Children with cystic fibrosis look set to be denied lifesaving treatment

2 mins read

Tuesday 14 November 2023

Families have warned that latest draft guidance from the National Institute for Health and Care Excellence (NICE) will shorten their children’s lives. 

NICE, the body that reviews and evaluates treatments, has concluded that some effective therapies available on the NHS should no longer be prescribed to new patients on cost grounds. 

Cystic fribrosis (CF) is a progressive genetic condition that leads to problems with breathing and digestion over time. Around one in 2,500 babies are born with the condition.  

NICE has spent a year evaluating a relatively new class of “modulator therapies” that can improve lung function, growth and weight gain in people with CF. 

Patients with CF told the NICE evaluation team that the new treatments had transformed the disease from a progressive and life-limiting condition into a manageable chronic condition. 

But the therapies are expensive, and NICE is now recommending that no new patients are offered the drugs. Patients already on the drugs will continue to receive them. 

“We are being forced into a situation where we are going to have to watch our daughter deteriorate year on year until she dies at a very young age. I just can’t do that,” Charlotte Perkins, mother of a 17-month baby told the BBC last week. 

Share your views in the NICE consultation

People have until 24 November to respond to a consultation on the guidance.

You can read the draft guidance and submit a response on the NICE website. Respondents will need to create a free account with NICE to submit a response. 

Will the proposals affect your child?

Do you want to share your experience and concerns? Please email