Eric Avebury writes: Rare disease Day – a new report on care co-ordinators

rare-disease-day-logoTomorrow is International Rare Diseases Day, and I shall be asking in the Lords what contribution the Government are providing for the International Rare Diseases Research Consortium.

The EU defines a rare disease as one that affects no more than 5 in every 10,000 persons. But since between 6,000 and 8,000 such diseases have been identified, about 3.5  million patients are affected in the UK. Three quarters of these diseases can affect children, and nearly a third of the youngest patients die before their fifth birthday.

The Department of Health launched a draft plan for rare diseases a year ago and undertook a consultation on it leading to the definitive plan, scheduled for the  second half of this year.

Rare Disease UK, the National Alliance for people with rare diseases and their supporters, launched a report today on the value of care coordinators to both patients and the NHS, a win-win scheme that benefits patients and saves money for the NHS.

The UK participates in rare disease research at the European level, a programme costing  €38 million over the next six years. The theme of tomorrow is ‘Rare Disorders without Borders’, highlighting the importance of sharing medical research and practice on diseases which may only affect a few people in the UK.

Yesterday in the European Parliament two Alliance of Liberals and Democrats MEPs were hosting a stakeholder meeting to discuss revision of the Transparency Directive on medicinal products, that would improve the pricing transparency of medicinal products and thus help to lower the cost of treatment of rare diseases.

This year also marks the 30th anniversary of the US Orphan Drugs Act, which gave special marketing protection to the developers of drugs for rare diseases. This side of the Atlantic, the European Medicines Agency provides incentives to SMEs for the development of orphan medicines. An article in the December issue of the magazine Health argued that the current orphan drugs régime led to higher prices and there seems to be a case for it to be reviewed at EU level.

Here, the Prime Minister launched the report Strategy for Life Sciences: One Year On in December, announcing a new focus on genomics, a field which is set to change the way disease is diagnosed, prevented and treated. The NHS is earmarking £100 million towards sequencing the genomes of 100,000 patients with cancers and rare diseases.

The UK is already a leading player in life sciences, with a pharmaceutical industry worth £30 billion a year; medical and biotechnology sectors turning over £20 billion a year, and a similar huge potential for innovation in our universities and the NHS. The combination has made this country an attractive destination for overseas investors, to the tune of £1 billion in 2012.

As an example of how the life sciences strategy will enhance our capability on rare diseases,  the UK Research Partnership Investment Fund is putting £10 million into an £85 million partnership between University College London and the Great Ormond Street Hospital, for a Centre for Children’s Rare Diseas Research.

So in spite of the economic problems we face, there is a good story to tell on how  we are tackling rare diseases with more resources and greater collaboration, both domestically and internationally.

* Eric Lubbock, Lord Avebury, is a working peer, and Vice-Chair, Parliamentary Human Rights Group. He blogs here.

Read more by or more about or .
This entry was posted in News.
Bookmark the web address for this page or use the short url http://ldv.org.uk/33273 for Twitter and emails.

2 Comments

  • Richard Dean 27th Feb '13 - 4:30pm

    5 in 10,000 people is a lot really, it’s 30,000 in a population of 60 million, and seems obviously something we should be doing something about.

    But if 3.5 million people are affected in the UK, and three quarters are children, and a third die before their 5th birthday, that’s a HUGE problem. Are these statistics really correct? Can some clarification be provided please?

Post a Comment

Lib Dem Voice welcomes comments from everyone but we ask you to be polite, to be on topic and to be who you say you are. You can read our comments policy in full here. Please respect it and all readers of the site.

If you are a member of the party, you can have the Lib Dem Logo appear next to your comments to show this. You must be registered for our forum and can then login on this public site with the same username and password.

Your email is never published. Required fields are marked *

*
*
Please complete the name of this site, Liberal Democrat ...?




Recent Comments

  • User AvatarEddie Sammon 22nd Oct - 2:25am
    It's a good article, but the banks are whooping most areas in society when it comes to promoting immigration and race equality, so there was...
  • User AvatarRichard Dean 22nd Oct - 2:10am
    I agree too. The polarization that is evident in Nick's choices, and reactively (I suppose) in this article's headline, does no electoral good whatsoever.
  • User AvatarRichard Dean 22nd Oct - 2:03am
    I wonder if Peter Hayes's experience tells more about LibDems that immigrants. It seems like he's saying that they're ok if they serve you well,...
  • User AvatarSue Doughty 22nd Oct - 1:29am
    Tony Greaves is right about the democratic deficit (and yes, it is totally unfair to attack him because he is in the Lords - rather...
  • User AvatarSteve Way 22nd Oct - 12:39am
    @Psi Not sure where my comment says that it shouldn't be done? It should have been done earlier, that would have been the right thing....
  • User AvatarRoland 22nd Oct - 12:38am
    "I had the misfortune of staying in a Travel Lodge, breakfast was in a greasy spoon ‘restaurant’ staff were English and did not care, compare...