Mum takes action to force NHS into funding life-saving drug

THE mother of a Totnes girl with cystic fibrosis is taking legal action against the NHS after it refused funding for a drug which could keep her six-year-old daughter alive.

A consultant has recommended Katie be given the drug, but the NHS says it is too expensive. Sarah Burgwin says it is “shameful” six-year-old Katie Stafford is being denied access to the drug 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 has told Sarah 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 an alternative treatments.

Cystic fibrosis, a life-limiting condition which attacks the lungs, 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.

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.

Katie Stafford