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5 March 2015

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?

By Michael Brooks

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.

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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.