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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Rarely has it mattered so little if Manchester United won; rarely has it been so special they did

Team's Europa League victory offers chance for sorely needed celebration of a city's spirit.

Carlo Ancelotti, the Bayern Munich manager, memorably once said that football is “the most important of the least important things”, but he was only partly right. While it is absolutely the case that a bunch of people chasing around a field is insignificant, a bunch of people chasing around a field is not really what football is about.

At a football match can you set aside the strictures that govern real life and freely scream, shout and cuddle strangers. Football tracks life with such unfailing omnipresence, garnishing the mundane with regular doses of drama and suspense; football is amazing, and even when it isn’t there’s always the possibility that it’s about to be.

Football bestows primal paroxysms of intense, transcendent ecstasy, shared both with people who mean everything and people who mean nothing. Football carves out time for people it's important to see and delivers people it becomes important to see. Football is a structure with folklore, mythology, language and symbols; being part of football is being part of something big, special, and eternal. Football is the best thing in the world when things go well, and still the best thing in the world when they don’t. There is nothing remotely like it. Nothing.

Football is about community and identity, friends and family; football is about expression and abandon, laughter and song; football is about love and pride. Football is about all the beauty in the world.

And the world is a beautiful place, even though it doesn’t always seem that way – now especially. But in the horror of terror we’ve seen amazing kindness, uplifting unity and awesome dignity which is the absolute point of everything.

In Stockholm last night, 50,000 or so people gathered for a football match, trying to find a way of celebrating all of these things. Around town before the game the atmosphere was not as boisterous as usual, but in the ground the old conviction gradually returned. The PA played Bob Marley’s Three Little Birds, an Ajax staple with lyrics not entirely appropriate: there is plenty about which to worry, and for some every little thing is never going to be alright.

But somehow the sentiment felt right and the Mancunian contingent joined in with gusto, following it up with “We’ll never die,” – a song of defiance born from the ashes of the Munich air disaster and generally aired at the end of games, often when defeat is imminent. Last night it was needed from the outset, though this time its final line – “we’ll keep the red flag flying high, coz Man United will never die" – was not about a football team but a city, a spirit, and a way of life. 

Over the course of the night, every burst of song and even the minute's silence chorused with that theme: “Manchester, Manchester, Manchester”; “Manchester la la la”; “Oh Manchester is wonderful”. Sparse and simple words, layered and complex meanings.

The match itself was a curious affair. Rarely has it mattered so little whether or not United won; rarely has it been so special that they did. Manchester United do not represent or appeal to everyone in Manchester but they epitomise a similar brilliance to Manchester, brilliance which they take to the world. Brilliance like youthfulness, toughness, swagger and zest; brilliance which has been to the fore these last three days, despite it all.

Last night they drew upon their most prosaic aspects, outfighting and outrunning a willing but callow opponent to win the only trophy to have eluded them. They did not make things better, but they did bring happiness and positivity at a time when happiness and positivity needed to be brought; football is not “the most important of the least important things,” it is the least important of the most important things.

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