Emotive campaign over £105,000 per year cystic fibrosis drug

Organisation: Position: Deadline Date: Location:

Campaigners in the UK are calling on the government to urgently intervene over the cost of a drug that could extend the lives of thousands of children but which manufacturers have set at £105,000 per patient per year, a price the National Health Service (NHS) cannot afford.

The Guardian reports that for more than two years an emotive battle has been raging over Orkambi, which tackles the cause of cystic fibrosis. Previously, children with the genetic disorder did not often survive to adulthood and even today, half will be dead before the age of 32. Orkambi could extend lives, but negotiations over the price the NHS should pay have been in stalemate since July, with the pharma company that makes it refusing to reduce the price.

The report says countries all over the world are struggling with the high prices of new medicines, which has led to anger and campaigning, particularly over cancer drugs. The NHS has forced down prices by requiring drugs to go through a cost-effectiveness appraisal and capping the price it will pay.

Vertex, based in Boston in the US, has priced the drug at £105,000 per patient per year, which NHS England says is unaffordable. Vertex turned down its (the NHS) offer in July of £500m over five years for access to Orkambi and its other cystic fibrosis drugs, saying it was inadequate and unfair. It has also withdrawn the newer and more effective drug Symkevi from the approval process, which means it will not be available to any NHS patients.

According to the report campaigners have said the company had earned $2.5bn from sales of the drug in 2017, which was discovered thanks to funding from a cystic fibrosis charity and taxpayers’ money from the National Institutes of Health in the US. Vertex paid $3.3bn for the patents. Its CEO, Dr Jeff Leiden, was paid at least $17.2m in 2017, according to SEC filings reported last year.

The report says there is an urgency to the campaigning. Christina Walker, whose eight-year-old son Luis has the disorder, said: “We think the government has got to step in otherwise it could be months and months. It could be another year. Cystic fibrosis doesn’t wait for all this. My little boy has got an infection he may never eradicate that is multi-resistant to antibiotics. The cumulative effect is what shortens people’s lives.”

The campaigners want the government to set aside Vertex’s patent on Orkambi to allow cheap versions to be made through a legal provision called crown use. The report says under the 1977 Patents Act, the state can effectively override a patent in the national interest. It has rarely been used, but was invoked in the 1960s to obtain cheap generic versions of the antibiotic tetracycline for NHS hospitals from Italy, against the wishes of the patent holder Pfizer. It has more recently been threatened but not used in disputes over hepatitis C diagnostic tests and machines for treating kidney stones.

The report says Vertex would be compensated under the UK law with a much smaller sum than it could otherwise earn from its monopoly of the three cystic fibrosis drugs. Another combination is also in the pipeline that Vertex hopes could treat 90% of patients.

Parents backing the proposal said in a letter to Theresa May and health secretary Matt Hancock that access to the drugs was urgent. They wrote: “We cannot explain, let alone expect you to understand, the sheer dread and helplessness it causes us to know that we are likely to outlive our children. The anguish in knowing that a drug exists that can change that – sparing them unnecessary suffering and decline in health – but that they are denied access, is unbearable.”

Dr Andrew Hill of the pharmacology department of the University of Liverpool advised the access to medicines group Just Treatment, which is launching the campaign with the parents, on the likely cost of a generic version of Orkambi. “It could be made for a maximum of £5,000 (per patient, per year),” he is quoted in the report as saying.

He said drug companies were not respecting the value for money judgments of the National Institute for Health and Care Excellence (Nice), which advised NHS England that the cost was prohibitive. “It is happening with so many cancer drugs and now cystic fibrosis,” he said. “The government has to have some more teeth. It needs to do something different.”

A spokesperson for the Department of Health and Social Care said in the report: “It is absolutely right that patients should have access to cost-effective, innovative medicines on the NHS at a price we can afford. Despite being offered in the region of £500m over five years – the largest ever commitment of its kind in the 70-year history of the NHS – Vertex has refused to accept, putting Orkambi out of reach of patients. We’re aware there may be other avenues open to resolve this issue, but our approach remains urging Vertex to accept NHS England’s generous offer.”

The House of Commons health and social care select committee has launched an inquiry into the availability of Orkambi and will hold a public hearing in March. In a letter to the committee last month, NHS England’s national director of specialised commissioning, John Stewart, said: “If companies like Vertex continue to price products significantly above the value they bring, then the situation is unsustainable. Vertex is an extreme outlier in terms of both its pricing and behaviour.” He added: “The prices being demanded by Vertex don’t come close to meeting the cost-effectiveness thresholds that all other companies are working towards.”

The report says Vertex declined to answer questions put to them. Portland, its public relations consultancy, said Vertex would be participating in the public hearing. It said: “Out of respect for the parliamentary process, we will not be providing further updates at this time. Vertex welcomes the committee’s interest in access to cystic fibrosis medicines and we look forward to continuing to support its inquiry into this important issue.”

The report says the company has responded to an open letter by campaigners who wrote on behalf of their children suffering with the disease. The company said it reinvests nearly 70% of its revenue back into its research, which has allowed it to discover medicines like Orkambi.” It said: “Precision therapies like ours are delivering unprecedented results, but they also present new economic challenges. We recognise fully the constraints that health systems around the world are working under, which create unenviable choices for the health authorities.”

Health authorities “need to adapt their systems to the realities of modern medicine,” the letter went on. “Vertex shares the responsibility with governments and regulators to do all that we can to agree to a fair price that reflects the value of these medicines in all countries where they are needed.”

The report says there are more than 10,000 people with cystic fibrosis in the UK and half could benefit from Orkambi because they have the particular genetic defect that the drug targets. Vertex’s newer, similar drugs are likely to help even more.

The price of the drug has caused consternation around the world. The report says in the US, Orkambi costs $272,000 per patient, per year, which is not affordable for those without good medical insurance. In Canada, where the drug costs the equivalent of £146,000, patients have launched a class action in the courts against the government for access to it.


In the US, Senator Bernie Sanders has sent a letter to Catalyst Pharmaceuticals asking it to justify its decision to charge $375,000 annually for a medication that for years has been available to patients for free. Reuters Health reports that, according to the letter, the drug, Firdapse, is used to treat Lambert-Eaton Myasthenic Syndrome (LEMS), a rare neuro-muscular disorder. The disorder affects about one in 100,000 people in the US.

The report says the government is intensifying its scrutiny of the pharmaceutical industry and rising prescription drug prices, a top voter concern and a priority of President Donald Trump’s administration. Both the Democratic-led US House of Representatives and the Senate, controlled by Republicans, have begun holding hearings this year on the rising costs of medicines.

The report says in the letter, Sanders asked Catalyst to lay out the financial and non-financial factors that led the company to set the list price at $375,000, and say how many patients would suffer or die as a result of the price and how much it was paying to purchase or produce the drug.

The report says Catalyst declined to comment on Sanders’ letter.

The Guardian report
Reuters Health report

Receive Medical Brief's free weekly e-newsletter

Related Posts

Thank you for subscribing to MedicalBrief

MedicalBrief is Africa’s premier medical news and research weekly newsletter. MedicalBrief is published every Thursday and delivered free of charge by email to over 33 000 health professionals.

Please consider completing the form below. The information you supply is optional and will only be used to compile a demographic profile of our subscribers. Your personal details will never be shared with a third party.

Thank you for taking the time to complete the form.