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New system for appraising medicines for rare diseases to be developed in Wales

The All Wales Medicines Strategy Group has been asked to develop and implement a new system for identifying, appraising and monitoring orphan and ultra-orphan medicines. The request, announced this week (30 April 2014) by Welsh health minister Mark Drakeford, follows a review published in 2013 of the current appraisal system.

The review included suggestions that an orphan and ultra-orphan medicine group be established to provide recommendations to the AWMSG. It also suggested that the process for accessing these medicines should be “simplified and streamlined”, however it did not propose that a dedicated fund for such medicines should be created. Professor Drakeford explained that the aim of the new system is to “ensure that patients with rare diseases have fair and equitable access to appropriate, evidence-based treatments”.

Professor Drakeford also revealed that a public consultation will take place into a review of the individual patient funding request process in Wales. The consultation period closes on 25 June 2014. Further details can be found on the Welsh Government website.

Citation: The Pharmaceutical Journal DOI: 10.1211/PJ.2014.11138036

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