The next decade promises a wave of innovative and potentially curative gene and cell therapy products representing breakthroughs for patients with devastating and costly diseases, many of which lack current treatments. Patients with certain forms of inherited blindness, rare genetic muscle and neurological disorders, hemophilia, untreatable cancers and other serious conditions may soon have transformative treatment options for the first time in the form of gene therapies, gene editing technologies and cell therapies such as CAR-Ts (chimeric antigen receptor T cells), and other types of regenerative medicines. As discussed in our previous article, many of these therapies are expected to provide durable and profound treatment effects with a single administration of therapy, effectively curing the disease or condition. (Also see "Curative Regenerative Medicines: Preparing Health Care Systems For The Coming Wave" - In Vivo, 15 November, 2016.) However, traditional payment and financing models based on cost-per-unit of product or per procedure may be suboptimal to support adoption, patient access and continued innovation of these therapies.
These therapies are expected to deliver their benefits over a long period of time, ranging from several years to the...
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