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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Grenfell survivors were promised no rent rises – so why have the authorities gone quiet?

The council now says it’s up to the government to match rent and services levels.

In the aftermath of the Grenfell disaster, the government made a pledge that survivors would be rehoused permanently on the same rent they were paying previously.

For families who were left with nothing after the fire, knowing that no one would be financially worse off after being rehoused would have provided a glimmer of hope for a stable future.

And this is a commitment that we’ve heard time and again. Just last week, the Department for Communities and Local Government (DCLG) reaffirmed in a statement, that the former tenants “will pay no more in rent and service charges for their permanent social housing than they were paying before”.

But less than six weeks since the tragedy struck, Kensington and Chelsea Council has made it perfectly clear that responsibility for honouring this lies solely with DCLG.

When it recently published its proposed policy for allocating permanent housing to survivors, the council washed its hands of the promise, saying that it’s up to the government to match rent and services levels:

“These commitments fall within the remit of the Government rather than the Council... It is anticipated that the Department for Communities and Local Government will make a public statement about commitments that fall within its remit, and provide details of the period of time over which any such commitments will apply.”

And the final version of the policy waters down the promise even further by downplaying the government’s promise to match rents on a permanent basis, while still making clear it’s nothing to do with the council:

It is anticipated that DCLG will make a public statement about its commitment to meeting the rent and/or service charge liabilities of households rehoused under this policy, including details of the period of time over which any such commitment will apply. Therefore, such commitments fall outside the remit of this policy.”

It seems Kensington and Chelsea council intends to do nothing itself to alter the rents of long-term homes on which survivors will soon be able to bid.

But if the council won’t take responsibility, how much power does central government actually have to do this? Beyond a statement of intent, it has said very little on how it can or will intervene. This could leave Grenfell survivors without any reassurance that they won’t be worse off than they were before the fire.

As the survivors begin to bid for permanent homes, it is vital they are aware of any financial commitments they are making – or families could find themselves signing up to permanent tenancies without knowing if they will be able to afford them after the 12 months they get rent free.

Strangely, the council’s public Q&A to residents on rehousing is more optimistic. It says that the government has confirmed that rents and service charges will be no greater than residents were paying at Grenfell Walk – but is still silent on the ambiguity as to how this will be achieved.

Urgent clarification is needed from the government on how it plans to make good on its promise to protect the people of Grenfell Tower from financial hardship and further heartache down the line.

Kate Webb is head of policy at the housing charity Shelter. Follow her @KateBWebb.