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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In the 1980s, I went to a rally where Labour Party speakers shared the stage with men in balaclavas

The links between the Labour left and Irish republicanism are worth investigating.

A spat between Jeremy Corbyn’s henchfolk and Conor McGinn, the MP for St Helens North, caught my ear the other evening. McGinn was a guest on BBC Radio 4’s Westminster Hour, and he obligingly revisited the brouhaha for the listeners at home. Apparently, following an interview in May, in which McGinn called for Corbyn to “reach out beyond his comfort zone”, he was first threatened obliquely with the sack, then asked for a retraction (which he refused to give) and finally learned – from someone in the whips’ office – that his party leader was considering phoning up McGinn’s father to whip the errant whipper-in into line. On the programme, McGinn said: “The modus operandi that he [Corbyn] and the people around him were trying to do [sic], involving my family, was to isolate and ostracise me from them and from the community I am very proud to come from – which is an Irish nationalist community in south Armagh.”

Needless to say, the Labour leader’s office has continued to deny any such thing, but while we may nurture some suspicions about his behaviour, McGinn was also indulging in a little airbrushing when he described south Armagh as an “Irish ­nationalist community”. In the most recent elections, Newry and Armagh returned three Sinn Fein members to the Northern Ireland Assembly (as against one Social Democratic and Labour Party member) and one Sinn Fein MP to Westminster. When I last looked, Sinn Fein was still a republican, rather than a nationalist, party – something that McGinn should only be too well aware of, as the paternal hand that was putatively to have been lain on him belongs to Pat McGinn, the former Sinn Fein mayor of Newry and Armagh.

According to the Irish News, a “close friend” of the McGinns poured this cold water on the mini-conflagration: “Anybody who knows the McGinn family knows that Pat is very proud of Conor and that they remain very close.” The friend went on to opine: “He [Pat McGinn] found the whole notion of Corbyn phoning him totally ridiculous – as if Pat is going to criticise his son to save Jeremy Corbyn’s face. They would laugh about it were it not so sinister.”

“Sinister” does seem the mot juste. McGinn, Jr grew up in Bessbrook during the Troubles. I visited the village in the early 1990s on assignment. The skies were full of the chattering of British army Chinooks, and there were fake road signs in the hedgerows bearing pictograms of rifles and captioned: “Sniper at work”. South Armagh had been known for years as “bandit country”. There were army watchtowers standing sentinel in the dinky, green fields and checkpoints everywhere, manned by some of the thousands of the troops who had been deployed to fight what was, in effect, a low-level counter-insurgency war. Nationalist community, my foot.

What lies beneath the Corbyn-McGinn spat is the queered problematics of the ­relationship between the far left wing of the Labour Party and physical-force Irish republicanism. I also recall, during the hunger strikes of the early 1980s, going to a “Smash the H-Blocks” rally in Kilburn, north London, at which Labour Party speakers shared the stage with representatives from Sinn Fein, some of whom wore balaclavas and dark glasses to evade the telephoto lenses of the Met’s anti-terrorist squad.

The shape-shifting relationship between the “political wing” of the IRA and the men with sniper rifles in the south Armagh bocage was always of the essence of the conflict, allowing both sides a convenient fiction around which to posture publicly and privately negotiate. In choosing to appear on platforms with people who might or might not be terrorists, Labour leftists also sprinkled a little of their stardust on themselves: the “stardust” being the implication that they, too, under the right circumstances, might be capable of violence in pursuit of their political ends.

On the far right of British politics, Her Majesty’s Government and its apparatus are referred to derisively as “state”. There were various attempts in the 1970s and 1980s by far-right groupuscules to link up with the Ulster Freedom Fighters and other loyalist paramilitary organisations in their battle against “state”. All foundered on the obvious incompetence of the fascists. The situation on the far left was different. The socialist credentials of Sinn Fein/IRA were too threadbare for genuine expressions of solidarity, but there was a sort of tacit confidence-and-supply arrangement between these factions. The Labour far left provided the republicans with the confidence that, should an appropriately radical government be elected to Westminster, “state” would withdraw from Northern Ireland. What the republicans did for the mainland militants was to cloak them in their penumbra of darkness: without needing to call down on themselves the armed might of “state”, they could imply that they were willing to take it on, should the opportunity arise.

I don’t for a second believe that Corbyn was summoning up these ghosts of the insurrectionary dead when he either did or did not threaten to phone McGinn, Sr. But his supporters need to ask themselves what they’re getting into. Their leader, if he was to have remained true to the positions that he has espoused over many years, should have refused to sit as privy counsellor upon assuming his party office, and refused all the other mummery associated with the monarchical “state”. That he didn’t do so was surely a strategic decision. Such a position would make him utterly unelectable.

The snipers may not be at work in south Armagh just now – but there are rifles out there that could yet be dug up. I wouldn’t be surprised if some in Sinn Fein knew where they are, but one thing’s for certain: Corbyn hasn’t got a clue, bloody or otherwise. 

Will Self is an author and journalist. His books include Umbrella, Shark, The Book of Dave and The Butt. He writes the Madness of Crowds and Real Meals columns for the New Statesman.

This article first appeared in the 25 August 2016 issue of the New Statesman, Cameron: the legacy of a loser