Vertex and NHS close in on cystic fibrosis drug pricing deal

Vertex could be close to a price deal with the NHS over its portfolio of cystic fibrosis drugs, after health ministers wrote to the US firm asking for a conclusion to talks after almost two years of wrangling.

Pharmaphorum understands representatives from the US pharma and NHS England are due to meet in the next few days to resolve a long stand-off over the price of a new generation of CF drugs in the UK.

Ministers are pushing for discussions following an online petition that received more than 116,000 signatures calling for Vertex’s latest CF drug to be made available, which sparked a Parliamentary debate last month.

In a letter to Vertex, junior health ministers Steve Brine and Lord O’Shaughnessy, called for an “urgent resolution” to the matter, although they also praised the company’s “significant contribution” to improving therapies for CF.


The NHS is short of cash, but there is a pressing need for better treatments, particularly as the UK has a relatively high incidence of CF, and the second largest disease population in the world.

It is two and a half years since Vertex’s Orkambi (lumacaftor+ivacaftor) combination for CF patients with certain mutations was approved.

But NICE rejected Vertex’s CF latest drug Orkambi for routine NHS funding in final guidance published in July 2016, saying the drug’s £104,000 per year price tag was too expensive.

Since then Vertex has been trying to reach an agreement with the NHS, and has asked for a deal that would set a price covering approved therapies and those in its pipeline.

With every new drug approved, the proportion of the 10,000 people living with CF that is eligible for treatment will increase.

Vertex is working on a triple therapy that would mean around 90% of the disease population could be treated if trials are successful.

A place to start

Pharmaphorum understands that Vertex has rejected a counter offer from NHS England that would price Vertex’s portfolio at the same level as its already-funded Kalydeco (ivacaftor) – which is available at a confidential discount to the NHS from its list price of around £182,600 per year.

But Vertex believes that the latest offer from NHS England is “a place to start” in the forthcoming negotiations.

In an interview with pharmaphorum earlier this year, Vertex’s senior research fellow and CF drug guru Fred van Goor said securing access to the portfolio of drugs would allow the firm to gather vital real-world evidence because of the UK’s large CF disease population.

“We are very excited about (the deal) for sure. What is important is that patients get access to our medicines,” he told pharmaphorum at the time.

David Ramsden, chief executive at the Cystic Fibrosis Trust said: “We continue to urge all sides to engage meaningfully and in good faith to reach a resolution as quickly as possible so that people with cystic fibrosis can access a potentially life-changing therapy .”

Don't miss your daily pharmaphorum news.
SUBSCRIBE free here.