CF patients furious as Vertex turns back on NICE

A group of cystic fibrosis patients could be denied access to a cutting-edge therapy after manufacturer Vertex refused to submit data to cost-effectiveness body NICE.

The manufacturer's decision prompted an angry response from patients worried that Vertex is making good on a veiled threat to withdraw from the UK unless it gets a higher price for its CF drugs.

NICE allows manufacturers to submit data for its assessments once drugs are recommended by Europe’s CHMP scientific committee.

This allows NICE to begin its assessments ahead of a European marketing authorisation, which nearly always follows within about three months of a CHMP positive opinion.

But in this case Vertex has refused to file data with NICE for its latest combination therapy Symkevi (tezacaftor+ivacaftor), which was backed by the CHMP at the end of July.

The new drug combination broadens the population of patients who are eligible for a therapy that treats the underlying cause of the disease.

It means that patients with two copies of the F508del mutation, or one copy of F508del and one of 14 other mutations, can receive therapy that corrects residual activity of the CFTR protein.

Vertex said it will not file the data until NICE changes the way it assesses drugs following a row that has raged on for more than two years over the price of its CF drugs.

The company said in a statement: “Vertex supports the need for robust and fair medicines appraisal in England but believes that NICE’s single technology appraisal has not kept pace with changes in medicine and has significant limitations in how it captures and values the full benefits of precision medicines such as tezacaftor/ivacaftor.”

“We would be happy to re-engage with NICE in the context of an appropriate appraisal process, which takes into account the structure of an innovative framework contract for all of Vertex’s cystic fibrosis medicines, on which we are currently negotiating with NHS England.

“Vertex remains committed to working with all stakeholders to ensure all eligible patients with CF in England get rapid access to all our current and future medicines.”

A spokesperson for the patient group ukneedsorkambi said: "We are naturally concerned by this development, particularly if it signals Vertex withdrawing from the UK which would be inconceivable and inhumane."

"The concern is that this signals a refusal from Vertex to contribute to NICE appraisal, such an action would clearly jeopardise ongoing negotiations with NHS England, which we hoped were to continue soon with a meeting between (NHS England chief executive) Simon Stevens and (Vertex CEO) Jeffrey Leiden."

David Ramsden, the chief executive of the Cystic Fibrosis Trust, said: “This is awful news for thousands of people in the UK with cystic fibrosis and it is a situation that I find deeply concerning.

“In the US, they have been able to get Tezacaftor/Ivacaftor since February, but this action makes access in the UK seem even more distant.

“People with cystic fibrosis are already being denied access to Orkambi, another life enhancing drug. Next week marks 1,000 days since it was licensed in the UK, yet it is still not available on the NHS.

“Every day that people in the UK are unable to access new precision medicines is a day too long and it means people who could see their lives prolonged are dying.”

In 2016, NICE rejected Vertex’s Orkambi (ivacaftor+lumacaftor) for patients with two copies of the F508del mutation.

Since then Vertex has offered NHS England a deal covering all of its cystic fibrosis drugs, and any approved in the future.

The price has not been disclosed, but pharmaphorum understands there is considerable distance between the two parties and the matter remains unresolved.

Vertex's CEO Jeff Lieden last month wrote to prime minister Theresa May, saying the NHS was devaluing CF patients.

The letter was seen as threat to pull out of the UK, as Lieden said that future investment in the country is "at risk" because of the ongoing row.

A NICE spokesperson said: “We stand ready to work with Vertex on both a review of our guidance on Orkambi, when the company is able to offer a price that reflects its value to patients and the NHS, and to start the evaluation of Symkevi (tezacaftor/ivacaftor), a new option in the treatment of cystic fibrosis.”