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NICE unable to recommend Orkambi on cost grounds

As the National Institute for Health and Care Excellence (NICE) opts not to recommend the gene-specific medicine Orkambi to NHS England, while acknowledging the clinical benefits of the drug, the Cystic Fibrosis Trust has put forward a solution that could speed up access and evaluation.

As the National Institute for Health and Care Excellence (NICE) opts not to recommend the gene-specific medicine Orkambi to NHS England, while acknowledging the clinical benefits of the drug, the Cystic Fibrosis Trust has put forward a solution that could speed up access and evaluation.

Orkambi has been shown to increase lung function and significantly reduce infection and hospital stays for those carrying two copies of the most common mutation of the cystic fibrosis gene, F508del, and in today’s draft decision NICE finds the treatment to be effective and important for managing cystic fibrosis, but uncertainty around its long-term impact and its high cost means it cannot recommend the drug.

The Trust has proposed a solution whereby Orkambi could be provided to all who need it while further evidence is collected on its long-term clinical impact using the UK Cystic Fibrosis Data Registry.

Ed Owen, Chief Executive of the Cystic Fibrosis Trust said: "It would be ethically unforgiveable if people with cystic fibrosis were treated like pawns in a bigger battle between the NHS and Vertex over price and longer term impact. But there is a solution that requires the NHS in England to work with Vertex to provide access to Orkambi and enable the UK Cystic Fibrosis Data Registry to elucidate its value more clearly over time.”

In a letter to Life Sciences Minister George Freeman MP today, Ed Owen urges intervention to support the Trust's plan, while the Government is currently looking at new arrangements to accelerate access to medicines for those that need them.

Write to your MP
Use this letter to ask your MP to ask the UK Government to support the Trust’s solution, granting access to the drug for those who would benefit, while assessing the longer-term impact of Orkambi.