Cystic fibrosis patients call for access to drugs
PATIENTS with cystic fibrosis are being denied access to new “precision” medicines which will prolong and enhance their lives, it is claimed.
More than 3,000 people have signed a petition which calls for drugs like Orkambi to be made routinely available on the Welsh NHS.
But they need 5,000 signatures by New Year’s Eve for the subject to be automatically considered for debate by Assembly Members.
The petition was started by Rhian Barrance, from Cardiff, whose best friend in Northern Ireland has a son born with cystic fibrosis.
After reading up on the condition, Rhian found out how Orkambi was being denied to those it could help across the UK – including 118 people in Wales.
Rhian, 31, said: “I realised that I could help people with cystic fibrosis across the UK by campaigning to make precision drugs like Orkambi available. I would say to people, wherever you live in the UK, please sign this petition and help prolong and enhance the lives of people with cystic fibrosis.”
Cystic fibrosis is a life-shortening, inherited disorder leading to severely reduced quality of life and life expectancy.
Half of all people who died with cystic fibrosis last year were under the age of 31.
People living with this devastating condition experience a build-up of thick, sticky mucus that causes chronic lung infections and progressive lung damage.
Daily life itself can be a struggle and people with the condition face a huge burden of daily treatments.
For some, even the most basic tasks cause such breathlessness that oxygen is required to help them breathe.
There are currently conventional cystic fibrosis treatments available on the NHS that target the symptoms of the condition, but precision medicines like Orkambi tackle the underlying genetic mutations which cause the condition.
Graphic design graduate Beth Clarke, who lives in Splott, Cardiff, was diagnosed with the cystic fibrosis at just 14 months old.
And now, at the age of 36, Beth and her family firmly believed they would be able to access treatments to make her life better.
Her mum, Michelle Morgan, said she feels “let down” that drugs like Orkambi are not routinely available.
She said: “We were always encouraging Beth to look after herself, to keep a good, healthy diet and to make sure she takes physiotherapy and stays fit.
“We promised her that one day new treatments would be available which could dramatically improve her life and that her daily struggles would be worthwhile.
“That day is here, but we are being made to feel like total frauds because despite our promises, the first family of dedicated cystic fibrosis drugs, which are available in many other countries around the world, are not available here.
“It is so unfair and all about money. Beth is currently in a stable condition, but her lungs are only at 65% capacity, and she has to spend three hours every morning and a further two hours every evening taking around 90 different medicines.
“She also regularly goes to hospital. We would love to see if Orkambi could help her.”
Michelle, 62, added; “We don’t understand why new drugs are coming forward that can help people with cystic fibrosis but are not available to people in Wales or the rest of the UK.
“Orkambi is available on compassionate grounds, but that is only for people who have a severely limited lung capacity.
“We know Orkambi is not a cure, but it could help prolong and enhance Beth’s life and we would ask people to sign this petition and urge Assembly members to do all they can to ensure people with cystic fibrosis gain access to precision medicine as it becomes available.”
At present the National Institute for Health and Care Excellence (Nice) are unable to recommend the drug for NHS use because it is not deemed cost-effective.
In response, a Welsh Government spokesman said: “We believe everyone should have access to cost-effective medicines to meet their clinical needs. To achieve this we are guided by the recommendations of the National Institute for Health and Care Excellence (Nice) and the All-Wales Medicines Strategy Group.
“In its appraisal completed in June 2016, Nice concluded that it was unable to recommend the use of Orkambi as its considerable cost was not in balance with its likely benefits and it could therefore not be considered a cost-effective use of NHS resources.”
The Cystic Fibrosis Trust claims Orkambi is suitable for around half of all CF patients and can reduce lung function decline by up to 42%.
To sign the petition, go to www.assembly.wales/en/gethome/e-petitions/Pages/petitiondetail.aspx?PetitionID=1266