Cystic fibrosis: NHS England approves Orkambi, Symkevi and Kalydeco

  • NHS England

  • curated by Dawn O'Shea
  • UK Medical News
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NHS England has secured an agreement with Vertex Pharmaceuticals to make all three of its UK-licensed cystic fibrosis medicines available on the NHS.

NHS England estimates that 5000 patients will be eligible to avail Orkambi (lumacaftor/ivacaftor), Symkevi (tezacaftor/ivacaftor) and Kalydeco (ivacaftor), which will be available within the next 30 days.

The announcement follows lengthy negotiations between the NHS and the manufacturers regarding the cost of the treatments, with the cost of Orkambi alone estimated at £104,000 (excluding value-added tax) per patient. However, NHS England says it has now agreed a confidential deal with the manufacturers which constitutes “good value for British taxpayers.”

The company has also agreed to submit its drugs, including its new triple therapy, for full NICE appraisal. Based on this agreement, NICE has backed the new deal. The company has also agreed to a flexible commercial mechanism which will ensure continued patient access to these three medicines following completion of the NICE appraisal. NICE previously decided against recommending Orkambi, and the assessment of Symkevi was suspended.

Commenting on the announcement, Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE, said: “We are pleased an agreement has been reached between NHS England and Vertex that brings these medicines to patients. It is also welcome that the company will now re-engage with the NICE process."

“We look forward to working with the company and patient organisations to get ready for the appraisals, including developing the approach to the collection of evidence.”

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