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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.

Precision medicines are a promising new type of treatment that tackle the underlying cause of cystic fibrosis (CF), the genetic defect, rather than just the symptoms. 

Kalydeco

The first precision medicine for cystic fibrosis, Kalydeco targets a mutation that only a little more than 4% of people in the UK have. Following successful campaigning by the Trust, the majority of people eligible for Kalydeco are receiving it. These people have shown significantly increased lung function, slower progression of lung disease and less than half as many hospital admissions as without it. Find out more about Kalydeco.

Orkambi

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

Our campaign

The Trust has been campaigning hard to ensure people gain access to Orkambi since it was authorised for use in the UK in November 2015. The response from the cystic fibrosis community has been fantastic: over 20,000 people have been involved in our survey, digital debate in Parliament, petition and e-action. This has helped gain widespread political support for the Trust’s proposed solution, which would see access to Orkambi while vital long-term data is collected from the UK CF Registry.

Despite this, and due to an outdated appraisal system, in June the National Institute for Health and Care Excellence (NICE) was unable to recommend Orkambi for use by the NHS in England, even though it recognised the treatment as effective for managing cystic fibrosis. 

In response, the Trust called for meaningful discussions between Orkambi manufacturer Vertex Pharmaceuticals and the Government by the end of July. The Government has confirmed that these discussions are underway and reassured us that everything possible is being done to find a way forward. The Trust has proposed a solution whereby Orkambi could be provided to all who need it while further evidence is collected on its long-term clinical impact using the UK Cystic Fibrosis Data Registry.

The latest update 

After December's parliamentary debate (see below) and pressure from the community, the Government were persuaded to meet with the us to discuss a way forward.

After a number of delays, on Tuesday 7 March 2017 we met with the new Health Minister, Lord O'Shaughnessy, who paid tribute to the community's incredible campaigning and the work that we have put in behind the scenes. We were reassured that the Government is still open to making a deal happen and is looking forward to hearing new proposals from Vertex. We are also pleased to hear that the Government is considering how the Trust's solution (see above) could break the deadlock.

We will not allow for either side to let this opportunity slip, and we will be monitoring negotiations and calling on the community to work with us to help put pressure on Vertex and governments across the UK.

The parliamentary debate

The discussions between Vertex Pharmaceuticals, the Government and the NHS had reached a deadlock. Vertex wrote to us to explain its position and we wrote to the Government to raise our concerns. An influential report into speeding up access to drugs like Orkambi - called the Accelerated Access Review - made several recommendations to the Government that could be key to breaking this deadlock, such as flexible pricing arrangements and the gathering of real-world data to prove how effective the drug is.

On 13 December 2016 we held a parliamentary debate called 'Implications of the Accelerated Access Review for cystic fibrosis and other conditions'. We teamed up with MPs Ian Austin, Kerry McCarthy and Ben Howlett for this debate to put pressure on the Government to help apply the flexibility recommended in the Accelerated Access Review to the Orkambi negotiations. You can watch the debate in full on ParliamentLive, or read our article on the importance of these debates. Below, watch Darren O'Keefe, Public Affairs Manager at the Trust, explain his top five take-home points.

This campaign isn't about medicines, it's about real people with cystic fibrosis and their health and hopes for the future. We asked five people with CF what access to precision medicines would mean to them - watch their responses in the video below.

Scotland

In May, the Scottish Medicines Consortium rejected Orkambi and Kalydeco for two-to-five-year-olds on the NHS in Scotland. In response we asked you to write to your MSPs, urging them to ask the Government to reconsider. We have been pleased to confirm that access to Kalydeco has now been granted and that discussions around Orkambi are ongoing.

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

  • 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
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