[Ip-health] Guardian: Calls for action on patients denied £ 100,000 cystic fibrosis drug

Thiru Balasubramaniam thiru at keionline.org
Mon Feb 4 00:56:39 PST 2019

https://act. .org/planb/ <https://act.justtreatment.org/planb/>



Calls for action on patients denied £100,000 cystic fibrosis drug

Exclusive: Ministers urged to consider revoking patent on life-extending
Orkambi, which NHS cannot afford

The patients facing shorter lives due to drug’s expense

Sarah Boseley Health editor

Sun 3 Feb 2019 23.11 GMT

Campaigners 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
NHScannot afford.

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.

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 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has also withdrawn the newer and more effective drug Symkevi from the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.

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 chief executive, Dr Jeff
Leiden, was paid at least $17.2m in 2017, according to SEC filings reported
by Reuters last year.

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. 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

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.

Bill Wiggin, the Conservative MP for North Herefordshire, will ask the
government to invoke crown use for Orkambi in an adjournment debate in the
House of Commons on Monday. “Vertex refused the largest ever financial
offer the NHS has made in its entire history for this medicine,” said

He added: “Crown use licensing can prevent pharmaceutical companies from
taking advantage of incredibly vulnerable people.”

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 told the Guardian.

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: “It is
absolutely right that patients should have access to cost-effective,
innovative medicines on the NHS at a price we can afford.

‘Drugs are too expensive for the NHS – and people are paying with their

“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

Vertex, in its 16 January letter to the committee from the company’s
European president, Simon Lem, said: “As long as Nice remains constrained
by its current approach to the technology appraisal process, and,
ultimately, by the budgetary limits set by the Department of Health and
Social Care, it is unlikely that the parties will arrive at an outcome that
grants access to our treatments for CF [cystic fibrosis] patients in

The company declined to answer questions from the Guardian. 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 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.”

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. In the US,
Orkambi costs $272,000 (£207,000) 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.

Thiru Balasubramaniam
Geneva Representative
Knowledge Ecology International
41 22 791 6727
thiru at keionline.org

More information about the Ip-health mailing list