Vertex “astonished” by NHS England’s response to CF drug

pharmafile | March 20, 2018 | News story | Manufacturing and Production, Sales and Marketing Vertex, biotech, cystic fibrosis, drugs, pharma, pharmaceutical 

Vertex has been battling to have its treatment, Orkambi, approved in the UK since 2016, when it was first rejected by NICE on cost-effectiveness grounds.

The debate has now spilled into UK parliament, after 100,000 signatures were put towards a petition to allow access to Vertex’s treatment for Cystic Fibrosis (CF) meaning that the issue had to be debated by the House.

Campaigners are urging the government to allow wider access to the treatment, which is currently only available through Vertex’s compassionate use programme.

Vertex has become known by national bodies to play hardball over the price, German negotiators managed only a 15% discount on the list price. This meant that the price was reduced to about €133,000 per patient per year.

The UK has a reputation for being equally difficult to negotiate with over price, a situation that has led to a deadlock for both the company and the country – meaning that it is now the patients who are losing out.

About 3,000 people across the UK would be eligible for the treatment but it could cost in excess of £100,000 per patient for every year of treatment.

Vertex has argued, with new data, that the long term benefits to patients justify the high cost, showing greater cost-efficacy than the data initially presented to NICE back in 2016.

A Vertex spokesperson said, “We are astonished and dismayed by NHS England’s initial response to our portfolio approach. It amounts to a refusal to make any additional funding available for Orkambi or future medicines. CF is a devastating disease where half of people die by the time they are age 31. The situation with CF in the UK is unique and needs a unique solution – this is what our portfolio approach that we proposed in February offers.”

Professor Carole Longson, former Director of NICE, said at the time of the initial rejection: “We know how important a new treatment option would be for people with cystic fibrosis; but for the benefits it offers, the cost of Orkambi is too high. We can only recommend treatments when we are certain they are both clinically effective and represent good value for money. If the company is able to put forward a proposal that provides Orkambi at a cost-effective price, we would welcome it.”

Ben Hargreaves

Related Content

FDA approves IMIDEX’s AI-powered device VisiRad XR

The technological pharmaceutical company IMIDEX has been granted clearance from the US Food and Drug …

Artiva Biotherapeutics announces FDA clearance of IND for AlloNK and Rituximab combo

On 16 August 2023, the US Food and Drug Administration (FDA) officially cleared Artiva Biotherapeutics’ …

Zumutor’s cancer drug trial cleared by FDA

On 11 August 2023, the biopharmaceutical company Zumutor Biologics announced that the trial of its …

Latest content