Fury as NHS rejects cystic fibrosis drug price offer

It may be the 70th birthday of the NHS but this was no day of celebration for a group of patients with cystic fibrosis who have been told by officials there is no way forward in a pricing row over the revolutionary drug Orkambi.

There are now widespread calls for health secretary Jeremy Hunt to resolve the matter as it is now more than two years since NICE rejected Vertex’s Orkambi (ivacaftor+lumacaftor), which treats the underlying cause of CF in patients with the F508del mutation.

It is also three years since Orkambi was authorised for use by European regulators.

For the last two years Vertex and NHS England have been in negotiations over a pricing scheme for Orkambi, which will also cover all future CF drugs from the US biotech.

But despite suggestions that a deal was imminent after a high-profile campaign and a debate in Parliament earlier this year, Vertex has released a statement saying that representatives of NHS England and from the company are still unable to agree on a deal at a meeting yesterday (Wednesday).

Vertex said it is unable to reveal details of the price that is on the table because of a non-disclosure agreement, but said that the offer it had made would create “budget certainty” for the NHS and allow patients speedy access to future drugs as they are approved.

Despite the confidentiality clause Pharmaphorum understands that NHS England and Vertex are still far apart in their thinking on price.

Health secretary Jeremy Hunt has called on the pharma company to waive its confidentiality agreement and reveal the price.

NHS England has also asked Vertex to reveal the price to allow public scrutiny, even though the company claims that the non-disclosure agreement was imposed by NHS officials.

In the meantime patients frustrated with both the manufacturer and NHS England are calling for health secretary Jeremy Hunt to intervene.

Christina Walker, whose eight year-old son Luis has CF and is eligible for treatment with Orkambi, said: “The NHS is supposed to be saving lives. The patient group is hugely disappointed, they are clearly miles away from each other on price.”

“Jeremy Hunt met with the CF trust the other day and we thought this was going to be a breakthrough. This is heartbreaking, where do we go from here?”

“Our cystic fibrosis does not stop because it’s the NHS’ anniversary day. We think that 20 patients have died since March. We are calling for Jeremy Hunt to intervene, time is ticking.“

Rebecca Hunt, vice president for corporate affairs at Vertex, said: “It’s so deeply frustrating. The CF community has been waiting for the outcome of this meeting. We are hugely disappointed.”

“To hear from the people we met with saying the NHS did not see a path forward is massively disappointing. I am calling on Jeremy Hunt and (NHS chief executive) Simon Stevens to get involved.”

“They are heralding a new era of precision medicines. Here we are on the 70th birthday of the NHS with NHS England denying access to these medicines. This is the best offer in the world.”

“It’s hugely disappointing the NHS England has not moved at all. They are holding us to the same threshold that they have stuck to from the beginning.”

Vertex said it is particularly keen to resolve the issue because of the high prevalence of the cystic fibrosis mutation in the country – it is more common in patients of Celtic descent and the UK has the second highest incidence of the disease in the world after the US.

An NHS England spokesperson said: “This country has a long established, internationally respected independent body NICE which recommends whether or not a drug company is proposing a fair price.  NICE has been clear that Vertex’s pricing is unsupportable. If Vertex really believe they are offering a reasonable deal they should waive their confidentiality clause and let patients and taxpayers judge whether it is fair.”

David Ramsden, the Cystic Fibrosis Trust's Chief Executive, said: “We are appalled and deeply frustrated to receive Vertex and NHS England’s statements. People with cystic fibrosis and their families have fought hard  to get these medicines."

“After years of being forced to wait, the cystic fibrosis community will simply not accept either party walking away from the table without striking a deal."

“Vertex and NHS England must get back around the table quickly and ensure these medicines are made available to people with cystic fibrosis.”