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Cystic Fibrosis Trust

Stopping the Clock

We are determined to ensure people with cystic fibrosis have access to life-changing treatments.

The Trust has been campaigning hard to ensure that people with cystic fibrosis can access innovative precision medicines. Most recently, the focus of our Stopping the Clock campaign has been precision medicine, Orkambi.

What is a precision medicine?

  • Whilst conventional CF treatments target the symptoms, precision medicines tackle the underlying genetic mutations which cause the condition. 
  • Precision medicines are so called because they only treat certain mutations. These drugs are part of a wider move towards personalised healthcare. 

Orkambi

Orkambi is the next precision medicine for cystic fibrosis. Orkambi targets a mutation that around 40% of people with cystic fibrosis in the UK have. Orkambi has been shown to boost lung health, significantly reduce infection and avert hospitalisations.

In June 2016, despite recognising Orkambi as an important treatment, the National Institute of Clinical Excellence (NICE) was unable to recommend Orkambi for use within the NHS. The Scottish Medicines Consortium (SMC) was similarly unable to recommend Orkambi for use within NHS Scotland in May 2016, but did recognise the drug as a beneficial treatment.

Find out more about Orkambi and our campaign.

Kalydeco

The first precision medicine for cystic fibrosis, Kalydeco, targets a mutation that little more than 4% of people with CF in the UK have.

Following successful campaigning by the Trust, the majority of people eligible for Kalydeco are new receiving it. These people have shown significantly increased lung function, slower progression of lung disease and just under half as many hospital admissions. 

Find out more about Kalydeco and our campaign.

​Triple combination therapy

In July, Vertex announced some encouraging results for their next generation of precision medicine, the triple combination therapy. This drug uses a combination of Kalydeco, Vertex's new product tezacaftor and one of three new compounds, to tackle the underlying cause of cystic fibrosis. This new drug could work in people with one copy of the F508del mutation and one copy of what is known as a 'minimal function mutation'. This triple combination could benefit around 90% of the CF population, with a severe and difficult-to-treat form of cystic fibrosis.

Vertex are hoping to start Phase III trials for the treatment in the first half of 2018.

Find out more about the triple combination therapy.

Campaign timeline

Take a look at our news stories below for more information on each stage of our fight for fair access to medicines.

2017

  • 12 October - Wales extends access to Kalydeco for people with an R117H mutation. 
  • 5 September -  Vertex issues an update on proposals to increase access to Orkambi.
  • 19 July - Vertex announces promising results from trials into their new triple combination therapy.
  • 26 June - we held a day of protests in England, Northern Ireland, Scotland and Wales, to call on Vertex and governments across the UK to agree on an affordable price that can make Orkambi immediately available to those who so desperately need it. Read about our protests
  • 21 March - we met with the new Health Minister, who reassured us that the Government is still open to a deal, and is looking forward to hearing new proposals from Vertex.

2016

  • 13 December - we hold a parliamentary debate encouraging the Government to help implement the recommendations in the Accelerated Access Review.
  • 5 December - NHS England announces that Kalydeco will be made available to two-to-five-year-olds in England, benefitting around 50 children.
  • 24 November - we've launched an e-action ahead of our parliamentary debate on Orkambi. Use it to contact your MP and get them to share your story to ensure Orkambi is made available for everyone who could benefit from it.
  • 27 October - Scotland becomes the first of the devolved nations to grant two-to-five-year-olds access to Kalydeco.
  • October - Orkambi negotiations between Vertex, the Government and the NHS get underway.
  • 17 June - NICE gives final rejection of Orkambi for use on the NHS in England - we suggest a solution using the UK CF Registry to give people access while allowing data on its efficacy to be gathered.
  • 19 May - Orkambi and Kalydeco are rejected in Scotland, hundreds of people use our e-action to contact their MPs to urge the Scottish Government to support our solution and have Orkambi and Kalydeco provided to those who need it by the New Medicines Fund.
  • 1 April 6,000 people use our e-action to contact their MPs in support of our proposed solution to the issues around providing Orkambi leading the Government to state it is ready to discuss proposals to allow people access to Orkambi at a cost-effective price. This follows NICE's initial rejection of Orkambi for use in the UK despite evidence of its efficacy, stating cost and a lack of long-term evidence about its benefits.
  • 2 March - Carly Jeavons, a young mother with CF, presented our petition at Downing Street.
  • 28 January - we launched a petition that 20,000 people went on to sign, calling for the NHS and drug companies to take responsibility for ensuring precision medicines are available to those who need them as soon as possible.

2015

  • December - the CF Community took to Twitter to tell Ian Austin MP how much precision medicines mean to them. He then used these powerful stories in a parliamentary debate that saw the Government go on record to support our campaign.
  • November - almost 1,500 passionate campaigners took part in our survey about what precision medicines mean to them, shaping our submission to NICE as it prepared to consider Orkambi.




Clara's story

Read about fourteen-year-old Clara, a keen runner and aspiring author, who was also the youngest person in the UK to go on the Orkambi trial.

Read on

What is cystic fibrosis?

Find out more about cystic fibrosis, its diagnosis and how it is treated, as well as useful links to our publications and other organisations who can help.

Read on

More information

If you would like to find out more about any of our campaigns, publications or anything else, get in touch and we'll be happy to help.

Get in touch