Setback for ill Otley boy's drug fund plea

Sam Brown suffers from the Morquio syndrome which affects the way his skeleton grows

A six-year-old boy with an incurable medical condition has suffered a setback after the drugs watchdog said it was not minded to recommend a drug that could help with his illness.

Sam Brown, from Otley, has Morquio syndrome, which affects the way his skeleton grows.

He has been taking Vimizim to treat it.

But the National Institute for Health and Clinical Excellence (NICE) says the drug is expensive and its clinical benefits have been "overestimated".

Publishing it's preliminary findings on the drug, also known as elosulfase alfa, NICE said that despite the drug being "likely to provide valuable clinical benefits for certain aspects of the condition" it would not "entirely stop disease progression".

It said the estimated cost to provide the drug per person per year was £395,000.

'Decision may change'

Katie Brown, Sam's mother, said the findings were a "massive blow" to her son who currently receives the drug on a compassionate basis.

She said: "He's been receiving the drug now for over three years and since that time he's grown, he's stronger and he's more physically capable than he ever has been.

"The reason these drugs are expensive is because this disease is so rare and that's not his fault, he never chose to be born with a rare disease."

Professor Carole Longson, NICE Health Technology Evaluation Centre director, said: "Because of this cost and the fact that the clinical benefits had been overestimated in the company's economic model, the committee expressed concerns about the value for money provided by elosulfase alfa treatment."

She said the committee has asked drug manufacturer BioMarin to provide more information before it makes a final decision, adding that the decision "may change after consultation".

NHS England is expected to make a decision on whether to fund the drug later this month.

A spokeswoman for BioMarin said it was aware of the findings and was "confident" it could address all of the committee's concerns.