We’re working with Muscular Dystrophy UK to make sure that emerging treatments for Duchenne get to those who need them, as quickly as possible. Since 2016, our organisations have been representing your interests at ongoing review meetings, monitoring the progress of the Translarna (also known as ataluren) managed access agreement (MAA). The meetings involve clinicians, NHS England and NICE, and a key topic of these discussions is data collection.
Importance of data collection
The MAA requires data about the clinical impact of the treatment and the impact on the quality of life of the children, their families and carers. This involves looking at how Translarna affects the person’s physical, mental and social well-being.
This data is very important. It will help in understanding the impact of Translarna on children and their families, and will be crucial in deciding the outcome of the MAA and long-term availability of Translarna.
We must all make sure that a strong evidence base can be presented about the clinical and personal impact of the treatment, when funding is reviewed in 2020. So it’s really important that you attend your six-monthly appointments for this data to be collected and recorded.
For more information
For more information about the MAA, please read our Frequently Asked Questions and if you’d like any more information, or have questions about Translarna or the MAA, please contact the two people leading this successful campaign: Clare Lucas (MDUK) or Shelley Simmonds (Action Duchenne).
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