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Gene therapies that are close to US approval include treatments for haemophilia B, sickle-cell anaemia and the neurodegenerative disease cerebral adrenoleukodystrophy. [...] But many of the target disorders are rare, limiting the population that can be treated. And there are often no previously approved drugs that work similarly, removing the pressure on companies to lower their prices.

Such therapies could cost $1 million per patient [...] the same price as Glybera, the gene therapy given the green light by European regulators in 2012, which has been taken by only one person so far. Experts attribute this low uptake to the high price and to doubts about its efficacy.

[...] For medicines that are already approved, one increasingly popular solution is a deal between insurers and drug companies that ties payments to how well medicines perform. Last November, for example, Boston-based Harvard Pilgrim Health Care, a major New England insurer, announced that it will cover treatment for its clients with Repatha (evolocumab), one of a new class of cholesterol-lowering medication that is made by Amgen and costs $14,000. But if patients don’t reach pre-agreed cholesterol levels, or if Harvard Pilgrim ends up paying more than it has budgeted for, Amgen will refund the insurer.

[...] The difficulties of paying for the fruits of the biotechnology revolution are something that governments are already struggling with. The state of Arkansas last year settled a lawsuit filed by three people who said they had been denied access to the $300,000 cystic fibrosis drug Kalydeco (ivacaftor) because of the cost. And in April, the Japanese government imposed a 50% price cut on a new hepatitis C treatment, Sovaldi (sofosbuvir). A US federal judge in Seattle, Washington, ruled on 27 May that states cannot delay treatment with Sovaldi, which costs up to $84,000, because of price concerns.