Forbes, August 31, 2018
Arlene Weintraub


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On August 23, Dutch gene therapy developer uniQure announced it had dosed the first patient in a phase 2 trial of a gene therapy to treat hemophilia B, helping push shares of the company up 10% over the following week to $42.21. The company is one of many biotechs pursuing “one-and-done” cures for genetic diseases—single-dose therapies designed to erase painful, costly and sometimes life-threatening diseases from patients’ DNA.

The science of gene therapy continues to advance at a rapid pace, with companies launching gene therapy research to treat everything from the nervous system disorder Batten disease (announced this week by Regenexbio) to overactive bladder (revealed Wednesday in Urovant Science’s announcement of a new licensing deal).

But as investor excitement over these advances rises, so do worries from health insurers and pharmacy benefits managers (PBMs) over the high costs associated with gene therapy. In an online survey of managed care organizations published in Health Affairs earlier this summer, four out of five national companies and nine of 16 regional payers rated their level of concern about the financial risks of gene therapy as five on a scale of one to five, meaning “very high.”

That’s why gene therapy developers and payers are starting to embrace the idea of alternative payment models, says Genia Long, co-author and senior advisor at the Analysis Group, a consulting company that conducted the survey in partnership with the National Pharmaceutical Council, a health policy research group that’s funded by the pharma industry.

“There’s mutual interest in finding out-of-the-box solutions,” Long says. [...]

biotechnology genetics health care costs health care finance insurance international