Family consider move to Scotland for girl’s health
A WELSH mum is considering moving her family to Scotland in a desperate bid to to get a drug that could extend her daughter’s life by 20 years.
Eight-year-old Sofia Bow was born with the genetic disorder cystic fibrosis which affects one in every 2,500 babies born.
Mum Rebecka, a solicitor from Merthyr Tydfil, has been fighting for the drugs Orkambi and Symkevi to be made available on the NHS for several years, but at a cost of £100,000 per person per year the Welsh Government has said it is not cost effective.
But this week hundreds of cystic fibrosis sufferers in Scotland celebrated a landmark decision there that a deal on costs had been reached with the Scottish Government.
Pharmaceutical firm Vertex International reached a pricing agreement with the government and said that, as a result, approximately 400 patients will be able to benefit from Orkambi and Symkevi.
The drugs will be made available for five years with a “confidential discount”.
It is great news for sufferers in Scotland, but bitter-sweet news for families living in others parts of the UK who do not have access to the drug.
Rebecka told BBC Breakfast she is going to continue lobbying the Welsh Government to follow suit, but did admit that if a decision was not made soon the family might have to consider a move to Scotland.
She said: “The ruling in Scotland is great, but it is bitter-sweet for us because we need it filtered to other regions. Vertex wrote to Welsh Government that they will liaise them and send some data through and we are stuck in the middle.
“We are hoping that Wales will do the same. It is an expensive drug but cystic fibrosis is an expensive condition, if you take everything into consideration like the daily regime and cocktail of drugs that sufferers have to take.
“To wear a parent hat, it is difficult to digest the amount of money being charged for the drug, but these companies have put billions into this research so we are stuck in between.
“A move to Scotland is something we have considered. My family are all based in South Wales, so it would be a huge decision to uproot everyone. Something we would have to consider if Wales is not given the drug. It is a huge decision, but it would add 23 years to her life.”
David Ramsden, chief executive of the Cystic Fibrosis Trust, hailed the access agreement in Scotland as a “landmark moment for the hundreds of people with cystic fibrosis and their families across Scotland who have tirelessly campaigned for years to access these drugs”.
He said: “This breakthrough is a victory for their perseverance and enduring hope. It means 350 eligible people living in Scotland will have access to drugs that stabilise their lung health and reduce the need for hospital admissions.
“We celebrate the news in Scotland today, but our campaign must continue to focus on Vertex and all parties in England, Wales and Northern Ireland.
“Those in need of the drugs have already waited too long and we must ensure that thousands more people are not subjected to a postcode lottery. Scotland’s success must now be replicated across the UK without further damaging delay.”
Cystic fibrosis is a debilitating, lifeshortening illness which creates a mucus build-up that causes chronic lung infections and progressive lung damage.
Orkambi is used to treat patients aged two and above, while Symkevi treats those aged 12 and up, and, while the drugs are not a cure for the disease, they can slow the decline in lung function, which is the main cause of death.
A Welsh Government spokesman said: “Our NHS relies on evidencebased appraisals to ensure that its limited resources are used to best effect. Since 2016, we have repeatedly invited Vertex Pharmaceuticals to engage in the All Wales Medicines Strategy Group’s appraisal process.
“Vertex subsequently made a commitment to do so but have not yet honoured that commitment.
“We invite Vertex, once again, to submit their evidence so that the NHS in Wales can make an informed decision about using their medicines.”