NHS England defers decision on Morquio drug by '12-16 weeks'

NHS England has today deferred a decision on Vimizim - the drug required by six-year-old Morquio syndrome patient Sam Brown from Otley - by a further 12-16 weeks.

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Morquio drug extension welcomed by family and MP

A little boy from West Yorkshire with the rare disease Morquio Syndrome will again be given the life-changing drug he needs.

Six-year-old Sam Brown from Otley was receiving Vimizin for free on a pharmaceutical trial. That fiinished in June. His family called on the Prime Minister for help and have been pressing since last year for NHS England and NICE to decide whether to fund the treatment, It's been announced today that Sam's free provision will be reinstated by the drugs company until at least October. His family's MP has been campaigning on their behalf

As well as Greg Mulholland MP, the news has been welcomed by Sam Brown's mother, Katy, though she warns that it is only a temporary solution.

Sam Brown with parents Katy and Simon

"We are incredibly relieved that Sam's treatment will now be reinstated by Biomarin until the end of October when NICE make their funding decision, and we wholeheartedly welcome this positive development. However whilst this provides immediate relief for patients like Sam who were part of the clinical trial, patients who were not still remain without access to Vimizim. The NICE decision is absolutely critical for the entire Morquio community. Today's news is a hugely encouraging step, but a positive funding decision from the NHS is the only viable long term solution."

– Katy Brown, Sam Brown's mothrer


Katy Brown's letter to John Bercow in full

Below is the full letter sent by Katy Brown to the Speaker of the House, John Bercow.

Dear Mr Bercow,

My son Sam is six. He has Morquio Syndrome, which causes shortness in stature, progressive physical disability and early death.

There is no cure.

Sam is a little boy full of fun and life who has no idea about the silent path this disease has set for his future.

For three years he has given his young life to medical research, taking part in a clinical trial for the first ever treatment for Morquio called Vimizim.

You see Morquio Syndrome is one of those conditions with an irritatingly long name.

It’s a good job our MP Greg Mulholland didn’t use its other name – Mucopolysaccharidosis Type IVa.

He might have been stopped much sooner. It is one of the conditions that Mr Mulholland was passionately referring to in a question to the House that you so rudely and abruptly stopped.

When you stopped Mr Mulholland, it demonstrated in an instant everything that is wrong with British politics.

You wielded your power, the House guffawed and jeered, you used derogatory, mocking language.

But here’s the blunt truth behind that stopped question. For eight months Mr Mulholland has fought valiantly to represent my family and my son.

He has stood up against deep injustice and worked tirelessly to get answers and a solution, all whilst health ministers have done nothing.

He was standing up for what is right, unlike many others. Two weeks ago Sam lost access to Vimizim, the drug that has given him back his childhood.

Why? Because of gross incompetence and deep institutional failings at NHS England.

And because of the repeated failures of health ministers to hold it to account.

On Thursday, July 2, NHS England neatly played a get-out-of-jail-free card. They decided after 14 months of deliberating and three changes in decision date to not make a decision on funding the drug at all… and passed the buck to the National Institute for Health and Care Excellence .

Nice, who have no expertise in ultra rare diseases and who are frantically trying to figure out how to deal with these complex conditions that they don’t understand.

This means that even in the event of a positive decision, Sam is likely to remain untreated for a further 6-9 months, with likely irreversible consequences.

And a positive decision is sadly an uphill struggle as Nice has drawn seriously flawed conclusions and not involved the right expert clinicians thus far.

I’ve heard far too many excuses, seen far too many meaningless letters, and been fed far too many broken promises. Not least from David Cameron himself.

It is abhorrent and entirely unacceptable. When you stopped that question on Tuesday, you not only took away Mr Mulholland’s voice, you took away my son Sam’s too.

A little boy with life and the system stacked against him.

You also took away a little piece of me, because despite what I’ve experienced in the past eight months, I’ve always grasped on to the hope that a little humanity remains at the heart of our political system.

I hope you accept now that this indeed was an urgent question, and one that David Cameron and Jeremy Hunt personally should be held to account to properly answer.

Yours, Katy Brown

– Katy Brown

Department of Health: Difficult decisions hard for patients and families

These are incredibly difficult decisions and we know how hard they are for patients and their families. It is right however that clinicians, and not politicians, make these assessments as they are best placed to do so based on the evidence available and their clinical expertise. Across the world health systems are facing growing pressures to fund new treatments arising from medical science. Through the UK's investment in rare disease research, genomics, and NHS England's new models of specialised commissioning, we are leading the way in tackling this global challenge.

