NHS England agrees deal for cystic fibrosis 'wonder drug' Orkambi

Joy for thousands of cystic fibrosis suffers as life-extending ‘wonder drug’ will be available on the NHS in England from next month after four years of haggling over its £100,000 a year price tag

• Up to 5,000 patients in England will benefit from the life extending medication
• It slow the decline of lung function and will be available to them within 30 days 
• Deal, thought to be in excess of £500m, gives NHS access to drugs for five years

Life-extending cystic fibrosis drugs will be available on the NHS next month after a deal was struck following four years of deadlock over their price tag.

Up to 5,000 patients in England will benefit from the £104,000-a-year pills Orkambi, Symkevi and Kalydeco after the deal with Boston-based maker Vertex.  

The deal is thought to be in excess of half a billion pounds and gives NHS England access to the medication for five years. 

All three drugs slow the decline of lung function – the main cause of death in cystic fibrosis. 

Health Secretary Matt Hancock hailed the news as ‘wonderful’ and said the drugs will ‘improve thousands of lives’. 

Up to 4,000 patients in England will benefit from the £104,000-a-year cystic fibrosis drug Orkambi after a deal was struck with the NHS

He said: ‘Some wonderful news this morning. We’ve agreed a deal to provide Orkambi and other lifesaving Cystic Fibrosis drugs on the NHS. 

‘The deal is great value for money for the NHS, and crucially, will improve thousands of lives. 

‘This deal – on the back of several others this summer – shows why we get some of the best value drugs in the world, and is another reason to be so proud of our NHS.’

Last month NHS bosses accused the US pharmaceutical firm of being greedy with its ‘monopoly pricing’.

Negotiations had been at a standstill since Vertex this year rejected an offer of £500million over five years for access to the drug.

The argument flared up again when the Scottish government announced it had managed to reach a deal last month – enabling the supply of the drugs to 350 cystic fibrosis patients north of the border.

Cystic fibrosis is a debilitating illness, which creates a mucus build-up that causes chronic lung infections and progressive lung damage.

The incurable disease affects around 70,000 people worldwide, including 3,000 in the UK, according to figures. 

Health Secretary Matt Hancock hailed the news as ‘wonderful’ and said the drugs will ‘improve thousands of lives’ 

NHS England chief executive Simon Stevens described the deal as a ‘long hoped-for moment’.

He added: ‘The UK has the second highest prevalence of cystic fibrosis of any country in the world, so today is an important and long hoped for moment for children and adults living with cystic fibrosis.

‘That fact also means that any drug company wanting to succeed commercially in this field needs to work constructively with the NHS.

‘I’m pleased that Vertex has now agreed a deal that is good for our patients and fair to British taxpayers.’ 

Orkambi, which was licensed in Europe in November 2015, is used to treat cystic fibrosis in patients aged two and older while Symkevi treats those aged 12 and up.  

The medications target the F508del gene mutation, which affects about 50 per cent of all people with cystic fibrosis. 

Labour leader Jeremy Corbyn said the deal was a victory for campaigners who ‘stand together and take on the powerful’.

He tweeted: ‘This is life changing for everyone with cystic fibrosis in our country, they’ve waited years for this drug. 

‘A victory for all the campaigners who have taken on big pharmaceutical companies’ greed and Government inaction. When we stand together and take on the powerful, we win.’

It comes after more than 117,000 people signed a petition calling on the Government to make Orkambi available on the NHS.

WHAT IS CYSTIC FIBROSIS? 

Cystic fibrosis is an incurable genetic disease that affects around 70,000 people worldwide.

A defective gene causes a build-up of mucus in the airways, making it increasingly difficult to breathe over time.

Mucus also blocks the natural release of digestive enzymes, meaning the body does not break down food as it should.

Signs and symptoms:

• A persistent cough that produces thick mucus (sputum)
• Wheezing
• Breathlessness
• Exercise intolerance
• Repeated lung infections
• Inflamed nasal passages or a stuffy nose

While healthy people cough naturally, that does not happen for people with CF.

Eventually, lung function depletes to the point that sufferers will need a double lung transplant to survive.

Source: Cystic Fibrosis Foundation

WHAT IS ORKAMBI?

Orkambi is a medication licensed for use by people in the UK with cystic fibrosis, but it is not offered on the NHS except in extreme circumstances.

The drug targets the F508del gene mutation, which affects about 50 per cent of all people with cystic fibrosis.

It is made of a combination of drugs – lumacaftor and ivacaftor – which work together to keep a healthy balance of salt and water in the lungs.  

There may be more than 4,000 people with life-limiting cystic fibrosis in the UK who could benefit from the drug.

But the National Institute for Health and Care Excellence (Nice) said in 2016  may not be cost-effective enough, at £104,000 per patient per year, to be offered on the NHS.

More than 117,000 people signed a petition calling on the Government to make Orkambi available on the NHS, and Parliament discussed the petition in March 2019.  

It was finally made available on the NHS in England on October 24 after four-years of negotiations. 

 

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