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

"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."

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

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.

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.

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.

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.