Help the NHS get a cure for cystic fibrosis, Ministers Asked | Science

Activists are calling on the government to urgently intervene on the cost of a drug that can prolong the lives of thousands of children, but that manufacturers have set at £ 105,000 per patient per year , a price the NHS can not afford.

For over two years, an emotional battle has been raging on Orkambi, who is attacking the cause of cystic fibrosis. However, children with the genetic disorder did not often survive until adulthood and today still, half of them will have died before the age of 32. years. Orkambi could extend life, but negotiations on the price that the NHS would pay are in stalemate since July, a pharmaceutical company that is refusing to reduce the price.

All countries of the world are fighting against the high prices of new drugs, which has provoked anger and campaign, especially against anti-cancer drugs. The NHS has lowered prices by requiring that drugs undergo a cost-effectiveness badessment and capping the price.

Vertex, based in Boston, USA, has evaluated the drug at £ 105,000 per patient per year, which the NHS England says is unaffordable. In July, Vertex refused its £ 500 million five-year offer for access to Orkambi and its other cystic fibrosis medications, claiming that it was inadequate and unfair. He has also removed the new most effective drug Symkevi from the approval process, which means he will no longer be available for any NHS patient.

Activists said the company had pulled $ 2.5 billion from the sale of the drug in 2017, uncovered thanks to funding from a charity for cystic fibrosis and the National Taxpayers' money. Institutes of Health of the United States. Vertex spent $ 3.3 billion on patents. Its director, Dr. Jeff Leiden, received at least $ 17.2 million in 2017, according to documents filed by the SEC with Reuters last year.

There is an urgency to campaign. Christina Walker, whose eight-year-old son, Luis, is troubled, said, "We think the government needs to step in, otherwise it could take months and months. It could be another year.

Are there precedents for the battle over the cost of Orkambi?

The fight for access to Orkambi – which manufacturers have set at over £ 100,000 per patient per year – has much in common with the fight against the cancer drug Herceptin in 2006. The underlying issues are the same New innovative drugs are being launched at exorbitant prices that pharmaceutical companies claim are needed to recover their costs, but health care providers and patients say they can not afford.

How can a medicine be so expensive?

The drugs with the highest prices are often the ones for which there are fewer patients, so less money to make. Cystic fibrosis is a rare disease that affects 10,400 people in the UK, where the genetic flaw is most prevalent, and around 100,000 worldwide. Medicines that cure a disease may be more expensive than those that need to be taken for many years.

How is the NHS trying to control the cost of medicines?

In 2017, the NHS drug bill rose to £ 16 billion and grew by 7% a year. The NHS has been actively trying to reduce costs by setting up an evaluation mechanism, managed by the National Institute of Health and Care (Nice), to ensure that only drugs of good value -prices are used. This has effectively capped and reduced the prices of drugs.

Has this effort been successful in the past?

The largest pharmaceutical companies are multinationals and all are turning to global markets, making it more difficult to obtain price reductions. But the NHS has been relatively successful in negotiating lower prices. The anti-cancer drug Kadcyla arrived in 2015 at a cost of £ 90,000 per patient per year. After much noise and negotiations, the manufacturer, Roche, accepted an undisclosed discount for the NHS.

A photograph: Fahroni / iStockphoto

"Cystic fibrosis does not wait for all that. My little boy has an infection that he could never eradicate and that is multi-resistant to antibiotics. The cumulative effect is what shortens people's lives. "

The activists want the government to cancel Vertex's patent on Orkambi to allow the manufacture of cheap versions through a legal provision called "use of the crown". Under the 1977 Patents Act, the state can effectively cancel a patent in the national interest. It was rarely used, but was relied on in the 1960s to obtain cheap generic versions of the tetracycline antibiotic for NHS hospitals in Italy, against the wishes of the owner of the Pfizer patent. More recently, he has been the subject of threats but has not been used in conflicts involving diagnostic tests for hepatitis C and kidney stones.

Under UK law, Vertex would be compensated with a much lower sum than it would normally earn with its monopoly of the three drugs against cystic fibrosis. Vertex hopes to be able to treat 90% of patients in the pipeline.

Bill Wiggin, Conservative MP for North Herefordshire, will ask the government to invoke Crown use for Orkambi during a debate adjournment in the House of Commons Monday. "Vertex has refused the biggest financial bid ever made by the NHS during its entire history for this drug," said Wiggin.

He added: "Crown licensing can prevent pharmaceutical companies from taking advantage of extremely vulnerable people."

The parents supporting the proposal said in a letter to Theresa May and Health Secretary Matt Hanbad that there is an urgent need for access to medication.

They wrote, "We can not explain, let alone expect you to understand, the fear and helplessness that this causes us to know that we will probably survive our children. The anxiety of knowing that a drug exists can change that – by sparing them unnecessary suffering and a decline in their health – but that access to them is denied, that is unbearable. "

Dr. Andrew Hill of the Pharmacology Department of the University of Liverpool advised the Just Treatment Drug Access Group, which launches the campaign with parents, about the likely cost of a generic version of the drug. # 39; Orkambi. "It could be done for up to £ 5,000 [per patient, per year], "He told the guard.

He added that pharmaceutical companies were not complying with the value-for-money judgments of the National Institute of Health and Care Excellence (Nice), which had informed NHS England that the cost was prohibitive. "It happens with so many anticancer drugs and now with cystic fibrosis," he said. "The government needs to have more teeth. You have to do something different. "

The House of Commons Health and Social Affairs Selection Committee has launched an Orkambi Availability Survey and will hold a public hearing in March. In a letter to the committee last month, John Stewart, national director of specialty orders at NHS England, said, "If companies like Vertex continue to offer products with a value well above the value that they have. they bring, the situation will not be sustainable. Vertex is extremely extreme in terms of price and behavior. "

He added: "Vertex's prices are far from the break-even point that all other companies are paying for."

Vertex, in his letter to the company's European Chairman, Simon Lem, addressed to the committee on January 16: "As long as Nice remains constrained by its current approach to the process of evaluating technology and, ultimately, by budget limitations set by the Department of Health and Social Services, it is unlikely that the parties will achieve a result giving access to our cystic fibrosis [cystic fibrosis] patients in England. "

The company refused to answer questions from the Guardian. Portland, its public relations consulting firm, said Vertex would participate in the public hearing.

"Out of respect for the parliamentary process, we will not provide any updates for the moment. Vertex is pleased with the interest of the Cystic Fibrosis Treatment Access Committee and we look forward to continuing to support its investigation of this important issue. "

The company responded to an open letter from activists who wrote on behalf of their children with the disease. The company said it was reinvesting nearly 70% of its revenue in its research, which allowed it to discover drugs like Orkambi. "

"Precision therapies such as ours produce unprecedented results, but they also present new economic challenges. We fully recognize the constraints on health systems around the world, creating unenviable choices for health authorities. "

Health authorities "must adapt their systems to the realities of modern medicine," the letter continues.

"Vertex shares with governments and regulators the responsibility to do everything in its power to agree on a fair price, reflecting the value of these medicines in all the countries where they are needed."

There are more than 10,000 people with cystic fibrosis in the UK and half of them could benefit from Orkambi because of the particular genetic abnormality of the drug. Vertex's newer and similar drugs are likely to help even more.

The price of drugs has caused consternation around the world. In the United States, Orkambi costs $ 272,000 per year per patient, which is not affordable for those who do not have good medical insurance. In Canada, where the drug costs the equivalent of £ 146,000, patients filed a clbad action in court against the government for access.