Parents plead NHS to allow ‘fighter’ son access to life changing drug before it’s too late

Sam Millar’s parents are angry at red tape holding back the drug, despite it being licensed in Scotland.

Two Scots parents are pleading with the NHS to allow their “fighter” son access to a life changing drug before it is too late.

Sam Millar from Edinburgh, was diagnosed with Duchenne muscular dystrophy, a disease that leads to loss of mobility, as well as heart and respiratory complications.

The eight-year-old been approved for givinostat, a treatment proven to slow the effects of the disease. But, his parents have been told that the NHS does not have the resources to administer it – despite it being licensed in Scotland on December 20 last year.

Dr Tracey Gillies, medical director at NHS Lothian said that newly licensed medicine is ‘not routinely prescribed’. She added that there are ‘processes in place’ to provide earlier access, with patients considered on an ‘individual basis’.

Parents Karla and Stephen Millar are now making a heartfelt please for the necessary resources to be provided, so that Sam can receive treatment as soon as possible before his symptoms become irreversible.

Stephen told Edinburgh Live: “For every day, week, and month that passes, Sam and other boys with Duchenne are getting weaker.

“The EAP has been available since November; we are now in March. Sam has been approved for early access, yet we’ve been told the NHS doesn’t have the resources to deliver the treatment now.

“We understand the complexities involved in approving new treatments, but the reality is that every day matters when it comes to Duchenne,” says Stephen.

“Sam is a fighter, and we’ll do everything we can to help him. But we need the Cabinet Health Secretary to step in and ensure that no child in Scotland suffers because treatment is withheld due to red tape.”

Karla added: “We’re running out of time. Every day counts for Sam. He’s a brave boy, but his muscles are weakening, and we fear that if the treatment isn’t given soon, his chances of living a longer, healthier life could be lost.

“We need the Cabinet Health Secretary to listen to our plea and ensure that Sam can access the treatment that could slow the progression of Duchenne.”

Edinburgh South West MP Scott Arthur has intervened and written to Neil Gray, MSP, on behalf of Sam. He’s called on the Cabinet Health Secretary to provide the Royal Hospital for Children and Young People with the resources needed to administer givinostat.

Duchenne muscular dystrophy affects approximately one in every 3,500 male births. There is currently no cure, advances in treatment can slow progression – if provided in time.

Dr Tracey Gillies, Medical Director, NHS Lothian, said: “We are unable to discuss individual patients without their consent. In cases where a newly licensed medicine is currently under evaluation by the Scottish Medicines Consortium (SMC) and awaiting guidance, it is not routinely prescribed in Scotland.

“There are processes in place to provide earlier access to medicines in the interim period and patients and their circumstances are considered on an individual basis.”