The Orphan Therapy Trap

A new publication draws attention to what is, perhaps, the biggest problem facing cell and gene therapies. You might imagine the most pressing considerations facing innovative medicines would be: Do they work, and are they safe? But tight on the heels of these questions are: What if they work and are safe, but nobody can afford to pay for them?

The price of medicines is a continuous source of political infighting in the U.S., while even in Europe, where socialised medicine predominates, the European Union has identified affordability of drugs to be one of the major drivers of unmet medical need. A sad truth is that advanced therapies are making this problem worse. They are the most expensive drugs on the market. For example, Luxterna, the recently approved gene therapy for certain genetic forms of blindness, is reported to cost $850,000 per patient. And it is not alone. By 2014, these high-cost drugs—though only representing 1% of prescriptions in the US—accounted for 32% of spending on medicines. Indeed, so expensive are these new therapies, they are pricing themselves out of the market. Of the 10 advanced therapies that have been approved by the European Medicines Agency, four are now unavailable, cost being a primary factor in the withdrawal of these drugs. 

Full article at Psychology Today

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