Magazine article New Statesman (1996)

Spare a Thought for "Orphan" Drugs

Magazine article New Statesman (1996)

Spare a Thought for "Orphan" Drugs

Article excerpt

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 30 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. …

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