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Press Release Patients face postcode lottery in accessing orphan medicines for rare diseases within the UK, warns new report

Patients with life-threatening rare diseases face a lottery because the availability of ‘orphan’ drugs varies in different parts of the UK, according to a major new study carried out by a leading think-tank.

Drugs for rare diseases (affecting fewer than five in 10,000 people) are known as ‘orphan’ drugs. Rare diseases collectively are likely to affect some 3.5 million people in the UK. 2020health’s report, “Orphan Medicines: Special treatment required?” compares guidance on access in the countries making up the UK.

In England, the National Institute for Health and Clinical Excellence (NICE), which provides guidance on the most effective way to treat disease, has only appraised 18 indications relating to the 68 orphan drugs licensed for use. That compares to 56 reviewed by the Scottish Medicines Consortium (SMC), and 51 by the All Wales Medicines Strategy Group (AWMSG). Of these appraised NICE has rejected around 33% of Orphan Medicines for use of the NHS and England, AWMSG rejected 59% and SMC refused almost half of 48%.

This means that patients face greater challenges in gaining access to treatment if they have a rare disease then if they have a more common condition. Often clinicians and patients face either no guidance to help make the case for funding locally, or different guidance depending on where they live. And that lack of guidance can lead to different local decisions. This places the burden of gaining access to orphan drugs that can make the difference to their lives on the vulnerable themselves, often unable to manage the task.

Professor Timothy Cox, Professor of Medicine at the University of Cambridge said: “It has often been noted that the rarity of a disease provides no consolation for those whose suffer from it. This publication delves further into what treatments are possible for patients so afflicted and how the barriers for access to effective therapy have been built up – and how they might best be overcome.”

The report urges Health Technology Assessment agencies throughout the UK to apply a wider assessment of values similar to the assessment made by Advisory Group for National Specialised Services for appraisal of Orphan Medicines.

Julia Manning, Chief Executive of 2020Health, said: “People with rare diseases are utterly dependent on the NHS and they deserve a fair deal. Patients, and their clinicians, need to know what drugs are available and these decisions made quickly and fairly wherever they live” She went on to say; “The UK must also play its role globally, as rare diseases need international collaboration to develop effective treatments, more so than in any other disease area. Greater collaboration between the UK and other EU countries would go a long way towards bringing more treatments to market, and then agencies like NICE, will need to work with others across the UK and the NHS, to address inequalities between patients in different countries.”

Mrs Manning concluded: “Access to life-saving drugs should not be determined by your postcode. The National Health Service should work for people living in all parts of the UK. Patients should be involved in early decisions about research and development for orphan drugs. Working with individual companies to identify targets and patient-focused outcomes measures would reap cost and health benefits for patients.”

Project steering group Chair Dr Jonathan Shapiro, Senior Lecturer at Birmingham University added “it’s not just a matter of delivery; the rules about the development and commercial viability of orphans drugs need to be different from those of normal, common or garden drugs. If the same rules are applied, the likelihood of new and important drugs being developed is bound to drop, which could only be a bad thing.”

Note to Editors
1. 2020health is grateful for all of the insights from experts; the content however of this report is independent and may not reflect the views of experts and sponsors but represents only the views of the 2020health project team. The research was funded by an unrestricted educational grant from Pfizer, Genzyme and Shire.

2. For interviews, comment or further information please contact either Andrew Collinson at Media Intelligence Partners on 07592 979054 or Julia Manning, 2020health Chief Executive, on 020 3170 7702 or or .

3. is an independent, grassroots think-tank passionate about creating a healthy society. We identify issues and bring informed people together to create these solutions. Please visit for more information and full publications.

ABPI statement on 2020 Health publication 'Orphan medicines: special treatment required?

14.11.12 - ABPI Chief Executive Stephen Whitehead has commented on the 2020 Health publication, 'Orphan medicines: special treatment required?', saying that report demonstrates the problems UK patients face in getting medicines for rare diseases.

National Health Executive Magazine Nov/Dec2012 (PDF, 782 KB)

National Health Executive Magazine Nov/Dec2012

Orphan Medicines: Special treatment required? Leela Barham and Julia Manning of 2020health discuss ways of improving access to orphan drugs

Patients with rare conditions face postcode lottery

Research shows that patients with rare conditions have less chance of accessing 'orphan' medication if they live in England read on Patients with rare conditions face postcode lottery