Mother sues NHS after snub for drug to give girl years more life
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 harms lung health, affects about 10,000 people in Britain.
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) said it is too expensive for the health service to provide.
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.
MPs on the Commons Health and Social Care Committee, leading an inquiry into 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 to make their quality of life so much better.”
Vertex said it had 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 NICE spokesman 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 Department for Health and Social Care spokesman said: “Despite being offered in the region of £500million 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.”