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Rare Disease UK (www.raredisease.org.uk/) is the national alliance for people with rare diseases and all who support them. RDUK was established by Genetic Alliance UK, the national charity of over 140 patient organisations supporting all those affected by genetic conditions, in conjunction with other key stakeholders in November 2008 following the European Commission’s Communication on Rare Diseases: Europe’s Challenges.

Subsequently RDUK successfully campaigned for the adoption of the Council of the European Union’s Recommendation on an action in the field of rare diseases. The Recommendation was adopted unanimously by each Member State of the EU (including the UK) in June 2009. The Recommendation calls on Member States to adopt plans or strategies for rare disease by 2013. 

A report was developed by Rare Disease UK (RDUK) called ‘Improving Lives, Optimising Resources: A Vision for the UK Rare Disease Strategy’. The report contains a section on ‘Access to orphan medicines’ in which it states ‘UK patients with rare diseases are being denied access to orphan medicines that have been granted European marketing authorisation.’ The Recommendation is that ‘There is an urgent need to reassess the mechanism and methodology by which the value of medicines for rare conditions is appraised for reimbursement on the NHS, to ensure improved and equitable access to licensed medicines from which patients will benefit.’       


RDUK organised a parliamentary reception hosted by Jackie Baillie MSP on 22 February 2011 at which Mr Will Scott – as Head of the Long Term Conditions Unit in the Scottish Government Health Directorates - accepted the Report ‘Improving Lives, Optimising Resources: A Vision for the UK Rare Disease Strategy’ on behalf of the Scottish Government.

RDUK has been made aware of a number of recent IPTR’s for treatment of patients with rare diseases, who have been refused funding despite the treatments being available as part of nationally commissioned services in England, or have been accepted by different health boards in Scotland.


RDUK hosted an event in conjunction with the Scottish Medicines Consortium on 23 June 2011. The aims of the event  were:

• To build a dialogue between different stakeholders from the rare disease community and the Scottish Medicines Consortium.
• To gain clarity about the assessment process for access to medicines for rare diseases.
• To explore how we can proceed in a constructive partnership with the Scottish Medicines Consortium.

Speakers from the Scottish Medicines Consortium Executive Board were invited to present at the meeting to explain the Scottish Medicines Consortium’s structure and decision making process. Following the presentations, attendees were asked to join in discussion in relation to the aims listed above. Representatives from different stakeholder groups, with an interest in rare diseases and the Scottish Medicines Consortium attended the event and were informed of processes used to appraise the value of medicines for rare diseases. Rare Disease UK remains concerned that the appraisal process does not adequately capture the unique nature of rare diseases and the problems developing medicines for rare diseases.

In addition, the following Parliamentary Questions have been asked and answered in relation to rare diseases and access to orphan  medicines:

- S3W-40250 - Richard Simpson:
- S3W-39573 - Nanette Milne:
- S3W-40248 - Richard Simpson:
- S3W-40247 - Richard Simpson:
- S3W-38010 - Richard Simpson:

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