Two drugs that are potentially life-extending for people with cystic fibrosis have been rejected for use in Scotland.
The Scottish Medicines Consortium heard ‘powerful testimonies’ from patients and doctors about the potential benefits of Orkambi and Symkevi.
But the body ruled them out, saying there were too many doubts about whether the drugs would prove cost-effective.
Both drugs, which cost about £100,000 per year per patient, slows the decline of lung function – the main cause of death in CF.
The decision will come as a blow to campaigners who have worked tirelessly in the hope of the drugs being made available nationwide.
The manufacturer of both drugs, Vertex, and the Scottish Government are now working ‘as a matter of urgency’ to make the drugs accessible.
The Scottish Medicines Consortium heard ‘powerful testimonies’ from patients and doctors about the potential benefits of Orkambi (pictured) and Symkevi
Cystic fibrosis is a debilitating illness, which creates a mucus build-up that causes chronic lung infections and progressive lung damage.
The incurable disease affects around 70,000 people worldwide, including 3,000 in the UK, according to figures.
Orkambi is used to treat cystic fibrosis in patients aged two and older while Symkevi treats those aged 12 and up.
The Cystic Fibrosis Trust previously claimed Orkambi could benefit more than 330 people in Scotland.
SMC chairman Dr Alan MacDonald said the consortium recognised the decisions would be ‘disappointing’.
‘We understand the profound impact that cystic fibrosis has on patients and their families and we are aware of the need for effective treatments that target the underlying cause,’ he said.
A ‘considerable number’ of Scottish patients have access to Orkambi and Symkevi (pictured) through the Peer Approved Clinical System Tier 2 (PACS Tier 2)
‘Patient groups and clinicians gave powerful testimonies about the impact of the condition and the potential benefits of these medicines.
‘As these are orphan medicines, our committee members were able to apply additional flexibility in their decision-making but there remained significant uncertainty around their overall health benefits in the long-term, in relation to their costs.
‘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 was aware the decision would cause ‘great disappointment’.
She said: ‘Since the Government became aware of the SMC decision last month we have been working with the SMC, the manufacturer and with other interested parties to develop the principles of a solution that can make possible the widest availability of these medicines, wherever clinically appropriate.
‘This work continues now, as a matter of urgency.’
A ‘considerable number’ of Scottish patients have access to Orkambi and Symkevi through the Peer Approved Clinical System Tier 2 (PACS Tier 2).
This allows doctors to apply for access on behalf of individual patients.
Manufacturer Vertex said it hoped to reach a solution with the Scottish Government regarding broad access to the drugs for eligible patients over the coming weeks.
The English health service has PREVIOUSLY offered Vertex £500million for a five-year supply for the nation but was turned down by the Boston-based firm.
The NHS in Scotland was considered to be closer to reaching an agreement. The negotiations have stalled in the rest of the UK.
Cystic Fibrosis Trust chief executive David Ramsden said: ‘The SMC’s advice will come as a shock to people with cystic fibrosis and their families in Scotland.
‘While talk of further negotiation between Vertex Pharmaceuticals and the Scottish Government is positive, people living in Scotland need these life-saving drugs now.
‘The tragic wait has to end and now is the time for the Scottish Government to remain committed and Vertex to do all they can to ensure that this chance does not slip away again.’
SNP MP Marion Fellows’ three-year-old granddaughter, Saoirse, was diagnosed with cystic fibrosis when she was three weeks old.
Ms Fellows said she was ‘disappointed’ about the decision made by the SMC.
She said: ‘We have waited patiently over the last three and a half years when Orkambi was first submitted to and subsequently not recommended by the SMC.
‘Along with everything that CF throws at us as a community, we have campaigned, given press interviews, attended meetings and lobbied parliament.
‘All of this while watching loved ones and children deteriorating and dying. It’s simply not acceptable in a civilised society that children and young adults are left to suffer when a medicine exists that would end that suffering.’
For more information on cystic fibrosis and the fight for Orkambi and Symkevi visit the Cystic Fibrosis Trust.
WHAT IS ORKAMBI?
Orkambi is a medication licensed for use by people in the UK with cystic fibrosis, but it is not offered on the NHS except in extreme circumstances.
The drug targets the F508del gene mutation, which affects about 50 per cent of all people with cystic fibrosis.
It is made of a combination of drugs – lumacaftor and ivacaftor – which work together to keep a healthy balance of salt and water in the lungs.
There may be more than 3,000 people with life-limiting cystic fibrosis in the UK who could benefit from the drug.
But the National Institute for Health and Care Excellence (Nice) said in 2016 may not be cost-effective enough, at £104,000 per patient per year, to be offered on the NHS.
More than 117,000 people have signed a petition calling on the Government to make Orkambi available on the NHS, and Parliament discussed the petition in March this year.
Nice is expected to review its stance on the drug in July 2019.
Daniel Bodio, an engineer from Swindon in his mid-30s, was prescribed the drug on compassionate grounds.
He said it improved his lung function from 25 per cent – when he was facing having to have a transplant – to 39 per cent within three weeks.
He told MailOnline last year: ‘The drug should be available to everyone – it brings normality to what is otherwise a difficult existence.’