Scotland Fails To Recommend Orkambi But 'New Medicines Fund' Should Enable Access

The Scottish Medicines Consortium (SMC) on May 9 published advice mirroring that of its neighbor NICE failing to recommend the use of Vertex's Orkambi on the UK's NHS for the treatment of cystic fibrosis. This comes despite positive reimbursement decisions in other European countries. However, Scotland's New Medicines Fund should ensure there is a route available for patients to access the medicine, Vertex believes.

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In March, NICE, the HTA body for England and Wales, said it recognized the clinical benefits of Vertex Pharmaceuticals Inc.'sOrkambi (lumacaftor/ivacaftor) in the treatment of cystic fibrosis (CF) but treatment costs in relation to its health benefits were not sufficient to recommend its use (Also see "'No Surprise' Says Vertex As NICE Fails To Recommend Orkambi In CF" - Scrip, 23 March, 2016.). The SMC says it agrees with NICE's conclusion on Orkambi and will also not be recommending the use of Kalydeco (ivacaftor) in younger children.

Orkambi is used to treat cystic fibrosis in patients aged 12 years and over who have the F508del mutation. "The Committee was unable to recommend [Orkambi] as there was too...

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