Bar on cystic fibrosis drugs will cost lives, warn campaigners
TWO ‘life-saving’ cystic fibrosis drugs have been rejected for general use by the NHS in Scotland.
The Scottish Medicines Consortium (SMC) yesterday ruled out Orkambi and Symkevi on cost grounds.
The move sparked an outcry from campaigners, who say lives are being lost.
Cystic fibrosis (CF) causes a build-up of sticky mucus in the lungs, leaving sufferers vulnerable to respiratory infections. Life expectancy for those with the condition is only 31.
David Ramsden of the Cystic Fibrosis Trust said the SMC decision will ‘come as a shock’ to sufferers and their families.
He added: ‘There would have been fewer deaths if these drugs had been available and we know that people have died during their wait. People need these life-saving drugs now. The tragic wait has to end.’
There are around 950 CF sufferers in Scotland. The rejected drugs treat the most common form, mutation F508del, which affects around 350 people.
Both were developed by the firm Vertex. Orkambi, which was licensed for use in the UK four years ago, is used to treat patients aged two and older, while Symkevi is for those aged 12 and above.
The drugs are available to a small number of NHS patients on a case-by-case basis. One of them, Kelli Gallacher, 25, from Alexandria, Dunbartonshire, has been prescribed Symkevi.
Her mother, Maggie, said: ‘My daughter has been lucky enough to be granted Symkevi and it has made the world of difference to her. As a mother it is wonderful to know she now has a future. But there are so many more people out there who are not so lucky and it is for them that we need to keep fighting.’
The SMC said it had received ‘powerful testimonies’ from patients and medics about the ‘potential benefits’ of the drugs.
Chairman Dr Alan MacDonald said: ‘We understand the profound impact that CF has on patients and their families and we are aware of the need for effective treatments that target the underlying cause.’ But ‘there remained significant uncertainty around their overall health benefits in the longterm, in relation to their costs’.
He added: ‘In order to be able to accept these medicines the committee will need to be satisfied of their cost-effectiveness and we continue to work with the company to achieve that.’
Health Secretary Jeane Freeman said she understood the ‘great disappointment’ caused by the SMC decision. She added: ‘The Government hopes that all parties continue to work together to achieve a positive outcome for all the children and adults with CF in Scotland.’
A spokesman for Vertex said it is working with the Government ‘to develop a solution to allow broad access for all eligible CF patients in Scotland’.
‘We need to keep fighting’