A MOTHER has urged a pharmaceutical company to put people before profit and reach a deal on cystic fibrosis drugs that could transform the life of her two-year-old son.

Andrea Jones, who lives near Mold, in Flintshire, is backing a campaign by the Cystic Fibrosis Trust which is hoping to persuade The National Institute for Health and Care Excellence (NICE), NHS England and the pharmaceutical company Vertex, to progress with negotiations over the availability of the drugs Orkambi and Symkevi.

Orkambi is the second precision medicine that targets the route cause of the disease and would benefit about half of people with cystic fibrosis (CF) in the UK, including Andrea's son Archie.

It has been licensed for use for over three years, but a deal is yet to be struck that makes it available for patients in England on the NHS. During that time, a third drug to treat the root cause of the condition, Symkevi, has been approved for use but is also not available on the NHS. Orkambi is available in other countries, including Germany, Italy, Sweden and the US. Patients in Germany have had access to the drug since 2015.

The Government has stressed it will not pay a higher price for Orkambi, leaving the care of thousands of patients in doubt with the Department of Health and Social Care insisting that an offer put forward by NHS England was the "best possible" and the only one on the table.

Talks between Vertex and the NHS on the pricing of Orkambi, which could dramatically improve the health of about half of people with the condition, have gone on for about three years with no breakthrough.

The NHS has offered £500 million over five years and potentially more than £1 billion over a decade for access to Orkambi and other game-changing drugs in the pipeline, one of which could treat 90 per cent of patients.

But Vertex has rejected this offer, saying it is 90 per cent below the price paid in other countries and, if accepted, would lead to the firm going bust within three to five years.

Both NHS England and NICE have criticised the behaviour of the firm and called it an "extreme outlier" when it comes to pricing.

Cystic fibrosis is a genetic condition affecting more than 10,400 people in the UK, caused by a faulty gene that affects the movement of salt and water in and out of cells. It causes sticky mucus to build up in the lungs and digestive system, which causes lung infections and problems digesting food.

"If Archie was to have Orkambi he would have fewer hospital admissions and he would have a much better lung function," says Andrea, who recently raised almost £8,000 for the Trust after staging a charity rugby match between Barbariad Gogledd Cymru and Chester RUFC. "It would improve a lot of things in his life."

Archie's condition has a huge impact on Andrea and her partner Liam Gill's day to day lives. "Years ago they had no treatment but now they have daily antibiotics and physio," she says. "It is things like that we've adjusted to.

"We have to be very careful about cross-infection as well. Archie is not allowed to be around anyone with a cough or cold because it can make him really poorly.

"We try to do normal things like take him swimming but we have to be very aware of soil and water - they can hold a lot of bacteria, so we use a lot of antibacterials too."

Answering questions relating to company profits, Dr Jeff Leiden, chief executive of Vertex, said the company had invested 11 billion US dollars between 2000 and 2016 in research and development but had suffered net losses in 20 out of the 25 years the firm has been in business.

"The NHS are refusing to finance the drug because it's £105,000 per patient per year, so it is a very expensive drug," continues Andrea. "But it actually only costs £5,000 to make it, so that's the frustrating thing. We have seen lots of people go down to London to protest and there are talks again next week, so hopefully they will give some kind of answer but they have been meeting for the last two weeks which does feel massive after the deadlock of the last three years.

"I don't even think Orkambi is the best drug out there because it increases heart rate after time, so it can bring blood pressure problems later in life. However, Symkevi actually tries to treat the root cause of cystic fibrosis rather than just masking it.

"If Archie can get that in his lifetime it will help immensely with opening the gates for the salt and water flow through the body and washing out the mucus. It would help reduce all the physio and maybe he wouldn't have to take so much medication every day."

With the Health and Social Care Committee currently holding an inquiry into the availability of Orkambi on the NHS for people with cystic fibrosis in England, Wales and Northern Ireland, Andrea remains positive despite the frustrations which are shared by many in the CF community. The inquiry is seeking to learn why, after three years of negotiations, the relevant parties have yet to agree terms that will make the life-changing medicine available to people with cystic fibrosis and has requested written submissions from NICE, NHS England and Vertex Pharmaceuticals.

David Ramsden, chief executive of the Cystic Fibrosis Trust said: "We share the deep frustration of people across the cystic fibrosis community that little new evidence was given in today's committee hearing. We've heard enough words, what we need to see is action. We hope that when the parties meet next week, at the forefront of their minds are the lives of people with cystic fibrosis.

"All three parties - NICE, NHS England and Vertex - must reach a compromise and end the agonising three-year wait for these life-saving medicines. Each day that passes without a deal means the risk of more damage to the lives and prospects of thousands of children and adults with cystic fibrosis across the UK. This tragic situation must end. We hope the Committee can support efforts to find a deal. We know from countries around the world that it is possible to reach an agreement and we remain committed to supporting all parties to find a compromise that works. We don't want to see people with cystic fibrosis once again be subjected to the anguish of the divisive public spat. No more excuses, we need action now.'

"We are quite a small community compared with other conditions, so we all support each other," adds Andrea. "We are trying and we need to get the debate into Parliament if they do not reach an agreement next week. We are determined not to give up."

To sign an online petition urging the NHS to supply new lifesaving drugs for cystic fibrosis go to: https://petition.parliament.uk/petitions/231602.