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.

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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?

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