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This debate pack has been compiled ahead of the debate on Access to drugs for ultra-rare diseases to be held on Tuesday 16 June 2015 at 2.30pm in Westminster Hall. The Member in charge of the debate is Greg Mulholland. Debate packs are produced quickly after the announcement of Parliamentary business. They are intended to provide a summary or overview of the issue being debated and identify relevant briefings and useful documents, including press and parliamentary material. More detailed briefing can be prepared for MPs on request to the Library.

Rare and ultra-rare diseases each affect a very small number of patients, but overall about 3 million people in the UK will be affected by one of these conditions.  The UK Rare diseases Strategy, published in November 2013, and to be implemented across the UK, seeks to address some of the specific challenges relating to these conditions, such as improving diagnosis, and investing in research for these conditions. 

Drugs for rare diseases (orphan medicines) are usually granted marketing authorisations through a centralised procedure at the European Medicines Agency and services for rare conditions are the direct commissioning responsibility of NHS England in England.  More information on the process for consideration of new treatments is provided in the briefing in this debate pack.

Prior to the Westminster Hall debate, Digital Outreach and Genetic Alliance UK organised a digital Rare Disease debate on Twitter.  The debate started at 2pm on Monday 15 June. A number of patients and their families, patient groups, charities and the Member in charge of the Westminster Hall debate, Greg Mulholland all contributed. 

The idea for digital debates linked to debates in Westminster Hall came from the Speaker’s Commission on Digital Democracy, which stated: 

“We believe the public want the opportunity to have their say in House of Commons debates; we also believe that this will provide a useful resource for MPs and help to enhance those debates. We therefore recommend a unique experiment: the use of regular digital public discussion forums to inform debates held in Westminster Hall.”

The Twitter debate formally finished at 4pm yesterday but it is likely that people will continue to contribute using the hashtag #RDdebate throughout the Westminster Hall debate today. To keep up with the debate on Twitter please use this link: Twitter Rare Disease debate.

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