News from News.scotman.com August 13, 2007

Parents' dismay over Scots NHS drug ban
LYNDSAY MOSS HEALTH CORRESPONDENT (lmoss@scotsman.com )
FAMILIES of children with a rare disease were devastated yesterday after a drug commonly available in England was rejected for use on the NHS in Scotland.
The Scottish Medicines Consortium (SMC) said Elaprase offered only limited improvements in those with Hunter syndrome. It said it could not recommend the drug for use in Scotland, given its multi-million-pound costs.
But the families of children with this terminal disease expressed dismay, warning they would be forced to move to England to get the drug.
There are only five patients - four of them children - in Scotland with the condition, which causes a characteristic facial appearance and abnormal organ function. Sufferers, mainly boys, have a life expectancy of just ten to 20 years. Elaprase - an enzyme replacement therapy - can help to improve the quality and length of some patients' lives.
An SMC spokesman said: "We were disappointed to not accept this drug for use in Scotland.
"But this drug appears to offer only limited improvements to quality of life for patients, and some of the claims made about it are highly questionable."
The SMC said that treating seven patients over five years - the estimated future demand in Scotland - would cost the NHS £10 million.
"This is by far the most expensive treatment presented to the health service in at least the past five years," the spokesman said.
"We would not have been doing our job if we recommended a drug so overpriced in relation to its benefits."
But the MPS Society, which represents those diagnosed with conditions such as Hunter syndrome, said it was disappointed. The drug is the only one available to treat the illness, which affects about one in 150,000 births in the UK.
Christine Lavery, the chief executive of the MPS Society, said: "This is a bitter blow for these patients, clinicians and ourselves, and in contrast to the situation in England."
South of the Border, the drug is available under a special scheme for "orphan drugs" - those used to treat very rare conditions. The MPS Society said that "virtually all" patients with Hunter syndrome in England had been offered Elaprase.
The family of one young sufferer in the Grampian region, who asked not to be named, expressed its disappointment at the SMC's decision.
"We are proud of our Scottish heritage and devastated that the only hope our son may now have is for us to leave our home, family and friends to live in England, where our son will receive the treatment he needs to save his life," the family said.
Families will now have to pay for the drug or appeal to health boards for funding. If that fails, they can take legal action to try to force the NHS to fund it.
• BOB and Liz McDowall from Glasgow cannot believe the change in their son thanks to the drug Elaprase.
David, 13, who has Hunter syndrome, started taking the drug as part of medical trials over three years ago.
But they could face a legal battle to make sure he continues to receive the drug after its NHS use in Scotland was not recommended.
Mr McDowall said if the NHS refused to pay for the drug, the family would have to move to England to ensure David continued to receive it.
"Since he started taking the drug we have seen an incredible improvement.
"It has probably saved his life," he said.
"Boys who have this condition have stunted growth, but David has sprouted.
It seems to have stopped the disease progressing."
Mr McDowall said it was "dreadful" that other families would now not benefit from Elaprase.

http://news.scotsman.com/health.cfm?id=1280992007

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