Young English sufferers of cystic fibrosis (CF) face new uncertainty after regulators published proposals which, if enacted, would restrict access to potentially life-changing treatments on cost grounds. The National Institute for Health and Care Excellence (NICE) on Friday published draft guidance that it would not recommend three treatments known as modulator therapies, manufactured by Vertex Pharmaceuticals for use on new patients. The draft guidance, open for consultation until Nov. 24, would not affect those who already take Kaftrio or Symkevi, which are currently recommended for patients aged 6 and over, or Orkambi, which can be used at age 1 and over.

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