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Cystic fibrosis drugs to be available on NHS in England within 30 days

BMJ 2019; 367 doi: https://doi.org/10.1136/bmj.l6206 (Published 24 October 2019) Cite this as: BMJ 2019;367:l6206
  1. Zosia Kmietowicz
  1. BMJ

Every patient in England with cystic fibrosis who could benefit from one of the new precision drugs that target certain mutations underlying the condition will have access to them within 30 days, NHS England has said after striking a deal with the US manufacturer Vertex Pharmaceuticals.

NHS England has been trying to secure access to Orkambi (a combination of lumacaftor and ivacaftor), Symkevi (tezacaftor and ivacaftor), and Kalydeco (ivacaftor) for more than 18 months. Pressure mounted last month after the Scottish government negotiated a confidential discount with Vertex to make Orkambi available to 400 patients.1

Vertex had wanted £105 000 (€123 000; $138 000) per patient for Orkambi. Last July it rejected what NHS England said was a “fair and final offer” of around £500m over five years …

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