Paying For Gene Therapy: Will Pharma Be First In Line?

With 15 new gene and cell-based therapies in late-stage testing, the time is now to consider a critical strategic question: is there a pricing model for these budget-busting treatments that works in both safeguarding returns to innovators and providing access to patients at a price society is willing to pay? An analysis by ZS Associates looks at the options for biopharma going forward.

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Striking a balance: affordability versus innovation • Source: Shutterstock

The first-ever US gene and cell therapy approvals of 2017 suggest that the industry stands on the brink of a fundamental shift in the way transformative therapies will be financed and accessed by patients. In quick order that acknowledged the clinical merits of the science, the FDA approved the first directly administered gene therapy, Luxturna (voretigene neparvovec-rzyl), as well as the first two CAR-T therapies, Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel).

What makes these therapies noteworthy is that we can call them potentially curative. They’re administered just once, or over a...

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