Latest update: 1,000 day Orkambi anniversary, Early Day Motion and tezacaftor-ivacaftor
1,000 days since Orkambi was licensed
15 August 2018 marks 1,000 days since Orkambi was licensed, but we’re still fighting for access to the drug for people with cystic fibrosis in the UK. Help us to highlight this by sharing your story on social media with your Health Minister: @MattHancock, @JeaneF1MSP, @wgcs_health or @hscpermsec. Don't forget to tell them what has happened to your or your family member's health in the past 1,000 days.
Use our template press release to contact your local media outlets.
Early Day Motion
Following CF Week and the community-led protests in London and Belfast, we’re keeping access to medicines on the political agenda with an Early Day Motion (EDM). The Trust has been working with the Rt. Hon Sir Mike Penning who has tabled this motion. Please contact your MP and ask them to sign ‘EDM 1327’ to support our campaign as we head into the parliamentary summer recess. Download our template letter when contacting your MP, and be sure to personalise it with your own story.
Contact your MP
What is an Early Day Motion?
An Early Day Motion, or EDM, is a statement put forward by a member of Parliament to call for action from the Government. MPs can show their support for it by adding their signatures.
Who can sign it?
Some members of Parliament may not be able to sign it if they also fulfil a ministerial role, but it is still worth contacting them to find out if they are able to support by either writing an individual letter or encouraging other MPs to sign it.
What will this achieve?
It is vital that we get as many MPs to sign this motion as possible across the UK to ensure that the Government keeps up the pressure on Vertex and the NHS to reach a resolution as a matter of urgency. It will also enable us to gain more UK-wide MP support for our campaign for access to precision medicines.
Contact firstname.lastname@example.org for more information.
New drug tezacaftor-ivacaftor (Symdeko) has completed clinical trials, but before any decisions on its use in the NHS are made, it must be granted a license for sale by the European Medicines Agency, which is due to be rubberstamped this year. We want to make sure that we are representing what matters to you when it comes to the treatment choices you make.
Please complete our short survey and make your voice heard in our evidence submission to healthcare watchdogs like the Scottish Medicines Consortium (SMC) and the National Institute of Health and Care Excellence (NICE) as they prepare to review this new treatment.
Fill in the survey
Find out more
Read a feature by Nick Medhurst, Head of Policy at the Cystic Fibrosis Trust, to find out more about the review process and how your views have helped in the past.
Writing to your MP
Below, we have put together some template information that can help you to ensure your voice is represented by your MP. You can also find out how many people in your area signed the Orkambi petition.
What should you tell you MP, and what do they need to do?
Use this template information when emailing or writing to your MP about how Orkambi or other precision treatments could help you or your friends or family:
- People with cystic fibrosis are being denied access to vital new precision treatments that NICE recommendations show can add years to their lives
- The UK Government must take responsibility for negotiating an agreement as soon as possible
- Drug company Vertex must ensure fair and responsible pricing for a deal to be agreed
- The UK Government must acknowledge the importance of finding a solution that guarantees we are never put in this position again for the future pipeline of treatments for cystic fibrosis.
Spread the word!
When you’ve written to your MP, it’s time to use your voice to rally round – ask your friends and family to write to their MPs too – share this page on Facebook and Twitter – or send them the link cysticfibrosis.org.uk/YourVoice.
What does your MP need to ask of the UK Government?
Your MP must ask the UK Government whether they will take responsibility for making progress in negotiations for access to Orkambi and future cystic fibrosis medicines.
Frequently Asked Questions
Here are some questions that your MP might want to know and some extra information that could help you to form your correspondence. You can find out more on our access to medicines FAQ page.
What is cystic fibrosis?
Cystic fibrosis (CF) is a life-shortening genetic condition that affects over 10,400 people in the UK. Last year, half of all people who died with cystic fibrosis were under the age of 31.
What is Orkambi?
Orkambi is the only precision medicine licensed for people with cystic fibrosis who have a specific set of CF-causing genetic mutations, known as your ‘genotype’. Orkambi works for people who have two copies of the F508del mutation – around 50% of people with CF in the UK.
Traditional treatments for cystic fibrosis aim to lessen symptoms and complications. However, progressive damage still occurs, meaning symptoms and complications will increase with age.
Orkambi is a revolutionary medicine that tackles the root cause of cystic fibrosis. It is proven to slow decline in lung function by 42% and cut the number of infections requiring hospitalisation by 61%. This gives people more control and greater quality of life.
It was licensed for use in Europe two and a half years ago and is available in Austria, Denmark, France, Germany, Luxembourg, the Netherlands, Italy, the Republic of Ireland, Greece, and the United States.
In 2016, both NICE and the SMC announced they were unable to recommend the drug due to its cost, despite acknowledging the drug is “important and effective”.
Since then, the Cystic Fibrosis Trust and the CF community have relentlessly called for negotiations and a fair, sustainable pricing deal for Orkambi, as people with cystic fibrosis wait and their health and quality of life declines.
By 2020, development of new drugs could mean 90% of people with CF will be eligible for treatment that targets the root cause of the condition. The UK Government must act now to agree a sustainable solution for access to cystic fibrosis medicines now and in the future.
What is Vertex’s proposal?
Drug manufacturer Vertex submitted a fresh proposal to NHS England in February 2018 for access to its current and future cystic fibrosis treatments including Orkambi.
The manufacturer claims this approach will help provide equitable access to their medicines for people with CF in a way that could enable the NHS to budget for the longer-term.