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Access to medicines for rare diseases in Scotland

Last Reviewed 23/03/2016

On Wednesday 23 March 2016, we launched our new Patient Charter ‘Patient perspectives and priorities on access to medicines for rare conditions in Scotland”’ The Charter is the product of a workshop, held by Genetic Alliance UK and attended by a broad range of organisations supporting patients affected by rare and genetic conditions, to gather their views as to the impact of the reforms on the transparency, accountability and appropriateness of the various routes by which rare disease patients in Scotland access the medicines to treat their condition.

The Patient Charter finds that while the reforms to Scottish Medicines Consortium processes carried out as a result of the 2013 new medicines reviews and Health and Sport Committee inquiry have improved transparency, it is not clear to what extent they have improved patient access to medicines for rare conditions. The Patient Charter makes a number of recommendations for further improvements to the Scottish Medicines Consortium appraisal process as well as to improve the transparency and accountability of the activities of the New Medicines Fund, the proposed transition from Individual Patient Treatment Request to Peer Approved Clinical System, and at the level of local health board Area Drug and Therapeutics Committees.

Our five key recommendations:

  • There is a need for an increase in representation and type of patient voice on all decision-making panels
  • Greater clarity as to the role of patient responses in Scottish Medicines Consortium decision-making is necessary
  • The New Medicines Fund should be retained with increased transparency on its use
  • Clarity on the process and timeframe of the transition to Peer Approved Clinical System is urgently needed
  • Careful monitoring of health board uptake of new medicines to local formularies may be necessary


Are you a Genetic Alliance UK member organisation? Show your support for this Charter by signing up to endorse the recommendations. To do this fill in the form (takes two minutes) or contact our Policy Officer, Louise.


The annexes and references for the report can be found here.

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