Rare Diseases

Charles Hendry Excerpts

Tuesday 17th December 2013 Westminster Hall

[Charles Hendry (Wealden) (Con)]

I am delighted to have the chance to speak in this debate, Mr Bayley, and I congratulate the hon. Member for Strangford (Jim Shannon) on securing it. I have been in the House for more than 20 years in total, and I cannot remember having a debate on rare diseases in that time. This is an important subject that we need to address in a constructive way, as the hon. Gentleman has done.

Looking at figures before coming into the Chamber, I saw that there were about 6,000 rare diseases, which is a shockingly high figure. I do not think that many people realise just how many different types of diseases there are. As a parent, one has only to watch a programme such as “Children in Need” to see how many diseases are out there, to see illnesses that we have never even heard of, but that are having such a devastating effect on families and lives, and to see how much work still needs to be done in the area. If the debate helps to take that forward, that is all to the good.

I want to focus on two aspects: first, the families and voluntary groups that work in this space and secondly, some of the academic research that is being done into rare diseases. So much of the work in this area is done by families who have been affected by a child or a family member who has been unwell. The work that they then do to raise funds, either to support other families or to carry out research, is an incredibly important part of the equation.

I want to refer to two examples. The first, Charlie’s Challenge, was named after Charlie Boutwood, a constituent of mine, who was 20 months old when he was found to have a brain tumour. He survived thanks to incredible medical attention, but his parents set up the Charlie’s Challenge charity to put money into brain tumour research, particularly relating to children. Although brain tumours are the biggest single killer of all the cancers of children and young people under 40, less than 1% of the research into cancers is into brain tumours and particularly the effect on young people. The work of Charlie’s Challenge is to see how it can provide additional support and research funding into such an important area. The second is Tildy’s Trust, which was named after Matilda Curran, a young teenager—the daughter of a very close friend—who was found to have leukaemia and who died of it two years after it was discovered. Her parents set up Tildy’s Trust in order to provide support, research and funding for families who are going through similar circumstances.

Those are just two examples of a large number of such organisations, which are driven by incredible commitment and passion by families who have been affected, and to whom we should pay tribute, because of the immense contribution that they make. Sometimes I feel that more could be done by Government to help to join up the work of those many different organisations to try to provide a greater central force to take forward that work and to help to co-ordinate the valuable work that they do.

[Alison Seabeck (Plymouth, Moor View) (Lab)]

The hon. Gentleman is making an important and quite sensitive point, because a number of these trusts are set up on the back of and because of people’s individual experiences and losses. It is difficult sometimes to get them to talk to each other, because they are so very personal to people.

[Charles Hendry]

I think that is absolutely true and there is a role that the Department can perhaps play in bringing people together, when they have similar goals, and if they can combine their force and strength, the overall gain may be bigger than the sum of the parts. Perhaps that is something to which the Minister could respond when he replies to the debate.

My second point relates to the university work that is being done in this area. When I left the Government in September last year, one thing that gave me the most joy was to be invited by the university of Edinburgh to become a visiting professor. I have been incredibly impressed by the work that is being done in this area—not in the part in which I am involved, which is the business school—particularly by the medical faculty. Work is being done to look comprehensively at the patient experience and at how to bring together all the issues that affect patients who often are being affected by rare diseases and how they can combine the necessary research into that as well.

The issue is not only about the role of world-class research, which I think Edinburgh has in abundance, but about how that is tied in with the role of benefactors, because the work of the university of Edinburgh has been made possible by a small number of extraordinarily important contributions. The Euan MacDonald centre for motor neurone disease research is funded by an immense donation by the family of Euan MacDonald, but that in turn led to an even bigger donation by J. K. Rowling for the Anne Rowling regenerative neurology clinic. It is through the work of the university—of examining how it can bring together its extraordinary genius in looking at the challenges that have been presented by these rare diseases—and tying that in to extraordinarily generous benefactors that offers us a really great way to address some of the issues. The work being done in Edinburgh is of course being done in many other universities in this country as well, but if we can help, through the Government, to try and co-ordinate that better, we will see real progress.

I remember hearing former President Jimmy Carter talking about what it meant to him, in his lifetime, to have played a leading role in the eradication of a single disease—river blindness. What an extraordinary, unbelievable ambition, and it is a magnificent thing to have been achieved, just as Bill Gates is achieving similar things in different sectors. The more that we can co-ordinate that work, expertise and genius in our universities with the good will of so many people outside it, the more, as a succession of Governments, we will be able to say that what we have done has led to the eradication of some of these diseases.

[Graham Stringer]

The hon. Gentleman is making very serious points about the excellent research that happens in the university of Edinburgh and in many of our other medical schools up and down the country, but he has not referred to a problem, which is that an excellent drug, or one that is likely to be excellent, can be developed, but because of the nature of rare diseases—that very few people suffer from them—it is very difficult to get it through the trials process, because that is designed to see whether the drug works on large numbers of people, quite sensibly. Does he agree with me that NICE should have different rules for drugs related to rare diseases?

[Charles Hendry]

I agree with the hon. Gentleman. A constituent has written to me just this week about meningitis and a drug that has gone through the trials process. It has been approved, essentially, but is only being made available to people who can pay for it at the moment. It seems to me that when treatments could be made available, expediting the process of approval and then ensuring that they are generally available as soon as possible, to as many people as possible, should be an objective of its own. I absolutely agree with what he said in that respect.

[Jim Shannon]

As I said in my introduction, the Teenage Cancer Trust indicated that that was one of its recommendations and it would like to see that happening. Perhaps the Minister can respond to that recommendation from the Teenage Cancer Trust. When it comes to the trial, effectiveness and availability of the drugs, we have to look outside the box. That is what we are trying to do, and I think that the hon. Gentleman is also saying that.

[Charles Hendry]

That is exactly what I am saying. The other thing that we owe tremendous credit to the Teenager Cancer Trust for is highlighting the way in which teenagers are dealt with in hospitals. The issue is that sometimes they are put in children’s wards, which is clearly not suitable for somebody who is in their late teens, and at other times, they are put in wards with people who were in their 80s and 90s, who are dying or are ill with other conditions. Someone who is a teenager is at a particularly vulnerable time of transition in their life, and it is incredibly important that their needs are dealt with in a specialist way and that they are surrounded by as many people of their own age group as possible. I think that is an important part of the treatment and recovery process.

I encourage the Government to continue what they are doing to try and address the issues. The strategy that is being put in place is of great importance, but more could be done to achieve a bigger overall gain from so many different fragmented, but immensely worthwhile contributions.