A YATE mum headed to Parliament this week, where access to a treatment that could change her three-year-old daughter’s life was being debated.

Liz Brennan is fighting to get access to Orkambi, which she believes would make a massive difference to the health of her daughter Annabelle, who suffers from cystic fibrosis (CF).

The drug has been approved for use in the UK for the last two years, but NICE have been unable to recommend it for general use within the NHS - on the grounds of cost effectiveness and a lack of long-term data.

The government debated access to the drug on Monday, after more than 110,000 signed a petition on the UK Parliament website.

This followed an announcement on Friday that the NHS had rejected the latest offer made by Orkambi manufacturer, pharmaceutical company Vertex.

Liz travelled up for the debate, where it she says it was standing room only.

"Sixty MPs attended the debate, and 22 gave speeches, including our MP Luke Hall, who did a wonderful job," she said.

"The public gallery was full, and the overspill gallery, where the debate can be watched on a screen was full too. There were protestors outside as well, it was a really good turn out.

"The MPs were really compassionate and speaking from the heart. They strongly defended our right to get this drug," said Liz.

"For the last couple of years we've been ignored and dismissed by ministers, but now we're getting their support. It gives us all hope, and shows what people power can do."

Thornbury and Yate MP Luke Hall was one of those to give a speech, in which he made the case for the NHS to pay for the drug.

He said: "The petition for today’s debate was signed by 470 people from my constituency.

"I am here speaking on their behalf, and on behalf of Annabelle Brennan, and her parents Elizabeth and Shane.

"Annabelle is three years old, and is a lovely young girl. I met her at a cystic fibrosis campaigning event in my constituency.

"She does things that other young three-year-olds do, of course, but she also has to endure a complex daily routine of physiotherapy, nebulisers, medication and antibiotics to help her to manage and live with the illness.

"I received an email from Annabelle’s nursery leader, who is also my son’s nursery leader, explaining that Annabelle’s time at nursery, and her interaction with the other children in the group, is affected by her condition, because she is under constant supervision and is not always able to be involved in all the activities like the other children.

"Every winter, there is an added risk of further infection due to the common cold, and, as time passes, the potential for more complications increases.

"I remember hearing a devastating description from Annabelle’s parents of how every day is like waking up on a cliff edge, not knowing when irreparable damage caused by CF will come. That highlights the importance not only of these debates and the pressure, but of getting a deal, so that Orkambi can be made available as quickly as possible.

"That would ensure that children such as Annabelle, and people living with CF throughout the country, can realise their full potential."

Addressing Health Minister Steve Brine, he added: "I finish by saying to the Minister, NHS England and Vertex, “Can we please ensure that a deal is reached as quickly as possible, because this drug has the potential to change thousands and thousands of lives?”

A spokesperson for Vertex said: “The scale of parliamentary engagement in this debate echoes and amplifies the cystic fibrosis (CF) community’s sense of urgency on access to new Vertex medicines.

“CF is a devastating disease where half of people die by the time they are age 31 – and the UK has the second highest number of CF patients in the world. 

“As many MPs reflected in the debate, the key challenge we face is the NICE approval process which was developed nearly 20 years ago before precision medicines like Orkambi for small patient numbers existed – as one participant in the debate said – ‘it is an analogue process for a digital age’.

“Every day matters for CF patients and we stand ready and willing to engage in meaningful dialogue about a way forward.”

An NHS spokesperson said: “The NHS can only offer treatments which are both effective for patients and offer good value for taxpayers, so it’s crucial that drugs companies work with the NHS to get a positive outcome. Following advice from NICE, the NHS has asked this particular drug company to review its proposed pricing, and unless this happens further progress at this time is frankly unlikely.”

Negotiations for Orkambi's use within the NHS continue.