Children with the rare, life-shortening disease Morquio syndrome will not get a new drug that their families and friends have been campaigning for on a compassionate basis because NHS England has decided to wait for the deliberations of the drug watchdog, the National Institute of Health and Care Excellence (Nice).
Their case has been brought up in the House of Commons by MPs including the LIberal Democrat, Greg Mulholland. There are around 35 children who, until a week ago, were supplied with the drug Vimizim by the manufacturer on a compassionate basis because they had been part of a clinical trial. But NHS England has declined to find the cost of the drug – around £400,000 per year, per patient – in advance of a full cost-effectiveness analysis by Nice.
Nice has already said it is minded to turn down the drug because the benefit is not sufficient for the price and it has asked the manufacturers to justify the cost or offer a discount. A decision from Nice is expected in 12to 16 weeks’ time.
NHS England’s decision will not be welcomed by campaigners. A second drug – Translarna (ataluren) for Duchenne muscular dystrophy – also awaits a ruling from Nice. Translarna (ataluren) also has a high price tag – in the region of £170,000 a year – and some uncertainty around benefit.
NHS England could pay for the drugs through critically urgent funding if it considered patients would suffer severe deterioration within three to four months without it, but neither drug is thought to have met that criteria.
NHS England is announcing a raft of decisions on specialised commissioning. These decisions are taken centrally, usually because of the small numbers of patients affected, and most are favourable, although it has announced it will cease to fund several treatments no longer considered cost effective. It will no longer pay for Eculizumab and Bortezomib for the prevention of organ rejection after a kidney transplant. Nor will it fund Sapropterin to treat the rare genetic condition phenylketonuria (PKU) in children.
James Palmer, NHS England’s clinical director for specialised commissioning, said: “These are really difficult decisions, which is why we rely on Nice wherever possible, and make use of the best available evidence as well as extensive engagement with clinical experts, patient representatives and the public. The new policies we have been able to approve will help us to improve and extend thousands of lives for years to come through prevention, identification and treatment.”
