Novartis Defends Price Tag of Its Million-Dollar Gene Therapy

In advance of a key report from the Institute for Clinical and Economic Review (ICER) – a nonprofit that assesses the economic benefits of new medicines – Novartis is preparing payers and policymakers for sticker shock over the price of its forthcoming gene therapy. AVXS-101, a treatment for spinal muscular atrophy, will cost $4 million per patient, which the company considers cost-effective.

In clinical trials, a single dose of AVXS-101 corrected a defective gene that causes spinal muscular atrophy. In a recent interview with The Financial Times, Novartis CEO Vas Narasimhan said the company is “brainstorming” alternative payment models for curative gene therapies. One option involves a third party underwriting “the catastrophic case of a child having one of these conditions,” he told the paper.

A final version of the ICER report, which can be crucial in winning over decision makers at health plans, will be available in March 2019.

Sources: The Financial Times, December 16, 2018; STAT News, December 17, 2018.