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Cystic Fibrosis Trust

Orkambi

Orkambi (ivacaftor/lumacaftor) is the second precision medicine to be licensed for use in the UK by people with cystic fibrosis (CF), although it is not currently provided by the NHS except in rare cases on compassionate grounds.

Campaigning for Orkambi in your region

England
Northern Ireland
Scotland
Wales

What is Orkambi?

Orkambi is the second such drug to be licensed for use in people with cystic fibrosis, the first was Kalydeco.

  • Orkambi treats the F508del mutation, which around 50% of people with CF in the UK have.
  • Orkambi is a combination medicine, made up of ivacaftor and lumacaftor. Lumacaftor helps get more proteins to the surface of cells in the body, and ivacaftor helps the chloride channels in the cells to operate more effectively. The combination of these two things helps to keep a healthy balance of salt and water in the organs – particularly the lungs. Orkambi is manufactured by Vertex Pharmaceuticals.

Is Orkambi available in the UK?

  • According to the 2014 UK Cystic Fibrosis Registry Report, there are 2,834 people in England, 243 people in Scotland, 118 people in Wales and 101 people in Northern Ireland who could benefit from Orkambi.
  • Orkambi has been licensed for use in the UK for people with CF over the age of 12 who have two copies of the F508del mutation. Despite this, the National Institute for Health and Care Excellence (NICE) has rejected its use on the NHS.
  • In May 2016, the Scottish Medicines Consortium (SMC) noted that Orkambi was a beneficial treatment.In June 2016, NICE recognised Orkambi as an important treatment.
  • Both the SMC and NICE were unable to recommend Orkambi for general use within the NHS/HSCNI on grounds of cost-effectiveness and a lack of long-term data.
  • Orkambi is currently only prescribed to people on compassionate grounds. Compassionate use means that Vertex Pharmceuticals provide the drug to people who fulfil a number of criteria. Contact your clinician for more information on compassionate use.
  • Orkambi was licensed for 6-11 year olds by the European Medicines Agency in Europe in January 2018.

What effect does Orkambi have?

96-week data shows that Orkambi can slow decline in lung function - the main cause of death among people with cystic fibrosis - by 42%. This data was unavailable to NICE when they appraised Orkambi.

Why is Orkambi not available to everyone who needs it?

The processes involved in making drugs available to the people they could help are complicated. Creating and testing a drug like Orkambi is very expensive and a lack of long-term data can make it hard for NICE and the SMC, the bodies that appraises these drugs for use in the NHS/HSCNI, to accurately assess their cost-effectiveness in order to make them available.

What are we doing about it?

Stay up to date with Stopping the Clock, our dedicated campaign to put pressure on the Government, NHS and pharmaceutical companies to ensure these drugs reach the people who need them without delay, or join the campaign and get involved yourself by taking part in a region-specific action below.

Supportive parliamentarians

Take a look at the list of parliamentarians who have supported the campaign for access to Orkambi.


In your region

Following the roundtable and debate we asked you to contact your MPs to ask them to contact Ian Austin MP to sign a cross-party letter. The letters were then sent to Greg Clark MP and Jeremy Hunt MP - scroll down in the PDF to see if your MP signed the letters.

Read the latest update on the Stopping the Clock campaign from Lynsey Beswick, Public Affairs Manager at the Trust.

England

A cross-party letter from Ian Austin MP was signed by 125 MPs and sent to Jeremy Hunt MP and Greg Clark MP. Please see the letter above to find out if your MP added their signature. Vertex met with NHS England on 25 April and we understand that discussions are ongoing.

Northern Ireland

We have sent a cross-party letter to Jeremy Hunt, Secretary of State for Health and Department of Health Permanent Secretary, Richard Pengelly, calling for a timely resolution to the ongoing negotiations between Vertex and Health and Social Care Northern Ireland. These letters have been signed by 66 MLAs from a number of different parties.

Read the letters:

We have received a response to our letters from Richard Pengelly, which you can read here:

Scotland

Following the recent Vertex proposal submission to NHS Scotland, Jackie Baillie MSP will be chairing a cross-party meeting at Holyrood on Wednesday 23 May at 5.30pm to bring together MSPs, Cystic Fibrosis Trust Chief Executive David Ramsden and a patient representative to discuss access to precision medicines in Scotland.

We're asking you to contact your MSP using the template letter below and request that they get in touch with Jackie Baillie's office to confirm their attendance. Don't forget to include your own story in the template to let your MSP know why the fight for access to medicines is so important. Please let us know if your MSP will be attending so that we may contact them to provide a briefing.

Download the template letter

You can also request that your MP engage with their local MSP counterpart to apply pressure in the devolved parliaments.

Wales

A petition to the Welsh Assembly calling for a resolution to negotiations between NHS Wales and Vertex surpassed our 5,000-signature target and the issue of Orkambi was automatically considered for debate by the petitions committee on 23 January and again on 13 March.

The Welsh Assembly petitions committee discussed access to Orkambi in Wales petition again on 15th May. It was agreed they will request an evidence session with Vertex and the Cabinet Secretary for Health in Wales. This is likely to take place in July and we will update when we know more. Latest correspondence available here including Trust responses. 

The Trust has been meeting and having discussions with Welsh MPs to continue dialogue in Wales. We will continue to apply pressure once the Assembly processes have been followed.

For the latest news on our Stopping the Clock campaign and other updates in the CF community, sign up to receive our e-newsletter.














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