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Devon mum fears for child's access to cystic fibrosis drug

BBC Matilda with inhaler and mum RoseBBC
Matilda's mother Rose said potentially not having access to the drug was "the most heartbreaking thing"

A mother from Devon fears her young daughter with cystic fibrosis may never get a drug that could prolong her life.

Matilda Blatchford, four, was diagnosed with the condition at three weeks old and her family said they hoped she would get Kaftrio from the age of six.

New draft guidance from regulator the National Institute for Health and Care Excellence (NICE) does not recommend it.

Helen Knight, from NICE, said its "cost-effectiveness" was being gauged.

Matilda Blatchford with inhaler
Matilda Blatchford's family hoped when she turned six she would get the drug Kaftrio

Matilda's mum Rose Blatchford said: "We had hopes of her having children, retiring ... without Kaftrio we don't know if any of that will be possible."

"Knowing that that drug's there and available and not having access to it, is the most heartbreaking thing ... so we feel totally helpless at the moment.

The drug costs £8,346 for a packet of 56 tablets, according to NICE.

It means NHS treatment costs could amount to between £100,000 and £200,000 per year for each patient.

The Department of Health and Social Care said: "It is vital that patients have access to new and innovative medicines like these, but the NHS must use its budget fairly and for the good of all patients."

It said it had invested more than £1.1bn of funding into research of rare diseases.

'Not right'

NICE published draft guidance on 3 November did not recommend Orkambi, Symkevi and Kaftrio for the treatment of cystic fibrosis.

A public consultation on the situation closes on 24 November and NICE is expected to publish final guidance next March.

Mrs Knight, NICE's director of medicines evaluation, said work was continuing "to deliver the best outcome both for people with cystic fibrosis and for the wider NHS".

Matilda's father Scott Blatchford said: "Matilda never asked to have cystic fibrosis ... but there's a drug out there that could treat it and give her a normal life. It's just not right."

Existing patients and new patients who have been started on the treatment will still have access to the drugs regardless of the outcome of the consultation, health bosses said.

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