Appraisal of two cystic fibrosis medicines goes ahead in Scotland

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Powerful testimony and compelling evidence presented on Ivacaftor and Orkambi.

The Scottish Medicines Consortium (SMC) heard moving personal stories from people with cystic fibrosis as it met today to consider the case for making two life-changing medicines available to people with the condition in Scotland.
Both Kalydeco for two- to five-year-olds with specific mutations (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R), and lumacaftor–ivacaftor therapy (Orkambi®) for people with two copies of the F508del mutation over the age of 12 were appraised for clinical and cost effectiveness.
Testimonies revealed personal accounts of the daily battle with cystic fibrosis and the potential of these new drugs, reflecting the Trust’s work to put the voice of the community at the heart of the Stopping the Clock campaign for fairer access to precision medicines.
Yvonne Hughes, the Cystic Fibrosis Trust’s Public Affairs Officer for Scotland, who attended the event said: “The power of the cystic fibrosis community’s voice is critical to the SMC’s understanding of this condition, and it is encouraging to see the committee really trying to understand the reality behind the science and statistics.
“We continue to engage with the process and are hopeful for a positive resolution for everyone affected by the decision.”
The decision will be announced on 9 May.

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