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Opinion
While it is easy to criticise different parties in these pricing debates, these are complex issues for which there are no easy answers.
ASSESSING THE VALUE OF NEW MEDICINES H
ow do you assign a value to saving someone’s life? Then, how do you pay the bill? Those are questions that manufacturers and health authorities are increasingly being asked to answer as pharmaceutical companies attempt to gain regulatory approval and subsequent reimbursement for innovative and, in some cases, curative new therapies; products with limited evidence and small patient populations.
Oliver Leatham – vice president and global head of value and access at Certara
On 24 May, the US Food and Drug Administration (FDA) approved Novartis’ Zolgensma, the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), a leading genetic cause of infant mortality. Zolgensma is a one-time treatment, which is designed to be curative, but it will cost more than $2 million, making it the most expensive drug ever brought to market in the US. While unprecedented, Novartis’ price for Zolgensma comes close to what the Institute for Clinical and Economics Review (ICER) estimated. By a measure of cost per life-years gained, ICER judges an appropriate value-based price for Zolgensma to be between $1.2 million and $2.1 million, which seems to favour both the manufacturer and the health system.