2017 has been a marquee year for gene therapy and medical technologies which harness—and manipulate—the body’s very biological building blocks to fight disease. But with great innovation comes profound questions about cost. And the expected sky-high price tags for new treatments in the cell-based therapeutics field will be an important factor for patients with deadly rare diseases.
A new report by the U.S. Institute for Clinical and Economic Review (ICER) suggests that the potential $1 million list price for Spark Therapeutics’ gene therapy Luxturna—which recently received a unanimous FDA advisory committee recommendation to treat a rare, inherited form of blindness, considerably bolstering its chances for regulatory clearance—isn’t validated by its expected benefit. (To be clear, no price has actually been set for Spark’s treatment since it hasn’t actually been approved yet.)
“[A]t a placeholder price of $1,000,000, the high cost makes this unlikely to be a cost-effective intervention at commonly used cost-effectiveness thresholds,” said the group.
ICER is a controversial entity among drug makers who argue it gives short shrift to life-changing medical innovation; Spark’s therapy, for instance, would only require a single dose for a potentially long-lasting benefit that gives sight to the blind. From an outside perspective, the notion of a $1 million drug might give many pause; for a patient who desperately wants to reverse his or her condition, the calculus could be more nuanced.
This debate isn’t limited to Spark, whose drug could be approved by mid-January or even sooner. Swiss pharma giant Novartis and U.S. biotech Gilead Sciences unit Kite Pharma both won landmark FDA approvals this year for cancer drugs that re-engineer the body’s immune system cells to kill cancerous cells, for instance. Novartis’ drug, Kymriah, comes with a $475,000 list price. However, that cost only kicks in if the treatment shows signs of working within the first month. Creative reimbursement arrangements like that may become the wave of the future in an age when medical technology and prices alike stun the mind.
This essay appears in today’s edition of the Fortune Brainstorm Health Daily. Get it delivered straight to your inbox.