Drugs: Ultra-rare Diseases

Debate between David Crausby and Greg Mulholland

Tuesday 16th June 2015 Westminster Hall

[Mr David Crausby (in the Chair)]

The Speaker’s Commission on Digital Democracy recently recommended the use of regular digital public discussion forums to inform debates held in Westminster Hall. A digital debate has taken place on Twitter ahead of today’s debate on access to drugs for ultra-rare diseases. For this reason, Mr Speaker has agreed that for this debate members of the public can use hand-held electronic devices in the Public Gallery. Photos, however, must not be taken. I encourage Members who wish to refer to the Twitter debate to call it the rare diseases Twitter debate, rather than using people’s individual Twitter names.

[Greg Mulholland (Leeds North West) (LD)]

I beg to move,

That this House has considered access to drugs for ultra-rare diseases.

I am delighted to have the chance to speak on this important topic today. I was also delighted to lead and take part in the historic Twitter debate yesterday, which was a great success. On top of the very strong show of support from Members of all parties, the fact that nearly 1 million people took part in the debate yesterday shows how important the issue is.

I got involved in the issue because Katy and Simon, the parents of Sam Brown, a six-year-old boy in my constituency, came to see me. In 2009, when Sam was 16 months old, he was diagnosed with Morquio syndrome, an ultra-rare disease that 88 people in the United Kingdom have. It is a degenerative life-limiting condition with a typical life expectancy of around 25 years. It limits considerably what those suffering from it can do. All of us here can only imagine what it must feel like as a parent to receive the devastating news that your child will deteriorate before your eyes, not live to an old age, and may not even see much, if any, of their adulthood. Imagine how it feels when a nurse rings up and says, “There might be a treatment, but it is only a trial.” Of course, on hearing such news, what parent would not want to sign up for a trial for the drug Vimizim, supplied by the drug company BioMarin? That is exactly what Katy and Simon did: they signed up Sam to the trial without hesitating.

For the past three years, Sam has been doing a 100-mile trip from Otley to Manchester every Thursday to get Vimizim, his enzyme replacement therapy. Without it, Sam would see his growth stunted more than it already is, with further skeletal deformities and possible heart and vision problems. With Vimizim, Sam’s parents, and, even more importantly, Sam’s medical team, say that he is clearly physically more capable and stronger, with more stamina than ever before. To quote Katy, his mother:

“The drug has given him the freedom to be a child again.”

I ask right hon. and hon. Members to take the opportunity to share the single, “There is a Boy”, produced by the Keep Sam Smiling campaign and produced at his primary school, the Whartons in Otley, where they have shown huge support to an ordinary little lad who wants to be an ordinary boy and an ordinary man. The video for the single shows Sam being a fireman, a doctor and an astronaut, the kinds of things that he has the right to hope one day to be, but he can have that hope only if he gets treatment and is able to continue to take Vimizim.

We are here today because, after three and a half years, in just nine days’ time, Sam’s access to Vimizim looks set to be cut off.