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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Trade unions must change or face permanent decline

Union membership will fall below one in five employees by 2030 unless current trends are reversed. 

The future should be full of potential for trade unions. Four in five people in Great Britain think that trade unions are “essential” to protect workers’ interests. Public concerns about low pay have soared to record levels over recent years. And, after almost disappearing from view, there is now a resurgent debate about the quality and dignity of work in today’s Britain.

Yet, as things stand, none of these currents are likely to reverse long-term decline. Membership has fallen by almost half since the late 1970s and at the same time the number of people in work has risen by a quarter. Unions are heavily skewed towards the public sector, older workers and middle-to-high earners. Overall, membership is now just under 25 per cent of all employees, however in the private sector it falls to 14 per cent nationally and 10 per cent in London. Less than 1 in 10 of the lowest paid are members. Across large swathes of our economy unions are near invisible.

The reasons are complex and deep-rooted — sweeping industrial change, anti-union legislation, shifts in social attitudes and the rise of precarious work to name a few — but the upshot is plain to see. Looking at the past 15 years, membership has fallen from 30 per cent in 2000 to 25 per cent in 2015. As the TUC have said, we are now into a 2nd generation of “never members”, millions of young people are entering the jobs market without even a passing thought about joining a union. Above all, demographics are taking their toll: baby boomers are retiring; millennials aren’t signing up.

This is a structural problem for the union movement because if fewer young workers join then it’s a rock-solid bet that fewer of their peers will sign-up in later life — setting in train a further wave of decline in membership figures in the decades ahead. As older workers, who came of age in the 1970s when trade unions were at their most dominant, retire and are replaced with fewer newcomers, union membership will fall. The question is: by how much?

The chart below sets out our analysis of trends in membership over the 20 years for which detailed membership data is available (the thick lines) and a fifteen year projection period (the dotted lines). The filled-in dots show where membership is today and the white-filled dots show our projection for 2030. Those born in the 1950s were the last cohort to see similar membership rates to their predecessors.

 

Our projections (the white-filled dots) are based on the assumption that changes in membership in the coming years simply track the path that previous cohorts took at the same age. For example, the cohort born in the late 1980s saw a 50 per cent increase in union membership as they moved from their early to late twenties. We have assumed that the same percentage increase in membership will occur over the coming decade among those born in the late 1990s.

This may turn out to be a highly optimistic assumption. Further fragmentation in the nature of work or prolonged austerity, for example, could curtail the familiar big rise in membership rates as people pass through their twenties. Against this, it could be argued that a greater proportion of young people spending longer in education might simply be delaying the age at which union membership rises, resulting in sharper growth among those in their late twenties in the future. However, to date this simply hasn’t happened. Membership rates for those in their late twenties have fallen steadily: they stand at 19 per cent among today’s 26–30 year olds compared to 23 per cent a decade ago, and 29 per cent two decades ago.

All told our overall projection is that just under 20 per cent of employees will be in a union by 2030. Think of this as a rough indication of where the union movement will be in 15 years’ time if history repeats itself. To be clear, this doesn’t signify union membership suddenly going over a cliff; it just points to steady, continual decline. If accurate, it would mean that by 2030 the share of trade unionists would have fallen by a third since the turn of the century.

Let’s hope that this outlook brings home the urgency of acting to address this generational challenge. It should spark far-reaching debate about what the next chapter of pro-worker organisation should look like. Some of this thinking is starting to happen inside our own union movement. But it needs to come from outside of the union world too: there is likely to be a need for a more diverse set of institutions experimenting with new ways of supporting those in exposed parts of the workforce. There’s no shortage of examples from the US — a country whose union movement faces an even more acute challenge than ours — of how to innovate on behalf of workers.

It’s not written in the stars that these gloomy projections will come to pass. They are there to be acted on. But if the voices of union conservatism prevail — and the offer to millennials is more of the same — no-one should be at all surprised about where this ends up.

This post originally appeared on Gavin Kelly's blog