Parents of children with cystic fibrosis who are desperate to use a drug that the NHS cannot afford are forming a buyers club to obtain a cut-price version made in Argentina where the patent does not apply.
Angry at the stalemate between Vertex, the US manufacturer of Orkambi, and NHS England, the families are forming the club to help each other through the legal and technical hurdles of importing the cheaper generic drugs for each child’s individual needs.
The concept was central to the Hollywood film Dallas Buyers Club, which was based on the real situation of people who could not obtain drugs for their HIV infections in the 1980s and 90s.
Orkambi’s list price is £104,000 per patient per year, which the NHS has said is unaffordable. Negotiations broke down last July. After an outcry this year triggered a health select committee hearing and a meeting between the health secretary, Matt Hancock, and Vertex’s chief executive, Jeff Leiden, the company returned to the negotiating table, but there is no sign of a deal.
Orkambi is the first drug to treat the underlying causes of cystic fibrosis, a progressive disease in which mucus clogs up the lungs. Infections cause lasting damage and early death. There are 10,400 patients with cystic fibrosis in the UK, 40% of whom could benefit from Orkambi, the brand name of a drug made from the chemical compounds ivacaftor and lumacaftor. The drug is more effective the earlier it is given in the course of the disease.
The Argentinian generic, made by Gador and called Lucaftor, costs 70%-80% less than Vertex’s list price, which four families in the UK have paid for privately. However, the generic drug costs more than £20,000 for a year’s supply, which will still be unaffordable for many.
The families are calling on the government to override Vertex’s patent and buy the generic drug for all those who need it. About 120 families in Argentina use Lucaftor, which has been certified as equivalent to Orkambi.
One of the parents, Robert Finlay (not his real name), went to a meeting with a representative of Gador in London. “To hold a box of it in my hand after trying to fight for this was really quite emotional,” he said. His daughter, Florence, seven, is well at the moment, but he knows of another child her age who is not. “Since last autumn she has lost 40% of her lung function. In the last six months she has been in hospital on intravenous antibiotics.”
Florence has recently picked up a bug. “It is another course of antibiotics for the next three weeks as a precaution,” Finlay said. “The sooner she takes Orkambi, the less damage will be done.”
Rob Long has been buying Orkambi from Vertex for his son Aidan, nine, since last June. “He was in good shape but obviously it is about keeping him in good shape – halting the decline, basically,” he said.
Aidan did well on the drug, growing taller and gaining weight. But after the trampolining champion fell and broke both arms last September, Long said he was glad Aidan was on the drug because he was unable to do the physiotherapy that all cystic fibrosis patients have to undergo to keep their lungs free of sticky mucus.
It was Long who found Gador. He said he was paying for Orkambi “because my son’s health is paramount”, and that everybody who needed it should have access to it. He started to search online and via social media for a cheaper generic version as soon as Orkambi was approved in Europe for children of his son’s age, in February 2018. “I couldn’t rest until I had explored every angle,” he said.
Eventually a Colombian told him about a version made in Argentina. He said he was sceptical and cautious at first, but in April he was able to share the news with other parents. “They were very excited,” he said.
Parents have been campaigning for access to Orkambi since it was licensed nearly four years ago. Last summer Vertex rejected an NHS England offer of £500m over five years and potentially £1bn over 10 years for access to Orkambi and other cystic fibrosis drugs in the pipeline.
NHS England recently revealed it had made a further offer, to include an increased price for Orkambi and another drug, Kalydeco, which the NHS already provides but which works for only a small number of people. It proposed a two-year managed access scheme, which would allow the NHS to collect data on how well the drugs work. The prices would then be revised up or down. Vertex made another offer this week, well above what the NHS is willing to pay.
Long, Finlay and others argue that the UK government could make the generic drug available on the NHS by using the crown use provisions of UK patent law which allows a patent to be disregarded in the national interest. Another option would be to launch a large-scale trial for cystic fibrosis patients in which it would be legal to use the generic drug, as the NHS has done with drugs to protect men at risk of HIV infection.
Long is worried Vertex will call on Donald Trump to intervene. Vertex has a history of political lobbying and the US government has always defended US pharmaceutical company patents. “The UK is looking for a trade deal and we are off to buy generics,” said Long. “A lot of politicians will back off very quickly.”
Meanwhile, families who say time is not on their side will attempt to find the money to buy Lucaftor themselves, which can legally be shipped from Argentina directly to individual patients in three-monthly packages. Some are likely to try to raise funds for the treatment online.
Vertex said it was committed to finding a way to provide access to Orkambi and its other drugs for all eligible cystic fibrosis patients in England. “We have been open to discussing multiple options and flexibilities which would allow cystic fibrosis patients to access currently approved medicines,” a spokesperson said.
They said the list price of £104,000 should not be interpreted as the offer to NHS England. “Companies who claim to be able to produce a product similar to Orkambi have not had to bear the cost of drug discovery and development; though it should be noted that even at a 70%-80% cost reduction from the list price, this is still higher than the amount NHS England offered to pay for Orkambi in 2018.”
A Department of Health and Social Care spokesperson said: “It is absolutely right that patients should have access to cost-effective, innovative medicines on the NHS at a price we can afford. Our approach remains to urge Vertex to accept NHS England’s generous offer.”