– Department of Health spokesperson

NHS England defers decision on Morquio drug by '12-16 weeks'

NHS England has today deferred a decision on Vimizim - the drug required by six-year-old Morquio syndrome patient Sam Brown from Otley - by a further 12-16 weeks.

Adam Fowler reports:

After a wait of more than six months, NHS England it will make its final decision on funding after after NICE, the National Institute for Health and Care Excellence has concluded its report into the drug.

NICE has said that doubts exist about the benefits and cost of this treatment in its recent draft guidance, and is therefore “minded not to recommend” the treatment for patients with Morquio A Syndrome. The manufacturer now has the opportunity to present additional evidence to NICE to address these concerns. If NICE ultimately does recommend the NHS funds this treatment then NHS England will do so. NHS England believes the manufacturer has an ethical duty to continue to support those patients it enrolled in clinical trials of elosulfase alfa whilst the NICE process is still ongoing.

– NHS England

Sam Brown received his last free round of treatment last week.

BioMarin, the company which provides Sam's treatment said it was "disappointed" by NHS England's decision.

BioMarin is disappointed by NHS England’s decision not to provide interim funding for Vimizim. Vimizim is the only enzyme replacement therapy for people with Morquio A syndrome, a rare genetic condition that is progressively degenerative and life limiting. BioMarin continues to work closely with all stakeholders to ensure that a long term funding solution for Vimizim is found for all patients with Morquio A syndrome, so that they are able to access this treatment in England.

– BioMarin spokesperson

Six-year-old Sam's free life changing medication supply ends

It is the moment that one local family have been dreading for years.

We have been covering for some time the case of six year old Sam Brown.

Sam whose family live in Otley, suffers from a debilitating genetic disease. For the last three years he has been using a life changing drug - supplied for free by the firm which makes it.

But today, the free supply came to an end - and the family, who believe it's Sam's only chance to live a long and healthy life, simply don't know if the NHS will pay for him to have the drug in future.

Adam Fowler reports:


Sam Brown's mother outraged after Tory donations appeal

The mother of Sam Brown is outraged after being asked to give money to the Conservative Party - by David Cameron - while she's fighting to get a life changing drug for her son.

Sam, 6, has the rare condition Morquio Syndrome which means his skeleton does not grow properly, but his internal organs do.

His mum Katy is still waiting to find out if NHS England will fund the drug he needs and has appealed to Mr Cameron.

Imagine her surprise when she got an appeal from the Tory leader in a campaign email asking her for money to help future generations.

Mr Cameron says he'll investigate as Sarah Clark reports:

Six-year-old Sam protests over drug funding at Westminster

People with ultra rare genetic diseases have protested at Westminster in their continuing campaign to try and persuade the health service to keep funding a drug which could change their lives.

They include six-year-old Sam Brown, from Otley, who has Morquio Syndrome.

It is a rare condition that means his skeleton does not grow properly, but his internal organs do.

Sam has been receiving a drug called Vimizin that helps him live a normal life.

It is expensive but it had been provided free by the drug company which produces it.

However, that deal is coming to an end.

Jon Hill reports:

Leeds MP blasts NHS England as 'cowards' in fight for drugs

An MP from Leeds says he is "disgusted" at the way NHS England have acted towards families, charities and ministers after senior officials failed to attend a meeting.

Greg Mulholland MP met life sciences minister George Freeman MP yesterday, and took along representatives from several charities and drugs companies.

He said that despite receiving a face-to-face assurance from NHS England official Anthony Prudhoe that he would be present at the meeting, NHS England failed to show up.

Mr Mulholland said he is "disgusted at NHS England cowards for failing to be answerable for their own mess."

He added that during the meeting yesterday, it emerged that NHS England had clearly been misleading the life sciences minister.

The MP has also tabled a parliamentary motion calling on Jeremy Hunt to use his power and intervene.

We have ministers saying the decision is not down to them, but rather NHS England. But NHS England are repeatedly failing to reply to letters or even turn up to meetings despite Anthony Prudhoe telling me they will attend, and ministers are wrongly claim they are unable to intervene when their power to do so is set out clearly in black and white.

I am disgusted at the NHS England cowards for failing to be answerable for their own mess, and for completely letting down the 180 people who desperately need treatment. Ministers absolutely must intervene, and with people's lives at stake, they must do so now.

– Greg Mulholland MP

Mr Mulholland is campaigning for NHS England to announce interim funding for the just 180 people across the UK who are affected by the ultra-rare diseases such as Morquio disease - which affects Mulholland's six year-old constituent Sam Brown from Otley.

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