Scotland strikes CF drug deal with Vertex, rest of UK still waiting

Patients in Scotland will gain access to Vertex’s cystic fibrosis drugs Orkambi and Symkevi, after the company signed an access agreement with health officials.

Officials have signed the deal despite rulings from the country’s Scottish Medicines Consortium (SMC) that the drugs were not cost-effective.

Financial details of the deal are not publicly available, but Vertex said that it would last five years, and that it has committed to collecting real world data on the drugs that will support any future submissions to the SMC.

But while this is good news for patients in Scotland, patients south of the border are still unable to get the medicines because of an ongoing stand-off over pricing.

Vertex has not said whether it offered a discount to get the deal with Scottish authorities over the line, but details could emerge in the coming weeks as they will have to be shared under the voluntary drug pricing scheme applying across the UK.

There are only 900 people in Scotland with CF, and this deal means that around 400 patients eligible for Orkambi or Symkevi will be able to receive Vertex’s drugs.

But with more than 10,000 people living with CF in the UK, most of the 6,000 eligible patients live in England, where the budget impact would be much higher for the country’s devolved NHS.

CF campaigners hope that this deal paves the way for the rest of the UK to gain access, but are concerned that such an agreement could not be reached in England because of the much higher costs involved.

Wales and Northern Ireland have already said they will follow the SMC’s lead and not fund the drugs, and there is no end in sight to the impasse in England where politicians have much less influence over how the NHS operates.

Campaigner Christina Walker, whose son Luis, 9, has CF and is eligible to receive Orkambi, said: 'I'm absolutely delighted for patients in Scotland, too much health has been lost waiting.

“Of course for my family and the other home nations patients we all want to know if this helps us. As there's a responsibility to share the detail of the deal with the other health authorities. NHSE will soon know how much over what was considered a cost effective price Scotland have paid and whether it's supportable in England, where there are ten times the number of patients.

“If Vertex has moved sufficiently then we call on NHSE to make a deal urgently. If not, then the various routes to supply patients with generic alternatives must be put into action immediately.'

A Vertex spokesperson said: “We are obliged by the terms of the agreement to keep the details of the pricing confidential. The authorities in Scotland worked with us in a collaborative and flexible way to find a solution so patients can have access to our precision medicines.”

Orkambi works in patients with two copies of the F508del mutation, while Symkevi works in CF patients whose CFTR protein is misfolded and cannot move to the cell surface.