‘Not acceptable’ disappointment as cystic fibrosis drug not approved in Scotland

People with cystic fibrosis in Scotland will continue to be denied potentially life-extending drugs after they were rejected for use by the NHS, prompting an outpouring of dismay and anger.

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.

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.

Marion Fellows with grand-daughter Saoirse (two and a half) at her home in Wishaw.

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.

Journalist and academic James McEnaney, whose wife Ruth has the disease, said: “Big thanks to the SMC for deciding that keeping people like my wife alive isn’t worth the money. Feel free to come and explain the decision to my five-year-old son any time. In Scotland we’ll fund utter bulls*** like homeopathy on the NHS, but saving the lives of people with cystic fibrosis isn’t cost-effective. Got it.”

Marion Ferguson, whose son Thomas has cystic fibrosis and who runs a group campaigning for precision medicines to treat the disease, 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.”

SNP MP Marion Fellows, whose three-year-old granddaughter Saoirse suffers from cystic fibrosis, said the disease was a heavy burden for children and their parents to bear.

“It’s not a pretty thing watching a three-week-old baby scream when they’re getting their first lot of physio and you’re told by their parents no, we’re not allowed to comfort her because her body has to get used to this,” she added.

SMC chairman Dr Alan MacDonald said he recognised the decision would be “disappointing” but there was “significant uncertainty” around the drugs’ long-term health benefits.

“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 added.

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 spokeswoman said.

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.

“I fully understand the great disappointment that people with cystic fibrosis, together with their parents, families and friends, will feel about [this decision],” she added.

David Ramsden, chief executive at the Cystic Fibrosis Trust, said the decision would come as a shock to people with cystic fibrosis and their families.

“While talk of further negotiation between Vertex Pharmaceuticals and the Scottish government is positive, people living in Scotland need these life-saving drugs now,” he said.

“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.”