014, the Foundation sold these royalty rights for $3.3 billion. The Foundation has stated that it intends to spend these funds in support of further research. Vertex said it would make the medication available free to patients in the United States with no insurance and a household income of under $150,000. In 2012, 24 US doctors and researchers involved in the development of the medication wrote to Vertex to protest the price of the medication, which had been set at about $300,000 per year. In the UK, the company provided the medication free for a limited time for certain patients, then left the hospitals to decide whether to continue to pay for it for those patients. UK agencies estimated the cost per quality adjusted life year (
QALY) at between £335,000 and £1,274,000 —well above the
National Institute for Health and Care Excellence thresholds. The medication was not covered under the
Ontario Drug Benefit plan until June 2014, when the
Government of Ontario and the manufacturer negotiated for what "Ontario Health Minister
Deb Matthews had called a "fair price" for taxpayers". The negotiations took 16 months and it was estimated that around 20 Ontarians required the medication at the time. The province of
Alberta began covering the medication in July 2014, and in September the province of
Saskatchewan became the third province to include it in its provincial medication plan. Delay in agreement on a price for Vertex to charge national health plans led to patient group protests in Wales, England, and Australia. , cost $259,000 a year in the United States. ==Research==