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Rare Diseases

Volume 604: debated on Tuesday 5 January 2016

A rare disease is a life-threatening or chronically debilitating disease that affects five people or fewer in 10,000. Research shows that one in 17 people will suffer from a rare disease at some point. In the UK, that equates to approximately 3.5 million people.

Same But Different, which is based in my constituency, is concerned about a number of the challenges faced by people with rare diseases. One key issue that it has raised with me is the level of support available at the time of diagnosis, particularly for parents of children with rare diseases. Will the Minister examine how we can signpost better help and support to those who have been diagnosed?

I am glad the right hon. Gentleman mentions that point; the House may not be aware that we recently added four new rare diseases to the newborn heel-prick test, which has helped to detect more than 1,400 children with a rare disease. I am disappointed to hear that he feels that some parents had issues with follow-up, and of course we will look into that, but I think he will find that the UK rare diseases strategy, which was published in 2013 and contains 51 commitments from government, covers that. The first report back on that strategy will take place this spring and it is being done by the UK Rare Disease Forum. I am happy to speak to him afterwards about whether the excellent organisation he names is part of that.

One such rare disease is Duchenne muscular dystrophy. I am sure the Minister is aware that we are awaiting what we hope will be a positive decision from NHS England on a drug called Translarna, which could help boys with the disease. We were due to have that announcement yesterday. Does she have any further and better particulars on that? Will she update us on when we can expect an announcement, which we hope will be a positive one?

I know that my colleague, the Under-Secretary of State for Life Sciences, is working very hard on that matter, and is hoping to make an announcement soon. I am sure that, at that point, he will be able to update my right hon. Friend.

With regard to ultra-rare diseases, I will be joining the family of seven-year-old Sam Brown on 23 January to celebrate the funding of Vimizim. I thank all those involved in that decision, including those in the Department. As well as an update on Translarna, can we also have an update on the possibility of funding another drug that we have been campaigning for, which is Everolimus for tuberous sclerosis?

I thank the hon. Gentleman for his words, as does my hon. Friend the Under-Secretary of State for Life Sciences. With regard to the matter that he just mentioned, I know that it is something that NHS England is reviewing and it will come forward with a view in due course.

Will my hon. Friend join me in praising the work of the Institute of Translational Medicine at the University of Birmingham Medical School? It is doing outstanding, world-wide standard work in developing cures and treatments for such rare diseases, and indeed for more common diseases such as cancer.

I absolutely join my hon. Friend in that and agree with his very well-deserved words of congratulation. I know that the Under-Secretary of State for Life Sciences has visited the institute and is—as everyone is—hugely impressed with it. I also join my hon. Friend the Member for Lichfield (Michael Fabricant) and others in congratulating Charlie Craddock on his CBE in the new year honours list.

Patients living with rare cancers often have fewer treatments available to them. Often, the only option is to use off-label treatments. The cancer drugs fund has helped patients gain access to those treatments, but, despite a Conservative party manifesto commitment to continue investing in it, the fund is now under threat because of central Government cuts. What assurances will the Minister provide to people living with rare cancers that off-label drugs will still be funded? Will she apologise for the uncertainty that those cuts are causing to the thousands of people who are affected by cancer in England?

I certainly do not recognise the shadow Minister’s characterisation of the cancer drugs fund. Some £1 billion has been committed to it and it is being reviewed. The fund was introduced by the previous Government, and we are very proud of it. It has made a big difference to the lives of more than 80,000 patients. More widely, the recent cancer taskforce published its report, “Achieving world-class cancer outcomes”, and it made many recommendations, which are particularly relevant to rarer cancers and blood cancers, many of which focus on improving access to diagnostic testing.

Of the 7% of the population that will suffer at some point in their life from a rare disease, 75% are children. Unfortunately, 30% of those will not reach their fifth birthday. What more can be done for Great Ormond Street hospital and for Birmingham children’s hospital, which do such excellent work?

My hon. Friend is quite right to highlight the number of people who will be affected by such diseases. There are between 6,000 and 8,000 rare diseases. Among the things that the Government are doing that will make a really big difference to some of the institutions that he mentioned and others, and particularly to sufferers, is the 100,000 genomes project, in which the Government have invested. The creation of a network of genetic medicine centres will underpin that further development of genetic testing services. As a very large proportion of rare diseases are genetically based, we want to make significant progress with that genomic work.