Mother takes legal action against NHS for access to Orkambi drug
The mother of a girl with cystic fibrosis is taking legal action against the NHS after it refused funding for a drug which could extend her daughter’s life.
Sarah Burgwin, of Totnes, Devon, said it was “shameful” that six-year-old Katie Stafford (both pictured) was being denied access to Orkambi.
The drug’s high price tag of £104,000 per year for one patient means it is only available in exceptional circumstances.
Despite its recommendation by a consultant, NHS England have told Katie’s mother her daughter does not warrant the treatment.
“They are putting a price on the life of my daughter. It’s shameful,” said Sarah. “What gives them the right to play God with my child’s life?
“Katie is a helpless child who did not choose to have this condition. She did nothing to get it apart from to have the misfortune of being born with it.
“It is heartbreaking to see Katie suffer when I know there is a drug out there that could help prevent her torment.”
Sarah has instructed law firm Hodge Jones & Allen to seek a judicial review of NHS England’s decision.
Partner Peter Todd said: “Thousands of parents have been left in a desperate position of watching their children deteriorate with this life-shortening condition, while knowing that there is a drug out there that can help improve their health and extend their lives.”
Lawyers believe Katie is an exceptional case because her learning and behavioural difficulties prevent her being administered alternative treatments.
Cystic fibrosis, a life-limiting condition which affects lung health, affects around 10,000 people in the UK.
Clinical trials have shown the drug can improve lung function in sufferers of the disease.
But the National Institute for Health and Care Excellence (Nice), which decides which treatments should be available on the NHS in England and Wales, says it is too expensive for the health service to provide.
The drug is already available in several European countries after being approved by the European Medicines Agency in 2015.
NHS bosses have been engaged in years of negotiations with Orkambi’s manufacturer Vertex Pharmaceuticals to secure an affordable deal.
This is despite the Government’s claims that the company was offered the “largest ever” financial commitment in the history of the NHS.
Earlier this year, health ministers wrote to the company urging it to drop Orkambi’s price, saying “time was of the essence” for cystic fibrosis patients.
A petition signed by more than 100,000 people also triggered a debate on the issue in Parliament this year.
MPs on the Commons Health and Social Care Committee leading an inquiry into the patients’ access to drugs, have threatened to publish the details of negotiations between Vertex, Nice and NHS England if a deal is not reached by November 30.
Sarah said: “I hope that we can win this legal battle, not just for Katie, but also so this can be a gateway for other children also getting this drug to make their quality of life so much better.”
In a statement Vertex said it held a “productive meeting” with Nice on October 4 and that it was “committed to continuing their discussions over the appraisal of treatments for cystic fibrosis”.
A spokesman for Nice said its guidance on Orkambi would be reviewed when it received new data or a revised price for the drug.
“We are glad Vertex has agreed to re-engage with Nice and NHS England,” he added.
A spokesman for the Department for Health and Social Care said: “Despite being offered in the region of £500m over five years, the largest ever commitment of its kind in the 70-year history of the NHS, Vertex has refused to accept, putting Orkambi out of reach of patients.
“We urge Vertex to accept the offer.”
A NHS England spokeswoman said: “Orkambi for the treatment of cystic fibrosis is not recommended by NICE and therefore is not routinely commissioned.
“Individual funding requests for treatments that are not routinely commissioned are difficult decisions, which is why they are taken by experienced teams on the basis of clinical evidence.”
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