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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Apprenticeships remain a university alternative in name only for too many young people

New research shows that those who do the best apprenticeships will earn higher salaries than graduates, but government targets undermine the quality of such schemes.

Rare is the week that passes by without George Osborne donning a hi-vis jacket and lauding the worth of apprenticeships. The Conservatives have made creating 3m apprenticeships a governing mission. Labour, both under Ed Miliband and Jeremy Corbyn, are scarcely less enthusiastic about their value.

The best apprenticeships live up to the hype. Those with a level five apprenticeship (there are eight levels) will earn £50,000 more in their lifetime than someone with a degree from a non-Russell Group university, as new research by the Sutton Trust reveals.

But too many apprenticeships are lousy. In 2014/15, just 3 per cent of apprenticeships were level four or above. Over the last two years, there have only been an estimated 30,000 apprenticeships of at least level four standard. So while David Cameron comes up with ever grander targets for the amount of apprenticeships he wants to create, he neglects what really matters: the quality of the apprenticeships. And that's why most people who can are still better off going to university: over a lifetime the average graduate premium is £200,000.

Proudly flaunting lofty targets for apprenticeships might be good politics, but it isn’t good policy. “The growth in apprenticeships has been a numbers game with successive governments, with an emphasis on increasing quantity, not quality,” says Sir Peter Lampl, Chairman of the Sutton Trust.

60 per cent of apprenticeships today are at level two – considered to be no better than GCSE standard. These might help people get a job in the short-term, but it will do nothing to help them progress in the long-term. Too often an apprenticeship is seen as an end in itself, when it should be made easier to progress from lower to higher apprenticeships. The Sutton Trust is right to advocate that every apprentice can progress to an A-Level standard apprenticeship without having to start a new course.

Apprenticeships are trumpeted as an alternative to going to university. Yet the rush to expand apprenticeships has come to resemble the push to send half the population to university, focused more on giving ever-greater numbers a qualification then in ensuring its worth. For too many young people, apprenticeships remain an alternative to university in name only.

Tim Wigmore is a contributing writer to the New Statesman and the author of Second XI: Cricket In Its Outposts.