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# When will Trikafta become available for Cystic Fibrosis patients on the NHS (England)?

### Question

Cystic Fibrosis is a genetic disease affecting mainly the lungs and the pancreas.(1) In 2012 an American company called Vertex Pharmaceuticals got approval from the FDA to market a drug called Ivacaftor, the first generation of "CFTR Modulators" which improves lung function in people with CF.(2) It was initially priced at $300,000 a year making it one of the most expensive drugs on the market. This was followed by Orkambi ($270,000 a year) in 2015,(3) and then in October 2019 by Trikafta, a triple combination therapy which is effective in around 90% of people with CF. Trikafta is marketed at around \$300,000. However, the initial evidence is that it is much more effective at improving lung function in CF patients than previous therapies.(4)

Because of the cost of Orkambi, NICE, the organisation responsible for evaluating drugs affordability and effectiveness decided that the drug did not meet its quality of life standards and recommended not fund it.(5) NHS England and Vertex then negotiated to get a deal to reduce the price, but it took until October 2019 for NHS England to reach a deal to make Orkambi available to all eligible CF patients.(6) This question asks:

When will Trikafta become available for Cystic Fibrosis patients on the NHS (England)?

By 'available for Cystic Fibrosis patients', we mean that there are at least 5535 English CF patients who must have access to Trikafta via the NHS.

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