Scots families still waiting for life-saving drug six months after NHS approval

Families are demanding to know why a drug which can extend the life of severely ill boys is not being made widely available, despite being approved by the NHS last year.

Following a meeting with dozens of those affected by Duchenne muscular dystrophy (DMD), an incurable and degenerative condition, health secretary Neil Gray has vowed to speak with NHS bosses to ensure the medication is rolled out as quickly as possible.

Givinostat has been authorised for use in the UK, but the NHS has only rolled it out in a small number of sites in England and Wales, and patients in Scotland and Northern Ireland have been unable to access it at all.

While some health boards could begin their rollout within weeks, others are months behind.

‘A postcode lottery’

Michael Harvey was diagnosed with Duchenne muscular dystrophy (DMD) when he was two years old.

His parents, Mhairi and Michael Harvey, described their little boy as “a fun-loving wee character” who keeps them on their toes and loves life.

But Michael’s muscles are continuing to degenerate while he waits for his local health board to deliver the drug.

“I used to be a nurse, and I understand the pressures and constraints, but I can’t understand why there has been no prospect of this being delivered in a six-month time frame,” Mrs Harvey told STV News.

“Especially when the health minister confirmed there are no financial constraints [on its delivery]. To make it almost a postcode lottery, it breaks our hearts,” Michael’s dad added.

Lawyer Aamer Anwar, representing the families, described the situation as “unbelievable”.

“Every single day a boy loses the use of a muscle, it doesn’t have to be their leg – it can be their heart, their lungs.

“There is nothing complicated about delivering this drug despite the excuses being given.

“Neil Gray said his government is ready to help clinicians remove any barriers that exist. He stated to the families that there are no financial barriers.

“He will seek an urgent explanation as to why NHS Grampian is so far behind. Families say we cannot have a two-tier health system across the country. “

Mr Anwar has threatened legal action on behalf of families affected by the condition if the medication is not delivered.

Decision for health boards

“I want to work with health boards to make sure they’re all able to deliver the drug on an early access basis as fast as possible,” Gray told STV News.

“I want this to be delivered equitably, fairly, and as close as possible for each person living in different parts of the country. I don’t want to see disparities between one part of the country and another.”

The decision to supply medications like Givinostat falls with individual health boards and not the Scottish Government.

For families in the Greater Glasgow and Clyde region, the health board said the drug is likely to be made available by the end of May.

Families affected by Duchenne meet with Neil Gray STV News

But for patients in NHS Lothian, it could be weeks longer and in NHS Grampian, they could be waiting for months for full SMC approval.

Dr Tracey Gillies, medical director, NHS Lothian said: “While Givinostat is being provided at no cost through an Early Access to Medicines scheme for a limited duration, the substantial extra clinical care and monitoring required for patients to receive the drug safely has not been funded.

“We are working at pace to explore how we can expand the clinical team and provide the resources needed to provide this drug safely for our patients.”