1. ITV Report

'It's a death sentence' Families devastated by delays in decision over life-changing drug

Families of children with a muscle wasting condition have suffered a major blow after a decision over a potentially life-changing drug was delayed.

The drug

Leo Le Gal from Ivybridge has Duchenne Muscular Dystrophy and is on a trial of Translarna. His family say the drug has slowed down the devastating symptoms of his illness.

His mother Ruth has been fighting for the NHS to approve Translarna, which is widely available in Europe.

Leo was treated with Translarna in a trial in America Credit: ITV West Country

It's a very serious illness, it is fatal if children continue to deteriorate without having access to medication the prognosis is grim. Many kids die by the age of 18.

Now that Leo's condition has been stabilised by this medication he's got a hopeful future.

– Ruth Le Gal, mother

The decision

It was hoped the drug would be approved for NHS use, but NICE has today announced in its draft guidance it will not yet recommend funding on the NHS.

They say more research is needed, and have asked the company to provide further justification for the cost of the drug before confirming its final position.

“The Committee heard from the patient experts that one of the most important aspects of managing DMD is maintaining their child’s ability to walk because this means they can continue to go to school independently and participate more fully in social and sporting activities with family and friends. There is, therefore, a clear need for a treatment that can prolong a child’s ability to walk but without serious side-effects.

"However, the Committee was not convinced that the proposed cost of [Translarna] was justified by the evidence presented."

"It's a death sentence"

Leo's mother is devastated by the decision, saying that denying children this medicine is akin to a death sentence.

Click to hear her powerful statement:

It's NICE saying to these kids 'Sorry, you can't have this medicine, we're going to leave you to die'

We're talking about children who are 5 or 6 years old.

– Ruth Le Gal

She added that there needs to be immediate access to this medication, even on a temporary basis, so that children will be able to take Translarna while they wait for the results of the research.

The Prime Minister accepts the petition in January 2015 Credit: ITV News

Ruth had a personal assurance from the Prime Ministerthat this was going to be looked into. She says she feels "let down".

About Duchenne muscular dystrophy

Children and adults in the UK suffer from the condition - all of them male

The condition is caused by the lack of a vital muscle protein called dystrophin, leading to muscles weakening and wasting over time and increasingly severe disability.

The vital heart and breathing muscles are affected, meaning that few people with the condition live to see their 30th birthday.

Today's news is extremely disappointing, and a bitter blow for all of us awaiting the news. Having spoken to some of the families affected, it really is impossible to describe their heartbreak.

– Robert Meadowcroft, Chief Executive of Muscular Dystrophy UK

"If we are not careful, it will be too late"

The charity Muscular Dystrophy UK says it fears that any more delays in approving the drug will mean that it will be too late for many boys to be eligible to take Translarna.

Robert Meadowcroft, Chief Executive of Muscular Dystrophy UK said “Duchenne is a life-limiting condition, and we must take every opportunity to protect children and their quality of life. The chance to be able to walk for longer can be immeasurably precious. We may not yet be able to halt the difficult physical and emotional challenges these children face, but with this drug, we have the chance to delay them."