NHS England calls Vertex an ‘extreme outlier’ in conduct of Orkambi negotiations

During a House of Commons Health and Social Care Committee witness session on the availability of Vertex’s Orkambi and other cystic fibrosis (CF) drugs on the National Health Service (NHS), representatives from NHS England and the UK’s pricing regulator National Institute for Health and Care Excellence (NICE) called Vertex an ‘extreme outlier’ in terms of its engagement with the UK’s healthcare authorities and their appraisal processes.

Negotiations between Vertex and NHS representations over Orkambi reached an impasse in July last year and NICE’s review of another CF drug, Symkevi, was suspended one month later.

NHS England national medical director Professor Stephen Powis said: "Unlike other companies who are willing to submit evidence and then work on the process…Vertex has come at this with a particular price in mind, and they come to try and get those processes modified to meet that price expectation, rather than submit to the process and work for the analysis of the evidence base."

Chief executive of NICE Sir Andrew Dillon stated: "It is really unusual to be a position where we simply seem to go round in circles to examine ever more details of NICE’s methodology. As if the solution to this problem is redesigning NICE simply to enable the NHS to pay Vertex more money."

"In order to unlock the potential of their existing and future treatments for patients with CF in England, it is absolutely essential that the company works with us," Dillon continued. "If they do, they will find there are flexibilities in our processes, which will help us all understand the additional value of their treatments and reach a fair price."

NHS England claimed that the quickest way to get Orkambi to patients was for Vertex to accept the offer on the table, rather than put pressure on the government to overturn Vertex’s patentas called for by the campaign Just Treatment.

The published offer is a payment of £500m over five years for Vertex’s entire portfolio of CF drugs, including those developed in the future. John Stewart, NHS England national director of specialised commissioning, noted the flexibility in the organisation’s offer, and how "this is not something that we tend to do."

The offer "would cover all existing licensed indications, Kalydeco, Orkambi and Symkevi, and when the future products become licensed, we would immediately reimburse in advance of NICE concluding an appraisal", Stewart said.

At that point, Vertex would be able to submit alternative prices to NICE for the new medications alongside "evidence and data that will support and justify a higher price".

However, Stewart clarified for the committee that there has been "no movement since June on price" from Vertex, and the price offered by Vertex, which is protected by a non-disclosure agreement, is miles away from the NHS’s offer.

NHS England did reveal that it believes it overpaid for Kalydeco in 2012, and this "probably set unhelpful, unrealistic expectations", and it is keen to "make sure that the entire Vertex portfolio is priced at a fair and sustainable level going forward".

Vertex explains its position on CF drug pricing

Vertex executives, including CEO Dr Jeffrey Leiden, responded to these allegations by explaining why they could not accept NHS England’s offer.

"We have been painted as not being willing to take the offer, I would say differently, we cannot take that offer.

"The English offer is around £10,000 per patient per year…If we accept that offer, that has been made public by the NHS, of course every other country will want that same offer.

"If we were to accede to that [in all the countries where Orkambi is available], our total CF revenues wouldbe £850m a year. Unfortunately, that would not allow us to develop the next set of CF medicines, because are spending $1bn just on R&D, so we would go out of business in three to five years."

He noted this would mean the company’s development of novel, efficacious triple combination therapies for CF would have to stop.

Leiden also declared that NICE’s policies have failed to adapt to progression in life sciences. In 17 countries he claims to have had successful reimbursement agreements, stating that Vertex "were able to modify their [the national regulator’s] assessment to capture the true benefits of these medicines to patients".

He accused the government of walking away from the negotiating table by publicly saying an offer he claims is "a 90% discount" on the price Vertex offers elsewhere is the last and best offer.

Leiden went on to express hope that his meeting with the Secretary of State for Health and Social Care Matt Hancock next week would get NHS and NICE back to the negotiating table. He said one of the ideas he would be suggesting a similar situation to Scotland where patients have access to the drug at a discounted list price while negotiations are ongoing.

The witness session was part of the Committee’s general inquiry into the pricing of Orkambi.

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