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First tested in patients a quarter-century ago, gene therapy — a risky approach aimed at fixing the malfunctioning genes at the root of some diseases — is finally emerging from the darkness after weathering high-profile tragedies, including the death of a teenage patient.
As it evolves from experimental to applied medicine, gene therapy might soon find itself steeped in a new controversy: soaring drug prices. No therapy is approved yet in the United States, so discussions about price — as well as crucial questions about how much patients will pay directly — are hypothetical. But industry leaders are already talking about ways to get ahead of potentially massive one-time price tags that could make insurers and patients balk.
A gene therapy approved in Europe in 2012 costs close to $1 million, and prices are expected to follow suit in the United States. The therapies in the pipeline are mostly for rare genetic diseases: sickle cell, hemophilia or immune deficiency. Their likely high prices stem from the expected value; unlike drugs that a person takes regularly, gene therapies are designed to be given once and have lasting effects.
But everyone involved anticipates the potential backlash against a seven-figure price tag, which is leading to radical proposals. Instead of paying for a treatment all at once, insurers and patients could make installment payments as long as the therapy works, similar to a mortgage on a house.
Read full, original post: Gene therapies offer dramatic promise but shocking costs