Gene therapies target rare diseases

The Western world’s first drug to fix faulty genes promises to transform the lives of patients with an ultra-rare disease that clogs their blood with fat. The only snag is the price.

The gene therapy for lipoprotein lipase deficiency (LPLD), a hereditary disorder that raises the risk of potentially lethal inflammation of the pancreas, is likely to cost more than $1 million per patient when it goes on sale in Europe this summer.

Rare or so-called orphan diseases are winning unprecedented attention from drug developers. More than a quarter of the 39 new medicines approved in the United States last year were designated for such conditions.

These are therefore exciting times for campaigners such as Briton Jill Prawer, who leads an LPLD support group, and others championing the needs of people with equally obscure illnesses. Naturally, she is delighted by the arrival of the LPLD drug Glybera. “It’s brilliant,” said Prawer, a 50-year-old mother of three who has suffered all her life from LPLD.

Until now, governments and insurance companies have largely accepted prices that can run into hundreds of thousands of dollars for products that treat orphan diseases. As only a handful of patients need the treatment, the overall cost to health budgets is relatively small. However, healthcare providers are increasingly having to balance the acute needs of the few against the wider interests of society, within constrained budgets. Scrutiny of the sky-high prices charged for this wave of new drugs is growing.

“More companies are getting into this sector because they’ve seen the eye-wateringly high prices that can be charged for some of these very rare disease medicines,” said Karl Claxton, professor of health economics at the University of York. “It’s unsustainable. Healthcare systems around the world are under increasing financial pressure and all of them are starting to look very carefully at what they get for their money.”

View the original article here: Rare diseases market booming as more drugs reach market, gene therapy poses new drug pricing challenge

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