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December 4, 2012 comments

What price an orphan this Christmas?

Tiny Tim wasn’t the only orphan to suffer a miserable Christmas at the hands of a merciless Scrooge.  The same fate awaits so-called orphan disease patients in the UK.  This year, it’s the turn of around 400 cystic fibrosis patients, who cannot access newly-approved Kalydeco on the supposedly comprehensive National Health Service (NHS).

Its easy to imagine the sadness of children from poor families pressing their nose against the glass of the toy shop window decked out with dazzling presents for Christmas, knowing these delights will be denied them but not their school friends.  How much worse must it be knowing there is a drug that could dramatically reverse your chronic illness, but being told you cannot have any?

Yet that is exactly what is happening, not for the poor children on the block but in the world’s seventh largest economy.

On the face of it, the cause of this wholly unacceptable state of affairs is the ‘scandal’ of drug pricing.  Vertex, makers of Kalydeco, want $294,000 for a year’s treatment.

“Can it be right for the NHS to offer free IVF to infertile couples while denying Kalydeco to CF sufferers?” @sciencescanner on Twitter

But actually, the ultimate cause is the myth we (in the UK) continue to perpetuate that its possible to offer comprehensive healthcare to all, free at the point of use.  While the development of new drugs is funded almost entirely with private capital, yet healthcare is delivered from the public purse there will always be a mismatch – with companies developing drugs no-one can afford to use.

What is urgently needed is a proper debate about healthcare priorities – a debate that is stifled by the increasingly hollow assertion that the state can cover all your healthcare needs.

Pricing drugs for ‘orphan diseases’ has caused intense public debate for a number of years.  Because the cost of developing a new drug is unrelated to the number of people who will use it, making the development of drugs for rare, or orphan, diseases economically viable requires the makers to charge a higher price.  At a minimum, the price will be the cost of developing the drug divided by the number of people who will take it.

Recent estimates suggest the development cost of a new drug is stable at just over $1billion – so when the denominator is very small, the resulting price can look difficult to justify.

Perhaps the most expensive drug on the formulary right now is the anti-C5 antibody Soliris™ from Alexion, with a list price around $400,000 for a year’s treatment.  It is used to treat a rare condition called Paroxysmal Nocturnal Hemoglobinuria (PNH), a life-threatening blood disorder that is fortunately very rare – …


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