Campaigns urge the government to urgently intervene in the cost of the drug, which may prolong the lives of thousands of children, but manufacturers have allocated £ 105,000 per patient per year, a price the NHS can not afford.
For more than two years, Orkambi raises an emotional battle that deals with the cause of cystic fibrosis. Earlier, children with genetic disease do not often experience adulthood, and even today, half of them will be dead before age 32. a pharmaceutical company that refuses to reduce the price.
Countries around the world are struggling with the high prices of new drugs, which has led to anger and campaigns, especially with regard to cancer medicines. The National Health Service has forced price cuts by asking for medicines to go through a cost-effectiveness assessment and limit the price they will pay.
Vertex, based in Boston, USA, estimated the drug at £ 105,000 per patient per year, which NHS England says is unavailable. Vertex declined his offer in July from five million pounds in five years for access to Orkambi and other CFCs, saying it was inadequate and unfair. He also withdrew the newer and more effective drug Symkevi from the approval process, which means he will not be available to all NHS patients.
Campaigners said the company has earned $ 2.5 billion in drug sales in 2017, which was found thanks to funding from a charity for cystic fibrosis and taxpayer money from the US National Institute of Health. Vertex paid $ 3.3 billion for patents. Chief Executive Officer Dr. Jeff Leiden has received at least $ 17.2 million in 2017, according to SEC, reported by Reuters last year.
There is an urgent need to run the campaign. Christina Walker, whose eight-year-old son, Louise, has this disorder, says, "We think the government should go in, otherwise it may be months and months. It may be another year.
"Cystic fibrosis does not wait for all this. My little boy has an infection he can never eradicate, which is multi-resistant to antibiotics. The cumulative effect is what reduces people's lives. "
Activists want the government to revoke Vertex's patent on Orkambi to allow cheap versions to be made through a legal provision called the use of the crown. Under the 1977 Patent Act, the State can effectively cancel a patent in the national interest. It was rarely used, but it was used in the 1960s to obtain cheap generic versions of the antibiotic tetracycline for NHS hospitals in Italy against the desire of the Pfizer patent owner. Recently, it has been threatened, but it is not used in disputes about diagnostic tests for hepatitis C and machines for treating kidney stones.
Vertex will be offset under British law with a much smaller amount than it could have gained from its monopoly on the three cystic fibrosis drugs. Another combination is also that Vertex hopes to cure 90% of patients.
Bill Wiggin, the conservative MP for North Erfordshire, will ask the government to refer to the use of a crown for Orkambi in a debate in the House of Commons on Monday. "Vertex has declined the biggest financial offer the NHS has made throughout its history for this drug," said Wiggin.
He added: "The licensing of Crown can prevent pharmaceutical companies from benefiting from extremely vulnerable people."
Parents who support the proposal say in a letter to Teresa May and health secretary Matt Hancock that access to drugs is urgent.
They write: "We can not explain, not to mention that you expect to know, pure fear and helplessness makes us know that we are likely to outlive our children. The difficult consciousness that a drug exists that can change that – saving them unnecessarily suffering and declining health – but giving them access is unbearable. "
Dr. Andrew Hill of the Department of Pharmacology at Liverpool University advised access to the Just Treatment group, which launched the campaign with parents about the likely cost of a generic version of Orkambi. "It can be done for a maximum of 5,000 pounds [per patient, per year]he said to the Guardian.
He said the pharmaceutical companies did not respect the judgments of the National Institute of Health and Care (NICE), which recommended NHS England to spend too high. "It's happening with so many cancer drugs and now with cystic fibrosis," he said. "The government must have a few more teeth. It has to do something different. "
The Health and Welfare Commission of the House of Commons launched a study on the presence of Orkambi and will hold a public hearing in March. In a letter to the commission last month, John Stewart, NHS National Director for England, said: "If companies such as Vertex continue to value products significantly above the value they bring, then the situation is unstable. Vertex is extremely extreme in terms of both pricing and behavior. "
He added: "The prices required by Vertex do not come close to meeting the cost-effectiveness thresholds that all other companies operate."
Vertex, in a letter dated January 16th to the Committee of European Company President Simon Lem, said: "While Nice remains limited by its current approach to the technology assessment process and ultimately by the budget constraints set by the Health and Social Affairs care, it is unlikely that countries will reach a result that gives access to our treatments for HIV [cystic fibrosis] patients in England. "
The company declined to answer questions from the Guardian. Portland, the public relations consultancy, said Vertex would be involved in the public hearing.
It says: "From respect for the parliamentary process, we will not provide new updates at this time. Vertex welcomes the Committee's interest in access to cystic fibrosis and we look forward to continuing to support his study on this important issue. "
The company responded to an open letter from campaign participants who wrote on behalf of their children suffering from the disease. The company reported that it reinvested nearly 70% of its revenue in its research, allowing it to find medicines such as Orkambi.
It says, "Precise therapies, like ours, give unprecedented results, but they also represent new economic challenges. We are fully aware of the limitations on which the health systems around the world are working, which create unpopular choices for health authorities. "
Health authorities "need to adapt their systems to the realities of modern medicine," he wrote in the letter.
"Vertex shares responsibility with governments and regulators to do their best to agree on a fair price that reflects the value of these medicines in all countries where they are needed."
There are more than 10,000 cystic fibrosis in the UK, and half can benefit from Orkambi because they have the specific genetic defect the drug is targeting. Probably the newer Vertex-like drugs will help even more.
The cost of the drug has caused horror around the world. In the US, Orkambi costs $ 272,000 (£ 207,000) per patient per year, which is not available to those who do not have good medical insurance. In Canada, where the drug costs equivalent to 146,000 pounds, patients have begun a group action in the courts against the government to access it.