What Vimizim means to Sam


What Does Vimizim Mean to Sam?

The following piece was written by Katy Brown in response to the nonsensical political and monetary concerns surrounding the funding of Vimizim, and brings to light the significance of this drug to the children and adults suffering from MPSIVA.

“So we have now welcomed in 2015. A brand new year full of hope and celebration. The New Year is always a good time for reflection I find, but I really had never anticipated that this year this for us reflection would include the ethics and workings of our National Health Service.

For the latter part of 2014 we have been fighting to keep our son Sam smiling. More precisely we have been fighting for the drug that he had been trialling for almost 3 years to be funded by the NHS. You see Sam has something called an ultra-rare disease. That’s not just rare by the way, but ultra-rare, which means that there are less than 500 sufferers in the country. Actually there are just 78 sufferers of Morquio Syndrome in England. Just 78. I think the definition of a rare disease stretches to 20,000 sufferers. Big difference! How a disease with 20,000 sufferers is funded, researched, prioritised, understood is very very different to one where there are just 78 people affected. A cluster of houses with 78 people living there probably wouldn’t even be called a village! 20,000 is a market town.

Let me paint you a picture of Morquio. An adult the same size as a 3 year old. Organs squashed into a very small space. Severe physical disability. Hearing problems, sight problems, heart problems, lung problems. Life expectancy on average of 25. No treatment. Until now.

Sam is 6. He hates being the smallest in his school and wishes he could run around and play football with his friends. A 12 hour round trip to Royal Manchester Children’s hospital every Thursday exhausts him. He has bruises on his wrists and elbows from cannula after cannula. He’s had major surgery, minor surgery and more MRIs than you can shake a stick at. But every day he smiles. He embraces life. He is a bright, articulate and engaging boy who is capable of achieving anything he sets his mind to. Except of course he has this Morquio thing which may well stop him dead in his tracks. Literally.

So after decades of research and development, years of clinical trial (all for which has led to significant investment in the NHS by the pharmaceutical company), families affected by Morquio should be raising a glass to 2015 as the monumental year when the first ever treatment for Morquio becomes available for sufferers on the NHS. Vimizim. This should be our year!

Yet instead of a happy heart, we enter 2015 with an air of fear and dread. I have learnt lots about the complexities of treatment for rare diseases over recent months, and the many ethical, social, political and economic pressures that have led to us now finding ourselves in a very uncomfortable and uncertain limbo. I’m naturally a very empathetic person. I’m also commercially astute. I’m a good reader of situations and of people. I can naturally see all sides of a story and draw my own conclusions. But you’re Sam’s mum, I hear you say, so you are naturally biased! Well, you got me there, of course I am! But as a mum I am also passionate to the end, and I will fight for what I believe is right. I also know my son and the impact that this treatment is having on him better than anyone paid to make a funding decision.
So, for what it it worth, I’d like to share my perspective on the many reasons I have heard sitting behind the reluctance of the NHS to fund Vimizim …. and why I believe with every fibre of my being that have to continue to fight for what Sam and all other Morquio sufferers truly deserve….

1. “It is too expensive”
What does that mean exactly? The reason Vimizim and other drugs for rare diseases are expensive is quite simply because the disease is rare – development is costly and risky and there is only a very small pool of people to sell the drug to and recover costs and make a profit. Did Sam choose to suffer from a rare disease? No. Should he suffer as a result just because the disease is rare? No.
I am not entering into a debate on what costs for drugs for rare diseases should be … that is the responsibility of others to determine. But using innocent children as a scapegoat for a difficult commercial conundrum is not acceptable.
In November 2014 the scorecard used by NHS England to appraise the funding of new drugs was legally proven to be discriminatory to ultra-rare diseases because of this very reason. And for the record NHS England did not choose to “pause” to consider how they move forward out of free will, had there been no legal challenge, a clearly discriminatory process would have been enacted, funding for Vimizim would have been declined, and the budget deficit for Specialised Services would have been steadied. Job done, some might say.

2. “It is not a cure”
No, it isn’t. But it will prevent & reduce costly, acute medical interventions.
And it is a first step in medical technology that might lead to a cure in the future.
And there are no horrendous side effects. It is win win.
I’ve heard politicians talk about “marginal benefits of high cost treatments”. My counsel would be – be very careful with glib generalisations about diseases you don’t properly understand. Vimizim is changing Sam’s life. This is clinically proven, but I don’t need clinical evidence. I can see it every day, we would not continue to put Sam through the considerable upheaval and distress that treatment entails were it not having a dramatic effect. Be very careful about how you put a value on that.

3. “The NHS is under unprecedented pressure with an ageing population and increasing numbers of treatments and therapies”
True – yes. Interesting – yes. Relevant? No.
Is this Sam’s fault? Is NHS England holding an innocent child responsible for demographic, political, economic and social factors spreading the breadth of the United Kingdom? Does the buck stop with Sam? Is Vimizim the straw that broke the camel’s back?
Without being rude, this is not Sam’s problem to solve.

4. “We have to make choices”
I agree. But here are a few potentially controversial observations:
– It is politically easier to say no to something new than it is to stop doing or changing an existing practice.
– It is politically easier to say no to ultra-rare diseases because you are upsetting and impacting fewer people, and representing organisations have less power, clout and resource to kick up a fuss. It’s quite handy that the general public for the most part have never heard of the condition, less bad publicity.
– The NHS have to make choices, true, but Sam has none. Again I am not making a judgement here but simply a factual observation. The NHS funds without question treatment and procedures to help people who have made certain lifestyle choices. Indeed it invests in prevention, education and a multitude of support services – rightly. Sam has never, ever had the luxury of making a choice. Yet his treatment is being questioned. How can that be right?

5. “Following the Health & Social Care Act 2012, these decisions are for NHS England as an independent body to make and not for politicians to influence”
I’m sorry, I don’t believe this is true, nor can it ever work. The government imposes budgetary constraints, it sets policy priorities. It is influencing decisions in reality every single day.

NHS England spent almost 18 months designing an appraisal process for specialised commissioning which was in November 2014 challenged and proven to not be fit for purpose and legally discriminatory. NHS England failed to replace a process that worked with a process that works! Goodness only knows how much taxpayers money has been wasted.. Forgive me for not trusting or believing that a replacement and legally robust process can now be put in place before April 2015. In the meantime families are left hanging, travelling huge distances if they are fortunate to have been part of a clinical trial, not knowing whether access to the drug will continue …. Because this is only available compassionately from the drug company who have no legal obligation to continue to provide it. Families who haven’t had access to the trial drug are meanwhile seeing their condition deteriorate, knowing there is a drug out there but that they can’t have it. The time bomb is ticking.
When exactly should politicians step in?

A 6 year old boy has his future mapped out by a cruel and debilitating disease. He’s unlikely to see his 25th birthday. But amazingly some clever scientists have developed a treatment and doctors have proved it works! It gives Sam the chance of a longer, fuller, independent life, giving back to society, changing the diagnosed trajectory. He’s also fortunate to live in a nation with a world renowned system priding itself on the principle of universal health care.
How can the decision be anything but a yes?”

Follow and like the Brown family’s fight for Vimizim on Facebook and Twitter (@KeepSamSmiling).