For more than 1,000 days a fight has gone on to access a ground-breaking drug which could make a significant difference to the long-term health of a Brackley couple’s daughter.
Paul and Catherine Upstone’s 14-year-old daughter Cerys has Cystic Fibrosis, a condition which causes a build-up of thick sticky mucus in the lungs, digestive system and other organs, which will require a lifetime of medications and physiotherapy to manage.
Cerys said: “I have to take antibiotics for symptoms and stuff like that.”
Catherine added: “She has two sessions of physiotherapy every single day and about four nebulisers a day – and last year she spent a total of six weeks in hospital, four of those in Great Ormond Street Hospital.”
The impact of the treatment and the infections related to CF are enormous with Cerys routinely having to catch up with school work during holidays.
Three years ago there was a breakthrough in the treatment of CF when the drug Orkambi was approved. The drug is licensed in the UK and available in many countries around the world.
Known as a precision drug, Orkambi has been shown to improve a patient’s health by targeting the underlying cause of CF and not the symptoms.
The joy, however at the possibility of an improved treatment regime for Cerys quickly grew into frustration as the manufacturer, the NHS and the National Institute for Health and Care Excellence (NICE) became embroiled in a dispute preventing its access.
Catherine said: “One of the hardest parts is knowing that so many other people have access, even our neighbours in Ireland have access to Orkambi.
“We can’t get it on the NHS. There has been a continuing circle of people blaming each other.”
The major sticking point is a financial one that still has not been resolved despite negotiations in June this year.
Catherine said: “NICE said that Orkambi is an effective medicine but not cost-effective at this time.
“That was three years ago. NHS England and Vertex have been in talks this year regarding a deal.
“Vertex say they offered their lowest price ever and NHS England came back with their final offer which was a £5 million investment into precision drugs for Cystic Fibrosis but it doesn’t come close to what Vertex are asking for.
She added: “Our plea as a family is to get everyone around a table talking so an agreement can be made and for our Health Minister to get involved. Currently Matt Hancock has ignored the CF community.”
During their fight the Upstone family have explored many avenues to highlight the need for Orkambi for thousands of sufferers across the country. Paul, Catherine and Cerys have taken to social media community groups, online forums, petitions and national and local media but perhaps nothing has been more powerful than an old fashioned letter penned by Cerys to the pharmaceutical giants Vertex.
In it she writes: “So much time has passed since the drug was approved and still nothing has happened.
“CF is very unpredictable and I fight infection after infection and everything that comes with this relentless disease.
“The situation is even more worrying, as with being a teenager I know my health could deteriorate even further and with CF being so unpredictable it could happen at any time.”
She describes further the physical and psychological toll the disease takes on a young person’s body, ending the letter with a heart felt plea.
She concludes: “Please Vertex help me. Help me reach my potential and help me maintain my health for as long as I possibly can.”
The family have also been working with their MP Andrea Leadsom who Catherine described thus: ‘She’s become my best friend’.
The MP said in a statement to the BG: “In February I attended a round table meeting on Orkambi and Cystic Fibrosis and following on from this, I also organised a meeting with some constituents, Vertex and the Cystic Fibrosis Trust where we discussed access to Orkambi for NHS England patients.
“I am committed to finding a solution that will enable rapid access to this pioneering drug and I am proud to say I have signed a cross-party letter to Vertex Pharmaceuticals calling for an urgent resolution.
“Although Vertex and the NHS are finding it difficult to overcome the various hurdles, I will continue to do all I can to support my constituents.”