People with cystic fibrosis in Scotland will continue to be denied potentially life-extending drugs after they were rejected for use by the National Health Service (NHS), prompting an outpouring of dismay and anger. According to a Scotsman report, the Scottish Medicines Consortium (SMC) said it had heard “powerful testimonies” from patients about the impact of Orkambi and Symkevi but had doubts over their cost-effectiveness.
The report says hundreds of Scots suffering from the disease, which is caused by a faulty gene and can be totally debilitating, had been hoping that the body would approve the two drugs. Families in England, where the drugs are also unavailable on the NHS, had said they would move to Scotland to allow their children to be treated if they were approved.
The report says Orkambi and Symkevi slows the decline of lung health in both children and adults – the main cause of death in those with cystic fibrosis – but the drugs are said to cost £100,000 a year per patient.
The Cystic Fibrosis Trust said the SMC’s decision would “shock” people living with the disease, who now faced another “tragic wait” for the “life-saving” medicines. SMC chair Dr Alan MacDonald said he recognised the decision would be “disappointing” but there was “significant uncertainty” around the drugs’ long-term health benefits.
Vertex, the pharmaceutical company that makes the drugs, said it was working with the Scottish government to allow “broad access” to the medicines for eligible patients. “We are all deeply committed to reaching such a solution as a matter of urgency and we are working closely with the government to finalise it over the coming weeks,” a spokesperson said.
Scotland Health Secretary Jeane Freeman said some patients in Scotland already had access to the drugs through an existing scheme, which has so far approved around 65 people on a case-by-case basis.
[link url="https://www.scotsman.com/health/not-acceptable-disappointment-as-cystic-fibrosis-drug-not-approved-in-scotland-1-4982252"]Scotsman report[/link]