We are supporting the CF community and will fight for access to Kaftrio for all ages. We will forward your story to NICE in support of Kaftrio for 2-5 year olds.
Email contactus@cysticfibrosiscare.org.uk.
On November 3rd, NICE released an update on their assessment of the effectiveness of modulator therapies. They acknowledged the clinical effectiveness and significant benefits of Orkambi, Symkevi, and Kaftrio for people with CF. However, they deemed these treatments too costly at their current list prices to recommend them for NHS use.
It is important to note that this decision is not final. There will be a 4-week consultation period, followed by a second committee meeting in December to discuss the feedback received during the consultation. During this period, there will be the opportunity to respond to the consultation and share the perspectives and experiences of individuals with CF and their families.
It has been clarified by NICE, the NHS, and Vertex that this does not affect people with CF who are currently undergoing modulator therapy.
Cystic Fibrosis Care are supporting Shannon’s petition to keep Kaftrio on the NHS. Please support her and sign it.
https://www.change.org/p/make-kaftrio-affordable-for-nhs
Indiana Smith Age 4 & Everyone Else That Has Cystic Fibrosis
NICE on the 3rd of November 2023 that Modulator therapies currently used to treat Cystic Fibrosis should no longer be funded on the NHS for those who can’t currently access them.
My 4 year old daughter Indianna Smith does.
Children with Cystic Fibrosis have to take multiple medications / antibiotics Physios / Nebulisers / Vitamins daily.
They have regular hospital appointments and have to undergo various different tests such as X Rays/ CT scans.
My daughter has stayed in hospital multiple times due to her condition.
As parents to children with Cystic Fibrosis we carry a huge burden of care and work hard daily delivering their treatments to keep them alive.
Without modulator treatment the condition can be fatal at a much lower age and cause suffering and an unfulfilled life for those unable to access it.
Kaftrio is known as a miracle drug, it has completely transformed and saved the lives of those who are fortunate enough to have already accessed it. It has so many positives including that it adds on 40+ years to the lifespan of someone with CF and enables them to live a more comfortable and longer life. It is currently licensed for 6+ in the UK.
However the news from NICE on Friday has questioned whether it will continue to be funded for those like my daughter who aren’t yet at the age to benefit from it. If this goes ahead it will be devastating for the CF community.
Please can you help us by raising this matter in Parliament and getting it back on the agenda for all of the children with CF that so desperately need and deserve the treatment and a chance at a better life.
The Cystic Fibrosis Community would be extremely grateful.
https://www.bbc.co.uk/news/articles/c725x2djn16o
BBC News reported:
7 November 2023
A mother said her daughter’s future has been “ripped away ” after NICE withdrew its recommendation for a number of drugs to treat cystic fibrosis (CF).
Sarah’s four-year-old daughter Lucy has the condition and her family believed she would be eligible for the drug Kaftrio at the age of six.
However, NICE’s draft guidance said it would not recommend three treatments, including Kaftrio, for new patients.
In a statement, it said it had been “evaluating the cost-effectiveness” of “these effective medicines” and a consultation with stakeholders would help it reach its final decision.
Sarah, who lives in Wingham, Kent, said: “I am utterly heartbroken right now, I have always been scared and worried about my Lucy’s future due to her CF, but now I am petrified.
“It feels like a lifeline that was being handed to 90% of the CF community has just been ripped away. To hear the news that she won’t be receiving Kaftrio ever is horrendous.”
Sarah described the decision of health bosses as “incomprehensible”.
Children with CF have a shortened life expectancy.
Lucy’s family say it’s “horrific” that she may be not be eligible for the “miracle” drug
The draft guidance from NICE is open for consultation until 24 November.
It would not affect those who already take Kaftrio or Symkevi, which are currently recommended for patients aged six and over, or Orkambi, which can be used at age one and over.
If passed, young children or babies who are yet to receive these therapies would not be guaranteed them under NHS care, despite NICE’s view there are major benefits.