Call The Midwife's Jenny Agutter begs NHS regulator not to deny children 'miracle' cystic fibrosis drugs
- Jenny Agutter is a carrier of the gene that causes cystic fibrosis (CF)
Jenny Agutter has warned that plans to restrict access to 'miracle' drugs for children with cystic fibrosis would be a 'death sentence' for a new generation born with the disease.
The actress, who is a carrier of the gene that causes cystic fibrosis (CF), urged the NHS regulator to think again after it announced it was not cost-effective for Kaftrio to be prescribed on the health service.
The National Institute for Health and Care Excellence (NICE) made the recommendation about the drug – dubbed 'almost a cure' for children with the debilitating lung condition – last month.
It indicated that the 8,000 UK patients already using Kaftrio – and its sister drugs – will be able to stay on them. But, under the proposals, unborn children or babies yet to be diagnosed may not be eligible.
Jenny Agutter has warned that plans to restrict access to 'miracle' drugs for children with cystic fibrosis would be a 'death sentence' for a new generation born with the disease
CF, which causes a build-up of sticky mucus in the lungs, digestive system and other organs, has been part of Ms Agutter's life since her niece was diagnosed as a baby, 45 years ago. Then, children with the disease were not expected to live past their eighth birthday.
The actress's great-nephew, Albie, was also diagnosed with the condition soon after he was born.
After NICE's announcement, the 70-year-old Call The Midwife star said she had been inundated with letters and emails from desperate parents fearful for their childrens' futures. She said: 'The series of drugs that include Kaftrio are revolutionary. Restricting their prescription on the NHS would be a death sentence for a whole generation.
'There will be families out there whose toddlers qualify for Kaftrio but who may go on to have other children with the condition that miss out. How can that be right?
'These drugs are expensive but surely not as costly to the NHS as CF patients whose health will be so much worse without them. To me, the policy not to fund the drugs appears financially short-sighted.'
Rachel McGrath (left) and Jenny Agutter. McGrath has been taking Kaftrio since the end of 2019 and has seen her physical health transformed as a consequence
Kaftrio, which has proven effective for around 90 per cent of CF patients, is made by US drug giant Vertex Pharmaceuticals. It only became available on the NHS in the UK from 2019 after years of wrangling with Vertex over cost. It is thought to cost the NHS more than £100,000 a year per patient.
Ms Agutter's niece, Rachel McGrath, has been taking Kaftrio since the end of 2019 and has seen her physical health transformed as a consequence.
Before, she would spend up to three hours a day on a nebuliser – a device that turns liquid medications into a fine mist so they can be absorbed by the lungs.
Ms Agutter's great-nephew, Albie, has a form of CF which does not make him suitable for the drugs.
But if NICE's recommendations are given the go ahead thousands of other babies and unborn CF sufferers who may be suitable will be automatically denied access to the medicine on the NHS.
Ms McGrath said: 'I hope it won't get to the stage that NICE stops funding Kaftrio for new sufferers. I hope it is simply them playing hard ball with Vertex.
'But that won't be any comfort to parents who may have babies newly diagnosed with CF and will be anxious by these developments. It's not just in the UK, all around the world people with CF face not having access to this revolutionary treatment because of cost.'
Campaigners are currently working on collecting evidence on the efficacy of the drugs to submit to NICE in a bid to alter its recommendation. A final decision by the body is likely to be made in the New Year.
