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Cystic Fibrosis Trust

Pressure grows for Orkambi access

As NICE deliberates final decision, Trust’s proposal gains hearing

The National Institute for Health and Care Excellence (NICE) met yesterday (Thursday 28 April) to discuss comments on its interim recommendation for Orkambi, a new potentially life-changing medicine that could benefit thousands of people with cystic fibrosis in the UK.

NICE’s guidance proposed that Orkambi should not be available for use in England because it was not cost-effective, despite acknowledging that it is a valuable new therapy for managing cystic fibrosis that has wider benefits to society, for people with cystic fibrosis and their loved ones.

At the meeting, held in Manchester with representatives of a wide range of organisations including the Cystic Fibrosis Trust, it was revealed that the British Thoracic Society (BTS), a membership organisation of doctors, nurses, respiratory physiotherapists, scientists and other professionals with a respiratory interest, have joined the Trust’s call for conditional approval of Orkambi, with use of UK CF Registry data to assess its long-term, real world impact.

NICE’s committee agreed that this model has the potential to address uncertainties in the trial data and explore the wider societal benefit of the medicine in more detail but claimed that the company, Vertex Pharmaceuticals, had not put forward a formal proposal to be considered.

NHS England did not send a representative to the meeting.

With over 5,000 people joining the Trust’s campaign to seek Government support for our proposal, the Trust is still awaiting a formal response from the Minister for Life Sciences, George Freeman.

Ed Owen, Chief Executive of the Cystic Fibrosis Trust, said: “We remain confident that the Trust’s proposed solution to enable access to Orkambi is the best way forward. We welcome the support for our proposal from the BTS and NICE’s support for exploring creative solutions where possible.

“However, time equals lives, and it is essential that the proposal is discussed directly by the two key parties in this process, NHS England and Vertex Pharmaceuticals.

“Vertex have indicated their willingness to begin these discussions and we hope that NHS England and the Government will do too soon. Today, we reissue our call for these negotiations to begin with the utmost urgency.”

NICE expects to issue its final guidance in July this year.

What's it like to watch the fate of a possible treatment hang in the balance? Read a firsthand account of yesterday's meeting by Lynsey Beswick, Public Affairs Officer.