Today’s a big day for Novartis

Novartis spent $8.7 billion on the belief that a gene therapy for spinal muscular atrophy could be a life-changing product for patients. So far, the science has worked out fine. Now the question is: How much is such a revolutionary drug worth?

Later today, the Institute for Clinical & Economic Review is expected to issue a report on the cost-effectiveness of that gene therapy, called AVXS-101. The group, abbreviated ICER, doesn’t have any broad authority or regulatory power, but its conclusions have a way of becoming cudgels in negotiations between manufacturers and payers over just how much a new therapy should cost.

And AVXS-101 is no average drug. In a small trial, 100 percent of patients were alive after 24 months, a stunning result in a disease that is most often fatal within two years of birth. Novartis, which hasn’t set a price for the therapy, believes it would be cost-effective at $4 million for a one-time dose. Today, ICER will begin a long, public conversation about what the world should pay for AVXS-101, with implications for the scores of companies — and billions of dollars — invested in gene therapy.