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Cystic Fibrosis Trust

Orkambi: Trust Responds to NICE

Interim NICE decision highlights need to gather clinical data without denying access to medicines.

As the National Institute for Health and Care Excellence (NICE) finalises its consultation period, the Cystic Fibrosis Trust has repeated its call to grant access to new medicine Orkambi for those who could benefit, while gathering further clinical data via the UK CF Registry.

At the start of the month the advisory body issued an interim recommendation against funding the drug through NHS England because of uncertainty around its long-term impact and the drug’s high cost, while acknowledging that the treatment is: “a valuable new therapy for managing cystic fibrosis that has wider benefits to society, for people with cystic fibrosis, and carers of people with cystic fibrosis”.

The Trust’s solution would see those who could benefit start taking Orkambi, while the UK CF Registry records data to build a comprehensive picture of the long-term effectiveness of the drug.

The voice of thousands

Over 5,000 supporters wrote to their MPs this month to support the Trust’s solution, and there is still time to join this e-action launched in response to NICE’s interim decision

Earlier this year the Trust delivered a petition to 10 Downing Street as part of its Stopping the Clock campaign, with 20,000 signatures calling on the NHS across all four nations and drug companies to ensure that new medicines like Orkambi are made available to those who would benefit, without delay.

Ed Owen, Chief Executive of the Trust, said today: “It is vital that the focus of the NHS, Government and Vertex is to ensure that people who need it are given access to Orkambi as quickly as possible.  Every day that passes is one day too late.

“While there appears to be some early signs that both the Government and Orkambi’s manufacturer, Vertex, recognise this, it is time to go beyond the words and take action.  By using the UK CF Registry we can build the longer-term picture of Orkambi’s impact and avoid people with cystic fibrosis becoming innocent victims of an unacceptable limbo between the NHS and the pharmaceutical industry.”

Read the Trust’s submission to the consultation.

NICE will meet again to discuss Orkambi and comments on the interim recommendation on 28 April, with a final decision expected in July.