Portree woman sees progress on cystic fibrosis treatment

The manufacturers of treatment which can “slow decline in lung function” for people living with cystic fibrosis has announced that those living with the illness in Scotland could have access to precision medicines through the NHS next year.

Vertex Pharmaceuticals which produces the treatments – Orkambi and Symkevi – made the announcement following “constructive discussion with the Scottish Government” with the next step in the process being the submission of an application to the Scottish Medicines Consortium, which is responsible for appraising whether the medicines are eligible to be made available on the national health service.

Earlier this month, the Free Press reported on the challenges faced by 23-year old Hannah McDiarmid who was diagnosed with cystic fibrosis aged 17 weeks-old.  Hannah, who lives in Torvaig near Portree on Skye, is campaigning so that those living with CF can access treatments such as Orkambi, a drug – which according to the Cystic Fibrosis Trust – can “slow decline in lung function – the main cause of death among people with cystic fibrosis – by 42 per cent.”

While Orkambi has been licensed for use in the UK, it is currently not available through the NHS as the Scottish Medicine Consortium – which provides advice to NHS Scotland about the value for patients of every newly released medicine – has deemed the submitting company, Vertex Pharmaceuticals’  justification of the treatment’s cost in relation to its health benefits “not sufficient.” Adding that the company did “not present a sufficiently robust clinical and economic analysis to gain acceptance by SMC.”

Speaking to the Free Press in December, Hannah, who has previously undergone operations for polyps in her nose, suffered stomach problems and chronic fatigue, believed it would be “too late” for her before cystic fibrosis suffers were able to access Orkambi and other new treatments for CF through the NHS.

After seeking comment from the manufacturer on whether they would resubmit an application for the treatments to the SMC, Vertex Pharmaceuticals Europe released a statement to Free Press which stated that “following constructive discussions with the Scottish Government” Vertex would “submit ORKAMBI (lumacaftor/ivacaftor) as well as SYMKEVI (tezacaftor/ivacaftor) to be used in combination with ivacaftor, to the Scottish Medicines Consortium for appraisal. If accepted by the SMC for use on the NHS in Scotland, eligible patients with cystic fibrosis in Scotland could have access to these precision medicines in 2019.”

Ludovic Fenaux, the Senior Vice President of Vertex International said: “Our recent conversations with the Scottish Government, and the SMC’s orphan medicines process have provided important flexibility for evaluating precision medicines, such as cystic fibrosis transmembrane conductance regulator (CFTR) modulators. Their methods reflect the innovative nature of medicines that have the potential to extend life for patients with rare diseases, like CF.” He added: “We are hopeful that, through this process, all eligible patients in Scotland could have access to our medicines soon.”

Responding to the announcement, David Ramsden, CEO of the Cystic Fibrosis Trust, said: “This is great news and offers real hope that people with cystic fibrosis across Scotland will soon be able to access these vital drugs. It is now critical that the statements are followed by rapid action and we see similar developments across the whole UK.  There can be no more delays!”

Hannah McDiarmid, meanwhile, told the Free Press: “I’m glad things are moving along and that hopefully the medicines can be approved.”

Vertex and the Scottish Government have also agreed to a confidential discount which will be applied to applications to the PACS Tier 2 process.  Should the appraisal be positive, patients in Scotland will join those in other countries around the world where all those eligible have access to lumacaftor/ivacaftor, including Austria, Australia, Denmark, Germany, the Republic of Ireland, Italy, the Netherlands, Sweden and the U.S.