Scientists undertake Gamma Knife surgery, one treatment for ocular melanoma. Photo: Bertrand Langlois/AFP/Getty Images
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Spare a thought for “orphan” drugs: the rare disease medicines that prove health is a numbers game

Oliver Sacks wrote of his imminent death with remarkable dignity, knowing science cannot help him. But what about the cases where it might?

There is remarkable dignity in the neurologist Oliver Sacks’s acceptance of his imminent death, which he revealed in a recent article in the New York Times. At the same time, he has little choice but to accept it: science cannot cure his cancer. More heartbreaking, in many ways, are those cases in which successful science is being held back by economics.

Life and death are ultimately a numbers game. Sacks’s illness began with an ocular melanoma. Each year, on average, five people per million in the US and Europe will develop one. For those over the age of 50, this happens four times as often. “Only in very rare cases do such tumours metastasise,” he wrote. “I am among the unlucky 2 per cent.”

It is tempting to think that such cases are rare but, in another sense, they are not. Diseases considered rare threaten the lives of fewer than five people in 10,000; yet there are roughly 7,000 different life-threatening rare diseases, affecting roughly 25 million people in Europe alone. Fewer than 300 of these have licensed treatment paths, which is why we so desperately need more “orphan” drugs – medicines for diseases designated as rare.

Developing orphan drugs is an unattractive prospect for pharmaceutical companies. Creating new medicines, even for common conditions, is time- and capital-intensive. With treatments for rare conditions, there is no likely return on the investment, as few will use them and national health services are unwilling to pay the prices necessary to make them commercially viable.

Hence the special designation. Orphan drugs are, in effect, subsidised at the research phase and granted exclusivity if they are successful in reaching the market. The programme seems to be working. In the US last year, the Food and Drug Administration granted 293 development efforts orphan status, an increase of 13 per cent on the previous year. Approvals of orphan drugs, releasing them for use, went up by 53 per cent. In Europe it’s a similar story. In 2011-12, designations of orphan drugs rose by 44 per cent.

It has been predicted that orphan drugs will represent nearly 16 per cent of global prescription sales by 2018, when they will be worth £82bn. Thanks to subsidies, they are almost twice as lucrative as standard drugs. You could consider this a good thing – especially if you suffer from a rare disease – but it has also triggered alarms. Austerity-hit governments are questioning the high prices of such medicines, given the research subsidy they are already paying.

This is particularly bad news for sufferers of “ultra-rare diseases” – those affecting fewer than one in 50,000 people – which make up almost one-fifth of EU orphan drug designations. Take atypical haemolytic uraemic syndrome. About 140 people in Britain have been diagnosed with this disorder of the small blood vessels that brings early death through kidney failure. A candidate drug costs roughly £340,000 per patient for each year of quality life added. Is that a good use of money?

In the UK, such decisions fall to the National Institute for Health and Care Excellence (NICE). In March, NICE’s highly specialised technologies evaluation committee will hold the first of five public meetings this year to discuss such issues. Patient groups will no doubt turn up to lobby for their particular cause – and why wouldn’t they? In the end, the decisions are always arbitrary.

The orphan drug effort is a laudable attempt to solve a most difficult problem. Yet it raises complex issues. Anyone who thinks that governments should always heal the sick is likely to be disappointed. Sometimes, we can do the science but we just can’t make the numbers add up. 

Michael Brooks holds a PhD in quantum physics. He writes a weekly science column for the New Statesman, and his most recent book is At the Edge of Uncertainty: 11 Discoveries Taking Science by Surprise.

This article first appeared in the 27 February 2015 issue of the New Statesman, Russia vs the west

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What David Hockney has to tell us about football

Why the sudden glut of blond footballers? A conversation I had with the artist back in 1966 gave me a clue. . .

In 1966, I went to interview David Hockney at a rather run-down flat in Bayswater, central London. He was 28 and had just won a gold medal at the Royal College of Art.

In his lavatory, I noticed a cut-out photograph from a newspaper of Denis Law scoring a goal. I asked if he was a football fan. He said no, he just liked Denis Law’s thighs.

The sub-editors cut that remark out of the story, to save any gossip or legal problems. In 1966 homosexual activity could still be an offence.

Hockney and a friend had recently been in the United States and had been watching an advert on TV that said “Blondes have more fun”. At two o’clock in the morning, slightly drunk, they both went out, bought some hair dye and became blond. Hockney decided to remain blond from then on, though he has naturally dark hair.

Is it true that blonds have more fun? Lionel Messi presumably thinks so, otherwise why has he greeted this brand-new season with that weird blond hair? We look at his face, his figure, his posture and we know it’s him – then we blink, thinking what the heck, does he realise some joker has been pouring stuff on his head?

He has always been such a staid, old-fashioned-looking lad, never messing around with his hair till now. Neymar, beside him, has gone even blonder, but somehow we expect it of him. He had foony hair even before he left Brazil.

Over here, blonds are popping up all over the shop. Most teams now have a born-again blondie. It must take a fortune for Marouane Fellaini of Man United to brighten up his hair, as he has so much. But it’s already fading. Cheapskate.

Mesut Özil of Arsenal held back, not going the full head, just bits of it, which I suspect is a clue to his wavering, hesitant personality. His colleague Aaron Ramsey has almost the full blond monty. Paul Pogba of Man United has a sort of blond streak, more like a marker pen than a makeover. His colleague Phil Jones has appeared blond, but he seems to have disappeared from the team sheet. Samir Nasri of Man City went startlingly blond, but is on loan to Seville, so we’re not able to enjoy his locks. And Didier Ndong of Sunderland is a striking blond, thanks to gallons of bleach.

Remember the Romanians in the 1998 World Cup? They suddenly appeared blond, every one of them. God, that was brilliant. One of my all-time best World Cup moments, and I was at Wembley in 1966.

So, why do they do it? Well, Hockney was right, in a sense. Not to have more fun – meaning more sex – because top footballers are more than well supplied, but because their normal working lives are on the whole devoid of fun.

They can’t stuff their faces with fast food, drink themselves stupid, stay up all night, take a few silly pills – which is what many of our healthy 25-year-old lads consider a reasonably fun evening. Nor can they spend all their millions on fun hols, such as skiing in the winter, a safari in the spring, or hang-gliding at the weekend. Prem players have to be so boringly sensible these days, or their foreign managers will be screaming at them in their funny foreign accents.

While not on the pitch, or training, which takes up only a few hours a day, the boredom is appalling, endlessly on planes or coaches or in some hotel that could be anywhere.

The only bright spot in the long days is to look in the mirror and think: “Hmm, I wonder what highlights would look like? I’ve done the beard and the tattoos. Now let’s go for blond. Wow, gorgeous.”

They influence each other, being simple souls, so when one dyes his hair, depending on where he is in the macho pecking order, others follow. They put in the day by looking at themselves. Harmless fun. Bless ’em.

But I expect all the faux blonds to have gone by Christmas. Along with Mourinho. I said that to myself the moment he arrived in Manchester, smirking away. Pep will see him off. OK then, let’s say Easter at the latest . . . 

Hunter Davies is a journalist, broadcaster and profilic author perhaps best known for writing about the Beatles. He is an ardent Tottenham fan and writes a regular column on football for the New Statesman.

This article first appeared in the 22 September 2016 issue of the New Statesman, The New Times