Thu, 31 Oct 2019
IT could change my life for the better.”
That was the reaction of cystic fibrosis (CF) sufferer Tilly Green, from Bradfield, after the NHS struck a deal to provide a life-changing drug.
Tilly, aged 11, and her parents Ross and Nikki have been campaigning for Orkambi to be made available through NHS England.
And last week, following a long-running battle and families talking of a buyer’s club to purchase the drug, the health service announced it had reached an agreement with Vertex Pharmaceuticals to provide Orkambi and two other cystic fibrosis drugs.
Reacting to the life-changing news, Tilly said: “I am really pleased that NHS England have now agreed with Vertex and decided to bring Orkambi to England.
“This could be life-changing for me because I have the same gene mutation that Orkambi helps.
“It could change my life for the better. Yay!
“I want to say a big thank you to my classmates and friends from when I was at Bradfield Primary School and my teacher Mrs Dennis for supporting me and the Orkambi campaign and also Richard Benyon for getting involved.
“A big well done to every CF sufferer and their families out there and another huge thank-you to the NHS for finally agreeing to change lives.
“Well done everyone, we did it!”
Orkambi is already available in several countries.
Although it was licensed for use in the UK, Orkambi could only be prescribed to people with CF on compassionate grounds.
CF causes fatal lung damage and affects around 10,400 people in the UK.
Only around half of those with the condition live to celebrate their 40th birthday.
Tilly currently takes around 40 different tablets each day for her CF.
But the family are hopeful that Orkambi will be prescribed at her next check-up in November.
Tilly’s mother Nikki said: “I don’t think we can quite believe it at the moment.
“When you have waited for something for that long it’s like ‘my goodness’.
“It’s wonderful news. Really wonderful news.
“It’s unbelievable, it needs to sink in.
“It’s been such a long time coming and such a lot of hard work and effort across the country.
“It should hopefully mean less infections, less tummy aches, less hospital stays and hopefully a longer and happier life.”
Mrs Green said the family had been looking at houses in Scotland, where Orkambi is available, in case the deal did not go through.
But now she hopes Tilly could have Orkambi within 30 days.
She said: “They are not hanging around with this.
“We could be walking out with a prescription.
“The buyer’s club, that’s something now we don’t have to consider, or moving.”
NHS England said it had been able to finalise the negotiation as Vertex had agreed confidential commercial terms that constituted good value for British taxpayers.
Vertex also agreed to submit its drugs for full National Institute for Health and Care Excellence (NICE) appraisal.
Newbury MP Richard Benyon, who was quizzed about Orkambi on a visit to Tilly’s school, said: “I am delighted that Tilly will now have access to this drug.
“I was so impressed when I visited her class at Bradfield Primary – she and her parents, Nikki and Ross, and her school friends have been inspirational in this campaign and this announcement will make a real difference to her and to others with cystic fibrosis.”
NHS chief executive Simon Stevens said: “The UK has the second highest prevalence of cystic fibrosis of any country in the world, so today is an important and long hoped-for moment for children and adults living with cystic fibrosis.
“That fact also means that any drug company wanting to succeed commercially in this field needs to work constructively with the NHS.
“I’m pleased that Vertex has now agreed a deal that is good for our patients and fair to British taxpayers.”