Mother: Early access to ground-breaking drugs is risk worth taking to improve son's life

Lawrence McGinty

Former Science and Medical Editor

Emily Crossley from Duchenne Children's Trust tells Lawrence McGinty about her son Eli's condition Credit: ITV News

I met a remarkable young boy the other day. Six-year-old Eli bounced into his living room and started to pull faces for our camera.

It's impossible to think anything is wrong with this vivacious young boy. But Eli carries a fatal genetic inheritance.

He has Duchenne Muscular Dystrophy, a terrible disease that will gradually waste his muscles.

Boys with DMD are in wheelchairs by their teenage years and rarely survive much past 21.

Eli is just one patient who could benefit from the new scheme to fast-track treatments that offer a lifeline to people facing a bleak medical future.

At the moment, new drugs have to go through extensive trials to show that they work and that they're safe. Those trials are incredibly expensive and take years.

In the meantime patients who could benefit are left seriously ill or even dying. The new scheme is intended to get the drugs to the patients much more quickly.

We're not talking about "routine" drugs here - the scheme is intended to apply to significant new medicines that could be breakthrough drugs.

And we're talking only about patients with life-threatening or debilitating diseases who have run out of treatment options - they're in the last-chance saloon.

So that's patients with genetic diseases like DMD, or cancer or dementia. But there are thousands, or perhaps tens of thousands of them.

Emily Crossley from Duchenne Children's Trust tells Lawrence McGinty about her son Eli's condition Credit: ITV News

Emily Crossley, Eli's mother, told me that patients facing the certain prospect of dying young are willing to take more risks.

After Eli was diagnosed three years ago she set up a charity, the Duchenne Children's' Trust, which has raised £1.5 million to sponsor research.

But if that research comes up with a new treatment, boys might have to wait years before it goes through the trials it needs to get a licence.

Emily Crossley's son Eli suffers from Duchenne Muscular Dystrophy, a disease that will gradually waste his muscles Credit: ITV News

In the new scheme, the safety authority, the MHRA, would approve significant new drugs before they get a licence. Patients could then receive treatments that could change their lives.

Drug companies have welcomed the scheme - although they don't like the fact that they would have to pay for the new medicines in the scheme.

And David Cameron says the scheme could attract cutting-edge companies to the UK.

So everyone benefits - patients get potentially life-saving drugs early, drug companies get their products into the clinics earlier, and the country benefits from bringing new business to Britain.

But wouldn't the scheme mean that patients were guinea-pigs for drugs that haven't been fully tested?

When I put that point to Emily, her answer was loud and clear. For her son, that's a risk worth taking for a treatment that could transform his life and future.

Read: Severely ill to get early access to ground-breaking drugs