Rare Diseases

Lord Dodds of Duncairn Excerpts

Tuesday 17th December 2013 Westminster Hall

[Jim Shannon]

I cannot speak about other places, but I do not believe that that has been the case in Northern Ireland—I am not aware of it. In my area, there are young people with Duchenne muscular dystrophy, and I met some young people with Duchenne at an event held every year in the Methodist centre across the way. I have been to the past couple of events, and there has been only one person from Northern Ireland there. With my background information, I would say that what the hon. Gentleman describes does not happen in Northern Ireland, but he says that it does in England, and that needs to be addressed. I hope that the Minister will take that point on board when responding.

The United Kingdom Primary Immunodeficiency Network, or UKPIN, is a professional organisation for all doctors, nurses and scientists working in this field, and it covers six rare diseases. UKPIN—I need to be careful how I say that, because UKIP has no role in this matter whatever—has stated that its aims are the registration and accreditation of specialist immunology centres for primary immunodeficiency, or PID; the development of a national patient registry, to which the Government have given a commitment in the rare diseases strategy; and the development and dissemination of guidelines and standards for best clinical practice. The UK PID registry has been supported by the Healthcare Quality Improvement Partnership, and has engaged the participation of 95% of UK centres since its establishment in 2008. To date, nearly 3,000 patients have been registered, making it the second largest patient registry in Europe, after the long-established French registry.

The figures for Northern Ireland suggest that PID is as common as cystic fibrosis. A first report on the matter is at the presses, and we will soon have the opportunity to read it. Perhaps the most pressing point that has been raised is that there is a need for meaningful national and international collaboration on both research and development of clinical practice to ensure maximum benefit for patients. Let us not disregard international collaboration: if something good is being done in France or Germany, it would be good to exchange expertise, so that we can all improve and move forward. Groups such as UKPIN can take the lead in some of these matters. The UK has already led in the development of novel treatments—for example, with gene therapy—and in implementing novel technologies such as next-generation sequencing. It is crucial that there is ongoing investment to maintain that research.

Rare Disease UK has welcomed the promise of greater co-ordination of care for people with rare diseases. As we hear all too often, patients and families have nobody to co-ordinate the multiple elements of care and treatment that their condition necessitates. How often have we heard that, when it comes to health care? It is not just about care for rare diseases. Members here today all regularly contribute to our discussions on health issues in the House, and all too often we hear of people who do not know where to go or who to speak to, and are not quite sure who they need to be in touch with to help them. That is a key issue; if we could address that, it would be helpful.

[Mr Nigel Dodds (Belfast North) (DUP)]

I am grateful to my hon. Friend for raising this important issue. He touched on support and services for families, particularly at the early stages of diagnosis of rare diseases. That is especially important when the case involves a child, as many do; we heard that earlier. Does he agree that that is one of the most critical matters that needs to be addressed in any UK strategy, and that it is particularly important in Northern Ireland? He and I have shared some experiences of the deficiencies in that regard.