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In advance of a key report, Novartis (NVS) is trying to prepare payers and policymakers for a forthcoming gene therapy that the company argues would be cost effective at $4 million to $5 million for each patient.

The drug maker held a media briefing on Monday to discuss a draft report due later this week from the Institute for Clinical and Economic Review on Zolgensma, which was developed to treat spinal muscular atrophy, a rare and inherited neurodegenerative disease that in its most fatal form causes children to die by the time they turn 2 years old. Trials showed a single dose corrected a defective gene.


In the last few years, ICER, which is a nonprofit, has become an unofficial force in the U.S. for assessing the economic benefits of new medicines with a series of reports that have compelled the pharmaceutical industry to rethink its approach to pricing. A final version of the report — which will also review the Spinraza SMA drug sold by Biogen (BIIB) — will not appear until March, but the draft is seen as crucial in winning over decision makers at health plans.

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