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Dr Sarah Wollaston MP, Chairman, Health Select Committee


Press Release Major new study launched by 2020health aims to shine a light on NHS attitudes to rare diseases.

We cannot abandon people with rare diseases, says leading health think-tank.

2020health Press Release

Press release: Tuesday 28th February - For immediate release


Major new study launched by 2020health aims to shine a light on NHS attitudes to rare diseases.


February 29th is International Rare Disease Day, and the leading health think-tank, 2020health, marks this day by launching a major new research study on the availability of medicines for those with rare diseases.

It is estimated that there are currently between 5000 and 7000 rare or very rare diseases.  Orphan diseases are defined as those with prevalence in the population of less than 5 in 10,000, according to EU policy.  Both the United States and the European Union have implemented legislation to incentivise the pharmaceutical industry to develop orphan drug treatments which have a lower potential return on investment as a consequence of small patient pools. According to 2020health, the UK must come into line with these international standards.

Julia Manning, Chief Executive of 2020health, says:

“There is currently no UK-wide orphan drug legislation, and no single approach to assessing orphan drugs even within England.  Our study will look at ways that would allow the NHS to become a world leader in dealing with terrible diseases which have been left in out in the cold for too long.”

Known as ‘orphan drugs’ 2020health believes that the NHS should have an orphan drug policy which can ensure consistent patient drug access across the UK, thus avoiding ‘postcode lotteries’.

The new Health and Social Care Bill will move both national and regional specialised commissioning groups that traditionally perform orphan drug appraisals into the hands of the NHS Commissioning Board, but apart from that, the Bill does not address in detail how the Government intends to handle specialised commissioning for rare diseases. 

Mrs Manning says:

“There is an opportunity here for us to think about how the structure of specialised commissioning will be defined, and how this will affect outcomes for patients with orphan diseases and the decision making process for orphan and ultra-orphan drugs.

“It would be absolutely against our values of societal justice and equality if for instance, patients in England could not access drugs that patients in Wales had access to.”

The team at 2020health is being led by Research Fellow and Independent Health Economist Leela Barham and Head of Policy, Dr Jonathan Shapiro. It is being supported by unrestricted educational grants from Shire and Genzyme. The aim is to prepare an independent, authoritative report making recommendations on access to medicines for people with rare medical conditions.

Leela Barham said, “The current differences in decision making processes leads to a lack of predictability for those we should all focus on - the patients - and that also has a knock on impact for incentives for future research and development. It would be in the best interest  of patients and their carers, government, clinicians and industry for there to be clarity about funding and access to medicines, both for those with rare diseases, and those seeking to treat them.”


Note to Editors

1.      For interviews, comment or further information please contact Julia Manning, 2020health Chief Executive, 07973 312358 or / Leela Barham, Research Fellow, 2020health

  1. The report publication due date is in September 2012
  1. is an independent, grass-roots, Think Tank for health and technology interested in realistic solutions. We identify issues and bring informed people together to create these solutions. Please visit for more information and full publications.