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- W2397917110 abstract "Gene therapies have the potential to cure rare conditions that often have no current efficacious treatments with a one-time treatment episode, relieving substantial unmet need and having profound positive impact on patients’ lives. However, with the first gene therapy now licensed and priced at around US$1 million per patient, cost and uncertain funding mechanisms present a potential barrier to patient access. In this article, we discuss the unique challenges presented by gene therapies, particularly concerning the uncertainty inherent in their clinical evidence package at launch and their affordability within strained healthcare budgets. We present several payment models that would allow for sustainable reimbursement of these innovative technologies and make recommendations pertinent both to those developing gene therapies and to those paying for them." @default.
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- W2397917110 date "2016-06-01" @default.
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- W2397917110 title "Gene therapies: the challenge of super-high-cost treatments and how to pay for them" @default.
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- W2397917110 doi "https://doi.org/10.2217/rme-2016-0010" @default.
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