NICE suspends review of Vertex’s Symkevi for cystic fibrosis

The UK’s National Institute for Health and Care Excellence (NICE) has suspended its review of Vertex Pharmaceuticals’ cystic fibrosis (CF) drug Symkevi (tezacaftor/ivacaftor) after the company failed to submit evidence to NICE regarding the clinical and cost-effectiveness of the therapy.

As a result, NICE has decided against reviewing Symkevi at a committee meeting in November. In its appraisal overview, NICE wrote: "Vertex Pharmaceuticals has not provided an evidence submission for this appraisal. Therefore, we are suspending the appraisal whilst we consider the next steps. Consequently the discussion of this appraisal at the committee meeting on the 8 November 2018 has been cancelled."

Symkevi is a combination therapy indicated for CF patients with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: two copies of the F508del mutation or one copy of the F508del mutation and one of 14 other mutations. It is the first drug that treats the CFTR protein defect in CF patients.

It received positive option from the European Medicines Agency’s Committee of Medical Products for Human Use in July 2018, but is yet to receive approval from the European Commission.

The drug was approved in February 2018 by the US Food and Drug Administration and in June 2018 by Health Canada under the name Symdeko.

Vertex responded to NICE’s decision by saying: "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."

Cystic Fibrosis Trust chief executive David Ramsden 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.

NICE’s suspension of its review of Symkevi follows two years of discussion between Vertex and the UK’s National Health Service (NHS) over the pricing of three cystic fibrosis drugs Orkambi (ivacaftor/lumacaftor), Symkevi and Kalydeco (ivacaftor).

The latest round of negotiations over Orkambi in July were inconclusive despite Vertex offering a pricing deal to the NHS over its current and future cystic fibrosis drugs.

Despite continued calls from patient groups, Vertex has refused to reveal the price it offered the NHS on the basis it has signed a non-disclosure agreement. However, NHS England has called on Vertex to waive the confidentiality clause.

Ramsden continued: "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."

This led Vertex CEO Jeff Leiden to write a letter to the UK Prime Minister Theresa May asking her to personally intervene and claiming the UK placed lower value on the cystic fibrosis than any other country.

A NICE spokesperson said the agency is "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."

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