Hope for wonderdrug to help little Harriet who needs to take a cocktail of drugs each day in battle with cystic fibrosis
A four-year-old cystic fibrosis sufferer is a step closer to receiving the treatment she needs, after health minister Matt Hancock met with bosses of US drugs firm Vertex.
Harriet Corr of Jarrow suffers from the condition which was diagnosed when she was just five-days-old.
The St Matthew’s Primary pupil needs a daily mixture of drugs and vitamins, as well as physiotherapy to deal with the cystic fibrosis which causes lung damage and affects about 10,400 people in the UK.
But the oral medicine Orkambi, which is used to treat cystic fibrosis, isn’t available on the NHS in England.
The drug costs a reported £104,000 per patient, per year, although Vertex disputes that figure.
The drug is already available in Scotland after a deal was struck with the Scottish government in September and Harriet’s family had even discussed moving there.
Harriet, who turns five on October 17, lives with her parents Emma, 39, dad Chris, 37 and big sister Nancy, seven.
Health Secretary Matt Hancock has now held talks with Vertex bosses and, although nothing has been finalised, both parties are confident of reaching an agreement. According to Vertex the drug could be available “within weeks”.
The family is excited by what an NHS agreement with Vertex could mean to them.
Emma, an English teacher, said: “I feel this is the closest we’ve ever come in all these years. Scotland has already got it.
“I’m really excited. This would be Harriet’s best birthday present ever; to sort this out after so many years.
“We’ve had our hopes raised so many times. But this is the closest we’ve been.
“The fact that both parties have come out with positive statements is encouraging. We’re hoping and praying. We always stay positive and it’s within reach.”
Vertex had turned down an offer from the NHS of £500 million over five years for access to Orkambi and other cystic fibrosis drugs. The company claimed that if the offer was replicated globally it would go out of business.
Mr Hancock said: “Cystic fibrosis is a terrible condition and I know the devastating impact it can have on people, their family and friends.
“I had a constructive meeting with Vertex, NHS England and NICE (the National Institute for Health and Care Excellence) to discuss how we can get treatments to those who desperately need it.
“A number of positive ideas were put forward and our teams are continuing to engage on the technical aspects of these ideas.
“I hope we can come to a speedy solution as soon as possible and get these treatments to the people who desperately need them.
In their statement, Vertex said: “Our commitment to securing access for patients is unwavering.
“We will keep working every day to make sure all eligible patients have access to our cystic fibrosis medicines in the UK.
“Since our last meeting (with Mr Hancock) we have met with NHS England nine times and brought numerous different solutions to the table.
“Today we brought another solution that would provide immediate access and are committed to trying to negotiate this to a formal agreement within weeks.”