Last week Action Duchenne attended a meeting of the Cross Party Group on Rare, Genetic and Undiagnosed Conditions at the Scottish Parliament.
The meeting was organised by Genetic Alliance UK (GAUK), a national charity working to improve the lives of patients and families affected by all types of genetic conditions, with an alliance of over 200 different patient organisations. The meeting focused on access to new medicines for rare conditions with the presentation of GAUK’s ‘Resetting the Model’ report.
The system used to assess medicines in the UK is somewhat fragmented – 16 different pathways currently exist to review medicines with different funding thresholds.
It is often unclear why one assessment route is chosen over another and the total value of rare disease medicines isn’t captured in terms of costs which fall outside of the NHS (such as social care). This causes a systemic problem with a conflict between the high costs of the medicines compared to the funding available.
In Scotland, the Scottish Medicines Consortium issued new guidance in June 2018 which aimed to bring consistency to the prescription of certain medicines not recommended for routine use in NHS Scotland.
The guidance asked the Scottish NHS Health Boards to establish a Peer Approved Clinical System (PACS) panel to consider relevant requests, introduced refreshed decision making criteria and established a National Review Panel to consider the requests. The National Review Panel, hosted by Health Improvement Scotland, replaced the Scottish NHS Health Boards’ local appeal processes.
Through the ‘Resetting the Model’ report, Genetic Alliance UK wish to deliver a vision of one single decision pathway which is fair and transparent for accessing rare disease medicines. This could have an impact on emerging treatments for Duchenne in the future. We will continue to update the community of any developments.
Image by Colin
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