NHS and Vertex remain deadlocked over price of cystic fibrosis drug

Both sides of the stalled negotiations on the provision of the cystic fibrosis (CF) drug Orkambi (lumacaftor/ivacaftor) accused the other of inflexibility while giving evidence to the Health and Social Care Committee’s inquiry this week.

Vertex Pharmaceuticals has been in dispute with NHS England over the high price the company wants for Orkambi—£105 000 (€123 000; $138 000) per patient per year. There has been mounting frustration from patients over the impasse. The committee received 334 written submissions to the inquiry, mostly from patients.1 After the hearing there was a demonstration by relatives of patients with CF and campaigners in Parliament Square.

Jeff Leiden, chief executive officer of Vertex Pharmaceuticals, told the committee he is meeting the health secretary Matt Hancock on Monday 11 March and would put forward some “new ideas” for getting through the stalemate. He suggested they could discuss a deal similar to that agreed in Scotland, which would see Orkambi supplied to NHS patients at a discount to the list price, pending further discussions about how to measure the drug’s cost effectiveness.

In July 2018, after 12 months of negotiations, NHS England published what they said was a “fair and final offer” of around £500m over five years for all of Vertex’s approved drugs and any that are approved in the future. Vertex rejected the offer and has also withdrawn Symkevi (tezacaftor/ivacaftor), a therapy that was in development, from the approval process.

Leiden told the committee: “It’s not that we won’t take that offer, it’s that we can’t.” He said every other country will want that same offer which amounts to around £10 000 per patient per year and that wouldn’t allow it to develop the next generation of CF drugs.

Leiden said Vertex had done successful deals in 17 countries over Orkambi and that NHS England is being offered the “best price in the world.” In his evidence Leiden criticised the National Institute for Health and Care Excellence’s (NICE) cost effectiveness assessments saying the system was 25 years old and not fit for purpose.

John Stewart, national director of specialised commissioning at NHS England, called Vertex an “extreme outlier” in terms of their behaviour when compared with other pharmaceutical companies. He said all Vertex were interested in was trying to change the NICE appraisal process and had made absolutely no movement on price.

Stewart said they had “overpaid” for Vertex’s other drug Kalydeco (ivacaftor) by £40m a year or £200m over the five years of the contract that has just ended. But he denied that NHS England is trying to “reclaim” that overpayment in their current negotiations over Orkambi.

Leiden faced pointed questions about his salary of $17m a year and the fact that the pharmaceutical company had a turnover of £5.3bn in the five years to the end of 2017 but paid virtually no UK corporation tax. He replied that the company would not pay corporation tax in the UK or the US until it had paid off its operating costs.

Andrew Dillon, chief executive of NICE, acknowledged that Orkambi works but said it was not cost effective. He said NICE had to be consistent with the methodology it uses for all drugs and that Orkambi at around £300 000 per quality adjusted life year was 10 times higher than its threshold. He called on Vertex to demonstrate flexibility.

Caroline Elston, director of the adult CF service at King’s College Hospital NHS Foundation Trust described the delays as “extremely frustrating.” She told the committee that although Orkambi’s effects on lung function are relatively modest it has a large impact on exacerbations and reduces hospitalisations. She said it stabilised patients so kept them well enough to hopefully be able to take advantage of the promising next generation triple therapy drugs in the pipeline.

In her closing comments, the committee’s chair Sarah Wollaston, said: “I hope that in all negotiations all parties will put patients front and centre.”