A new treatment for cystic fibrosis has received a lot of media attention in the UK after the National Health Service (NHS) chose not to fund it due to its high cost. Cystic fibrosis advocacy groups have been petitioning the government to intervene, and the singer Taylor Notcutt has given her support to the campaign through the release of her new single, a cover of the song ‘breathe with me.’
Cystic fibrosis (CF) is a life-shortening genetic condition. Those with the disease develop a build-up of mucus in their lungs and other organs that affect people’s ability to breathe, amongst other symptoms. The condition is life-long and affects over 10,400 people in the UK. Current treatments available on the NHS focus on treating the symptoms, rather than the underlying cause of the disease. Patients may be offered medicines to fight infections and reduce mucus in the lungs, as well as advice on helpful exercises and airway clearance techniques.
However, a recent drug called Orkambi (lumacaftor) addresses the underlying cause of CF. It is a precision medicine that targets a specific genetic mutation that 50% of people with CF in the UK have. Although it is not a cure, it has shown to be effective at treating CF; the drug slows the decline of lung function, the most common cause of death in those with CF, by 42%. Patients who take it can also have a reduction in hospital treatment of chest infections by up to 61%.
Although Orkambi has been approved for use in Europe since November 2015, the NHS does not currently provide it except in rare cases for compassionate reasons. The National Institute for Health and Care Excellence (NICE), which is an independent body that provides guidance on healthcare, reviewed the drug in July 2016 and found that although it is an “important treatment” it could not be recommended for NHS use due to its cost. The organisation cited a lack of long-term data on Orkambi’s effects, and its steep price of £104, 000 (~$140, 000) per person per year as the reasons behind their decision.
Patients, friends, and family of those with CF have been petitioning the government to intervene in this decision and make the drug available on the NHS. At its current price the vast majority of patients are unable to afford it, but if the NHS decided to offer the drug it would become available to all UK patients.
A petition, which can be found here, is calling on the British Government to find a way for the NHS to supply Orkambi “as a matter of urgency.” The petition reached 100, 000 signatures in just over 10 days, and as a result, sixty MPs debated the petition on the 19th March at Westminster. This is much higher than the average number of 12 MPs that attend debates. A letter-writing campaign also led to four-year-old Ivy Weir, who has CF, delivering one thousand handwritten letters to Downing Street calling on Prime Minister Theresa May to personally intervene and make the drug available. Ivy’s mum, Gemma, said,
“If other European Countries have been able to find a way then […] the UK should be able to as well.”
Theresa May has responded by saying that there are on-going talks between the NHS and Vertex, the company behind Orkambi, and that she supports a “speedy resolution” to the negotiations. The last reported meeting between the two organisations was on the 25th April, and the next is planned on the 25th May. In a statement, Vertex said that the talks had been “constructive” but that “there is still some way to go.”
As talks progress, CF advocates continue to put pressure on the government to make the drug available. Two protests have already been held in London this year, and a third will take place on June 22nd. The public is also being encouraged to become involved through the hashtag #YouAreMyVoice, which highlights the need for the small CF community to be supported by others. People have also been getting involved by sharing #OrkambiNow selfies, including the soap opera actor Tristan Gemmill.
The singer Taylor Notcutt has also released ‘Breathe With Me’, a song that supports those with CF and the on-going campaign.
Taylor says,
“If you listen to the lyrics of ‘Breathe With Me’ they are asking people, the general public, to think about how difficult life would be if you had to struggle to do the one thing most of us take for granted every day – breathing.”
“Try breathing through a narrow drinking straw for just a minute, while pinching your nose – that will give you some idea of how bad this disease can get. There are many complications too – it’s not just the lungs that are affected. If the public gets some idea of having to live like that every day, then I am sure that they will want to help too.”
The song, which benefits the Cystic Fibrosis Trust, can be downloaded here.
CF advocates are encouraging everyone to become involved with the campaign. People can do this through sharing the song on social media, writing to a local MP about making Orkambi available, signing the online petition, sharing selfies, using the #YouAreMyVoice, and attending the planned protest.
Would you like to become involved with this campaign? Share your stories, thoughts, and hopes, with the Patient Worthy community!
To find out more about CF, check out our partners Cystic Life and Strawfie Challenge.
