*** Breaking news*** On 12 August the Scottish Medicine Consortium announced it was not able to recommend Orkambi and Symkevi for routine use by NHS Scotland, adding that the company had failed to justify the price they were offering.
Read the whole story and find out how you can take part in a campaign day to demand Vertex and the Scottish Government offer a fair deal for people living with CF across the UK.
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.
Which precision medicines treat cystic fibrosis?
Two precision medicines that help to treat the underlying cause of cystic fibrosis have been licensed for use in the UK, while others are currently being tested on people with CF to determine how they work.
Orkambi (ivacaftor/lumacaftor) targets a mutation that around 50% of people with cystic fibrosis in the UK have and has been shown to improve lung health and reduce hospitalisations. Although it has been licensed for use in the UK, the National Institute of Clinical Excellence (NICE) have not recommended it for use on the NHS, and it is currently only available rarely on compassionate grounds.
Kalydeco (ivacaftor) targets a mutation that under 10% of people with CF in the UK have. Following successful campaigning by the Trust, Kalydeco is available to everyone over the age of two who is eligible and the drug has been shown to significantly increase lung function and reduce hospital admissions and the progression of lung disease .
Symkevi (a combination of ivacaftor and tezacaftor) targets multiple mutation combinations. These include people with two copies of F508del (which around 50% of people with CF in the UK have) and people with F508del and one of 14 less-common 'residual function' mutations (approximately 5% of people with CF in the UK). This treatment has been shown to improve lung function and reduce hospitalisations. Symkevi was licensed for use by the EMA in November 2018 but is not currently routinely available to the people who can benefit from it in any part of the UK.
Triple combination therapy
The triple combination therapy uses a combination of ivacaftor, tezacaftor and one of three new compounds, and could work in people with one copy of the F508del mutation and one copy of a 'minimal function mutation' - approximately 90% of the CF population.
How are we campaigning for access to these drugs?
The Trust has been campaigning hard to ensure that people with CF can access innovative precision medicines. Find out more about the work we're doing to extend access to precision medicines by clicking on the different drugs below, finding out how we're campaigning in your area, joining a campaign or exploring our campaign timeline and latest news.
Take a look at the list of parliamentarians who have supported our campaign for access to life-saving drugs.