Drugs: Ultra-rare Diseases

David Crausby Excerpts

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.

--- Later in debate ---

[Several hon. Members]

rose—

[Mr David Crausby (in the Chair)]

Order. I intend to call the three Front Benchers at 3.30 pm. Three Members are standing, so if they can keep their contributions to less than four minutes, they will all get in.

--- Later in debate ---

[Andrea Jenkyns (Morley and Outwood) (Con)]

I thank and commend the hon. Member for Leeds North West (Greg Mulholland) for his work.

I find it heartbreaking that we have today heard about many constituents who have had access to drugs that have given them hope and improved their lives but which have then been taken away. Like the hon. Member for Leeds North West, I have a constituent who suffers from Morquio, Angela Paton. She is 35 years old; it has taken 35 years to find a drug that works, and it is now being taken away from her. Matthew Firth is another constituent, a young man with special needs who can no longer get a basic cream that he needs.

The case of Abi Longfellow is much in the news at the moment. She is a 12-year-old girl with a rare form of dense deposit disease. She needs a kidney transplant to live. Her father has been prepared in the past 12 months to give her his kidney. He should have had the operation on Friday, but for that to go ahead she needs the drug eculizumab. NHS England and NICE say that the drug will not work in Abi’s case—she has a very rare form of DDD—but there is research from the US, Canada and Italy indicating that the drug does work.

I thank the Prime Minister for intervening and asking NHS England to examine the case, and I ask my hon. Friend the Minister to ensure that that happens. There is research available; I find it difficult to understand whether, when the likes of NHS England and NICE say the drug will not work, that is just a tick-box exercise, or whether they look at research from outside this country. It is important to consider that. I would like a joined-up approach between NICE, NHS England and the Department of Health. I ask the Minister to consider the matter comprehensively, and to ensure that NHS England and NICE look into it.

[Mr David Crausby (in the Chair)]

I will now call the Front-Bench Members; there may be a vote, in which case I shall suspend the sitting for 15 minutes. The new rules allow Mr Mulholland to wind up the debate briefly, if there is time, but for that to happen, the Minister must be allowed enough time.