Campaigning for Orkambi in your region
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.
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.
Take a look at the list of parliamentarians who have supported the campaign for access to Orkambi.
In your region
We are currently working on a number of regional campaigning actions that we would like you to get involved in. Scroll down to find out more.
Thank you to everyone who attended our parliamentary event on 29 November at the Palace of Westminster, sponsored by Ian Austin, MP for Dudley North. This event was a brilliant opportunity to get MPs from across all political parties in one room with representatives from the Trust and campaigners to share their own personal stories and highlight why our campaign is so vital to those living with the condition.
Following on from this event, a cross-party round table meeting chaired by Ian Austin will be held on the morning of 28 February to continue discussions with Vertex. We urge everyone who can, to please contact your MP and let them know about this event. MPs can contact Ian Austin directly for further details. For advice or support on this - or to find your local MP - get in touch with our campaign team at email@example.com.
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:
We have engaged with the Scottish Parliament and MSPs with the aim of debating the wider issue of access to medicines, such as Orkambi, in Scotland. We have motioned S5M-09067 for debate and we will keep you updated on the progress of the Motion and any subsequent debate as we learn more.
Find your MSP
We released a petition to the Welsh Assembly calling for a resolution to negotiations between NHS Wales and Vertex. We surpassed our 5,000-signature target and the issue of Orkambi was automatically considered for debate by the petitions committee on 23 January.
The committee decided against a debate on access to Orkambi, but we welcome their decision to take action by writing to the Welsh Health Minister regarding the current Welsh Government position, ahead of further potential discussions and debates on the issue.
Members of the petitions committee demonstrated an interest in the issue of our petition and recognised the situation as a stalemate which must be addressed. The importance of considering the more recent data available was also raised. A decision was made to have further discussion with the Minister with a view to having another meeting, which we expect will play out over the next couple of weeks.
A debate was not chosen as the best course of action at this stage, but in principle the nature of the petition is something that could potentially be debated in the future and 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.