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Cystic Fibrosis Trust

NICE rejects Orkambi

Fight goes on after decision against life-changing drug.

Despite recognising the life-transforming cystic fibrosis (CF) treatment Orkambi® as important and effective for managing the condition, the National Institute for Health and Care Excellence (NICE) has today rejected it for use in England because of uncertainty around long-term value and impact, and therefore cost-effectiveness.

With NICE’s assessment complete, NHS England and the drug’s manufacturer, Vertex, can still reach an agreement that will allow people to have access to the treatment. Therefore, the Cystic Fibrosis Trust is calling on NHS England and Vertex to have meaningful discussions by the end of July. The Trust is also offering a solution to support this process.

This solution would see access to Orkambi granted while the Trust's world-renowned clinical data Registry, which holds health data for 99% of people living with the condition in the UK, tracks the long-term impact of Orkambi.

The NICE decision is based primarily on short-term data, with no confident prediction of Orkambi’s long-term value. New long-term data not considered by NICE suggests Orkambi almost halves the rate of decline in lung function in people with CF over a two-year period, potentially adding years of life.

Community action

Action from the CF community has played a vital role in raising awareness of this issue at the highest level, with an amazing 26,000 people signing a petition and writing to their MPs. This led to crucial support for our solution from MPs and Government.

In today’s open letter, the Trust calls on NHS England and Vertex to recognise this support and pay attention to thousands of people with CF and their family and friends who have made the strength of feeling on this issue so clear.

Ed Owen, Chief Executive of the Cystic Fibrosis Trust, said: “We are very disappointed by this decision. It demonstrates the weakness of the current NICE assessment process that the long-term value of the drug in slowing the decline in lung health is not sufficiently recognised.

“Our survey of people with CF and their families has shown the primary importance that those affected by CF place on stabilisation – and new data published a few weeks ago, and not seen by NICE, shows that over a two-year period Orkambi almost halves the rate of decline in lung function.

“It is now vital that NHS England and Vertex sit down and negotiate a means by which this drug becomes available to those who need it while we put in place a system to measure the real-world impact through the CF Registry.

“This issue is about people’s lives and there is no time to lose. We want to see meaningful discussions underway by the end of July to design an access solution for Orkambi and we will not sit idly by if this does not materialise.”

Clara Mann, who at 14 years old is one of the youngest on the clinical trial for Orkambi, said: "We must get this drug to everyone that needs it. Orkambi has completely changed my life. Before I started on the trial my cystic fibrosis meant I was underweight, unfit, small for my age and really unwell… I now consider myself really fit and healthy - I love running and am training for a 10k run!”

If no progress on this issue is made by the end of July we will be calling on the CF community to take further action. In the meantime, if you would like any information about this campaign, please contact publicaffairs@cysticfibrosis.org.uk.

Ed Owen

This issue is about people’s lives and there is no time to lose. We want to see meaningful discussions underway by the end of July to design an access solution for Orkambi and we will not sit idly by if this does not materialise.

Chief Executive, Cystic Fibrosis Trust
Clara Mann

Orkambi has completely changed my life. Before I started on the trial my cystic fibrosis meant I was underweight, unfit, small for my age and really unwell. I now consider myself really fit and healthy - I love running and am training for a 10km run!

Former clinical trial particpant, currently on Orkambi