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At a Cost

Therapies aimed at changing patients' genes are poised for approval in the US and one has already reached the market in Europe, writes Erika Check Hayden at Nature News, but they come with a hefty price tag.

"Advances in science are presenting a social affordability question like never before," economist Mark Trusheim from the Massachusetts Institute of Technology tells her. "Do we want to convert the science into therapies that we actually would have to pay for?"

According to one estimate by Harvard Medical School's Stuart Orkin and Third Rock Ventures' Philip Reilly, who is also a co-founder of Bluebird Bio, gene therapy treatments could run as much as $1 million per patient. Ones approaching the approval stage in the US are aimed at treating hemophilia B, sickle-cell anemia, and cerebral adrenoleukodystrophy, Check Hayden says.

That's about what the one gene therapy treatment — for lipoprotein lipase deficiency — approved in Europe costs, she adds. So far, she notes that there's been just one patient who has taken it. That low uptake, she notes, has been attributed to both the cost and to doubts about that drug's efficacy.

For pricey drugs already on the market, Check Hayden writes that some insurers and drug companies have negotiated linking payments to how well the drug works. Such a scenario works best if there is more than one treatment option, but for some of these diseases, there's only one player and less motivation for the drug developer to negotiate, she adds.

But for some things, people might just pay. "Let's say that a gene therapy that really made a world of difference in the life of a small child should cost a million dollars for one event," Reilly says. "I can think of many things in medicine that cost that much or more, and we don't think twice about that."

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