I know many families affected by cystic fibrosis have placed a great deal of hope in this drug and believe particular care should be taken to assess its effectiveness.
I am aware that, following a public consultation, the National Institute for Health and Care Excellence (NICE) published final guidance in July 2016 which did not recommend Orkambi for the treatment of cystic fibrosis. NICE concluded that, compared to the current standard of care, the benefit Orkambi offered did not justify its considerable cost.
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