Nearly 8,000 packs of Orkambi, the breakthrough medicine for cystic fibrosis, have been destroyed by the manufacturer while it has been in a stand-off with the NHS over the high price it wants to charge for the drug.
The US company Vertex has distributed 80,000 packs of Orkambi around Europe from its base in the UK since the drug was licensed in 2015. Other European countries with smaller numbers of people affected by the genetic disease have agreed to a high price, sometimes in order to give access to patients before negotiating downwards.
While Orkambi is not a cure, it treats the underlying causes of the disease in those with a specific form of the condition for the first time, stopping its progression.
The UK has more than 10,000 cystic fibrosis patients – more than anywhere except the US – and 40% could benefit from Orkambi. The NHS in England has said the potential bill is unaffordable.
Vertex said in a letter to the Commons health and social care committee that 7,880 packs had been destroyed because they had gone beyond their sell-by date during the negotiations, which ground to a halt last summer.
Each pack covers 28 days, which would mean the destroyed packs amounted to the equivalent of more than 600 years’ supply – or six years for 100 patients.
The shadow public health minister, Sharon Hodgson, said in a tweet: “Thousands of cystic fibrosis patients across the UK are still waiting for Orkambi to become available on the NHS. To know that 7,880 packs of the drug were destroyed due to going out of date is a disgrace.”
The MPs’ committee summoned NHS bosses and the chief executive of Vertex, Jeff Leiden, to explain why the stalemate continued in a day of hearings this month. The committee chair, Sarah Wollaston, asked the company how much stock had been destroyed in the last year. Leiden said he did not have the figure to hand but would provide it. Stuart Arbuckle, Vertex’s chief commercial officer, told the committee: “I do not believe we have had any stock go out of date and be destroyed … we do not package it up until we are due to be selling it.”
He added: “I would be surprised if it was very much.”
The committee has now published the subsequent letter from Vertex on its website. Simon Lem, the vice-president and general manager of the company’s north European region, which has its headquarters in London, wrote: “More than 80,000 packs of Orkambi have been distributed for commercial sale in the EU since licensing in 2015.” A further 11,000 have been supplied for free, usually on compassionate grounds, including to 600 people in England.
“In 2018, 7,880 packs (each containing a 28-day supply) manufactured in 2015 and 2016 for the purposes of supplying 13 countries (including the UK) exceeded their expiry date and, consequently, were destroyed,” he wrote.
Christina Walker, whose eight-year-old son Luis cannot have the drug, said: “This is abhorrent to me as the mother of a child in desperate need of Orkambi but denied it because apparently Vertex can’t possibly sell it at the price the NHS are offering without going bust.”
David Ramsden, the chief executive of the Cystic Fibrosis Trust, said: “It’s heart-breaking that packets of lifesaving drugs have been thrown away because they’re out of date – what a desperate waste. Thousands of people have suffered while these drugs have been sitting on the shelf. It is vital the talks that have started deliver a real result now. All parties involved must reach a deal immediately so that we can put an end to any further unnecessary death and deterioration.”
Since the committee hearing, Vertex executives have met the NHS, the National Institute for Health and Care Excellence (Nice), which advises the health service on cost-effectiveness, and the health secretary, Matt Hancock.
Vertex has not said whether it will re-engage with the processes of Nice, which found that the drug was not value for money at £100,000 per patient per year. Hancock told the House of Commons, however, that they had been having “constructive discussions” and that he was “delighted that Vertex have finally agreed to participate in those discussions”.
