OnMedica – News – A group of medications urged to reduce £ 100,000 the price of a drug for cystic fibrosis

Activists say the drug could extend the lives of thousands of children

Activists are calling on the government to put pressure on manufacturers to reduce the price of cystic fibrosis treatment by £ 105,000 per patient per year, likely to prolong the cost of cystic fibrosis. lives of thousands of children.

Negotiations on the price that the NHS would have to pay for Orkambi have been stalled since July, as Vertex manufacturers declined to lower 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.

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. "

A spokesperson for the Ministry of Health and Social Affairs said: "It is absolutely right that patients have access to affordable and innovative medicines on the NHS.

"Although it is being offered in the region at around £ 500 million over five years – the largest of its kind in 70 years, the Vertex has refused to accept it, putting Orkambi out of reach of patients .

"We are aware that other ways could be considered to solve this problem, but our approach encourages Vertex to accept the generous offer of NHS England."

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 Jan. 16: "As long as the NICE will remain constrained by its current approach to the process of evaluating technology and ultimately by the limits budgets set by the Department of Health Care and Social Services, it is unlikely that the parties will achieve a result giving access to our cystic fibrosis treatment [cystic fibrosis] patients in England. "

The Vertex public relations consulting firm, Portland, announced that 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."