OTLEY boy Sam Brown's family are urging health chiefs to end the "torture of limbo" being faced by patients with rare diseases.

Katy and Simon Brown were given cause to hope recently when NICE (the National Institute for Health and Care Excellence) announced it was 'leaning towards' approving Vimizim,

The drug is the only treatment available for Morquio syndrome, a life and mobility limiting genetic condition which six year old Sam - who has been on a clinical trial for three years - suffers from.

But The Whartons Primary School pupil and fellow patients are still waiting, after many months of delay, to see if NHS England will fund Vimizim.

And NICE's recent, positive announcement came laced with another blow for families - as the date for a final decision now looks like it has been pushed back again, this time until early 2016.

Katy Brown has slammed the setback as "unexplained and unjustified", and is calling on the health regulator and the Prime Minister to speed things up.

In a letter that has been sent to the Chief Executive and Deputy Chief Executive of NICE, Andrew Dillon and Gillian Leng, she said: "I was very relieved to learn that NICE is now leaning towards a yes verdict on the funding of Vimizim for Morquio.

"Clearly this is not yet a decision, but is a welcome and positive step. I am now for the first time beginning to feel that the substantial body of patient evidence provided is being listened to.

"The significant disappointment in your announcement is the delay in decision date, from October to January.

"I fully accept that due process needs to be followed, but the further three month delay has neither been explained nor justified.

"This may seem trivial to you, but for families it is a cruel twist.

"It prolongs the torture of limbo which is emotionally debilitating, and also physically damaging for those sufferers not on the trial who remain untreated and without access to the drug.

"I urge you please to review these timescales, personally reflect if this truly puts patients' well being at the heart of the decision making process, and bring this matter to conclusion as swiftly as is humanly possible."

A decision in Scotland, meanwhile, which means the NHS there will not fund Vimizim, has been described by the Browns as "devastating" for families north of the border.

Posting on the Keep Sam Smiling Facebook page, they said: "Another example of a poorly thought through, poorly communicated process that has caused significant distress to families and left them with considerable uncertainty."

The Scottish Medicines Consortium said last week that: "While elosulfase alfa (Vimizim) has the potential to improve quality of life in the short term, the case presented by the - drug - company was not robust enough to convince the committee about the longer-term benefits when balanced against its extremely high cost."

Patients in Scotland with rare diseases can, however, ask for their requests to be assessed on a a case by case basis to see if they qualify for the Scottish Government's New Medicines Fund.