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Patients have their say on Orkambi

Lynsey Beswick, Public Affairs Officer at the Cystic Fibrosis Trust, will share her views on Orkambi® from a patient perspective with the Scottish Medicines Consortium (SMC) today.

The SMC provides advice to the NHS in Scotland on the clinical and cost-effectiveness of new treatments like Orkambi®, in a similar way to NICE in England, who also heard Lynsey's views at the first NICE meeting for the evaluation of Orkambi last week.

Orkambi, which combines ivacaftor and another compound called lumacaftor, could bring significant health benefits for those people with cystic fibrosis aged 12 and over who have two copies of the F508del mutation. According to UK CF Registry data (2014), 236 people in Scotland are eligible for the drug.

Darren O’Keefe, Public Affairs Manager at the Trust, said: “Today’s meeting is another important milestone towards achieving our goal of ensuring access to precision medicines to as many people as possible by 2020 as part of our wider fight for a life unlimited by cystic fibrosis. However, access is not guaranteed and we are prepared for a sustained campaign.”

The SMC will meet again in April, and their decision is expected in May; depending on the outcome, Orkambi could then be referred to the New Medicines Fund, which provides extra funding for treatments for people with rare or end-of-life conditions.

The Trust’s next step will be to hand the Scottish Government a petition calling on drug manufacturers and the NHS across all four UK nations to take responsibility for ensuring access to precision medicines. A phenomenal 20,000 people signed the Stopping the Clock petition, including 1,800 from Scotland.