NHS does not value CF patients, says Vertex CEO in letter to PM

Theresa May has got plenty on her mind after being forced to reshuffle her cabinet following the latest Brexit row – but amid the chaos Vertex is trying to get her attention over the ongoing issue of funding for cystic fibrosis drugs.

Since NICE rejected regular NHS funding for Vertex’ Orkambi (ivacaftor+lumacaftor) two years ago, which corrects the underlying cause of the disease in a subgroup of patients, the US firm has been in negotiations with NHS England over a deal to get the drug funded.

But the talks ended in acrimony last week with Vertex describing NHS England as “outrageous” after refusing the “best offer in the world” covering Orkambi and all future CF drugs from Vertex.

Now Vertex’s CEO Jeff Leiden has written to Ms May asking her to personally intervene – and the tone of the letter led to rumours that the US firm is going to quit the UK.

Vertex has said on record that it has no plans to leave the country and remains committed to UK patients – but in the letter Leiden said the UK was putting a lower value on CF patients than any other country.

He said: “They are placing a lower value on the life of a CF patient than other countries around the world – for example, patients have access to Orkambi in Ireland, Sweden, Germany, Austria, Italy, Denmark and the Netherlands.”

The company noted it has an international HQ in London, has invested in an R&D site near Oxford and employs 250 people, of whom over 110 are scientists.

But Leiden said that the UK must invest in the next generation of precision medicines to develop science and innovation after Brexit.

He said: “This requires appraisal processes to keep pace with the science; current processes have significant limitations in how they capture and value the full medical, societal, economic and innovation benefits of such precision medicines. Without this, any future biotech investment in the UK is at significant risk.”

Meanwhile patient groups have made fresh calls for Vertex to reveal the price it has offered for Orkambi, as well as on drugs approved in the future.

Vertex has said that the price secrecy is because of a non-disclosure agreement that it signed with the NHS when negotiations started.

Christina Walker, whose eight year-old son Luis is eligible for Orkambi, said the patient group UKneedsorkambi is “very concerned” about the tone of Leiden’s letter.

She said in a statement: “Mr Leiden says they have made the ‘best offer in the world’ to the NHS’. We’d like to know what that is. With the high number of CF patients in UK, the total budget implication of Orkambi at the list price of £100k would be around £400mn. That’s equivalent to 2.5% of the entire NHS drugs budget (£16bn) for just 4,000 patients.”

“The NHS in England currently spends no more than £150m on standard CF care, for around 8,000 patients. Setting a price for Orkambi which – when aggregated – would dwarf the size of the Cancer Drugs Fund is both unrealistic and unreasonable, as the UKneedsorkambi campaign group represented directly to the company in a letter delivered during the recent protest.”

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