Zolgensma is a life-saving drug but it costs more than a million and a half pounds per patient. Who decides if the NHS can afford it?
Claudia williams, narrating:
Hello, I’m Claudia and this is the Sensemaker.
One story, every day, to make sense of the world.
Today… the world’s most expensive drug, and how to get it.
In September 2020 Megan Willis gave birth to her first child, a boy called Edward.
But after a few weeks she noticed that Edward wasn’t really moving around much or holding his head up… A nurse reassured her it was nothing to worry about.
“And then a week later he was… my partner took him to the shops and on his way home, he just stopped breathing. And, um, luckily my partner looked in the mirror cause we had like, uh, a mirror over his seat, and Edward was just completely blue…”Megan Willis
Megan called her dad, who’s a paramedic, and he told her to get Edward to a hospital – now.
“And luckily we lived like five minutes away from the hospital so we raced up there. And then Edward went completely blue again, and again we had to resuscitate him as we were running into A&E, and then a nurse just took him and was like run, run. So me and this nurse – and she had Edward and she was giving him mouth to mouth and, um, we… he went into recuss and then that was it. Bedlam.”Megan Willis
When Megan told me this story on Zoom my heart was racing – even though I know that Edward survived that hospital visit.
In fact, he’s actually playing and eating snacks on the floor behind Megan as we’re chatting. You can hear him in the background sometimes when Megan talks.
At the hospital doctors diagnosed Edward with spinal muscular atrophy – or SMA.
It’s a genetic, progressive disease that affects the nervous system and causes muscle weakness and breathing problems.
About 65 babies are born with the condition every year in the UK and most are diagnosed with severe SMA, which has a life expectancy of just two years.
“Yeah o up until five years ago, I think, the prognosis was two years. Parents were just told: there’s nothing we can do. There’s no treatment, go home and love your baby and make him, make them, as comfortable as possible.”Megan Willis
When Edward was diagnosed in November 2020 he was right on the cusp of change.
A new one-off gene therapy called Zolgensma was transforming the future for children with SMA.
There was just one problem.
Zolgensma wasn’t yet available through the NHS.
And at £1.79m per treatment… it’s the most expensive drug in the world.
So who gets to decide what medicines are on offer on the NHS?
The National Institute for Health and Clinical Excellence, or NICE, was set up in 1999 to end “the postcode lottery” in England and Wales, when different treatments were available for patients depending on where they lived.
“In the two decades since it was established, NICE has built a world leading reputation for the vital work it does driving improvements in the quality and value for money of health and social care…”Clip from launch of new NICE 5-year plan
The purpose of NICE is to produce evidence-based guidance for health and social care.
As part of that work NICE assesses new and existing treatments and makes recommendations about what the national health service should spend its money on.
The NHS has limited resources so there are big ethical and moral questions at play.
Where should the NHS budget go? Whose life should be saved or improved… and how?
Each set of guidelines NICE makes is developed by an independent decision-making committee.
These are multidisciplinary, meaning they are made up of a real range of contributors: from specialists such as practitioners, providers, and researchers… to the people using services, their carers, and relevant volunteers.
These committees decide whether the benefit of the treatment outweighs the cost to the NHS. They consider how many extra years of life it might provide… what quality of life… whether there are other options… or how well-evidenced it is.
The NHS then goes on to make deals with pharmacological companies for the appropriate treatments.
It’s a model that’s been exported around the world.
But as you can imagine, this can be really controversial. Here’s one woman talking to BBC5Live about the decision to reject a breast cancer drug…
“Every day, every week, every month I get is a bonus. Like I said I’ve got a husband at home I’ve got four young children and they need their mum. There isn’t… how can they put a price on mu life? They can’t, they can’t do that and I need this drug…”BBC 5Live interview in 2014
The drug was, eventually, approved. But it shows just how emotionally complicated these processes are for the real people waiting for decisions.
The time that NICE’s rigorous recommendations can take has actually been widely criticised – although things are speeding up.
Megan Willis’s wait for a decision about Zolgensma was comparatively short… still, she tells me she thinks she has PTSD from that period. SMA is a race against time: the damage the disease does can’t be reversed.
Zolgensma was approved by the NHS in March 2021.
“Oh, it was incredible. It was just, uh, it was a moment that I’ll never forget. Because I felt like I had my baby back. So the best moment was when it was actually first approved. So that was in March and I Zoomed my whole family – this was back in lockdown obviously. And you know, we just Zoomed and everyone was crying and just laughing and like, oh… it felt like it had been lifted off my shoulders.”Megan Willis
Only… that quickly changed. NICE approved Zolgensma for babies under 6 months old who weren’t on any other treatment.
Edward was already 6 months old and was trying another treatment.
Eventually a national multidisciplinary team looked at his case and approved him for Zolgensma in August 2021.
It’s changed his life.
“I think we’re just at the beginning of what this is actually going to offer Edward and what is actually going to do for his life. Because it takes a year to actually set in, for it to really start working. But just what we have seen is, um, he can now sit up, he’s got full head control. He can talk, he can drink water, which he couldn’t drink before. He can roll. He laughs, he plays, he’s naughty, which he was never naughty before…”Megan Willis
It’s such a new treatment that there’s no real blueprint for Edward’s future. Instead what the drug has given the family is hope.
But Megan is conscious of all the families still waiting for decisions.
And there will be some of those who – inevitably – find themselves on the wrong side of the cost-benefit line.
Today’s story was written by me, Claudia Williams, and produced by Ella Hill
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