Viewpoint: Outrage over $2 million price tag for new gene therapy is perplexing and disappointing

6-3-2019 travel lnp photo
Image: Cure SMA

As someone who has lived with spinal muscular atrophy for all 30 years of my life, I was perplexed and disappointed that the recent approval of Novartis’ gene therapy Zolgensma was immediately overshadowed by outrage over the drug’s price: $2.125 million.

How are we going to get treatments for rare diseases if there’s not a financial incentive for doing it?

As a professor of economics and finance, I believe that the cost-related complaints being thrown around social media are short-sighted. Shortly after Zolgensma’s price was announced, I even told a friend, “This is a good problem to have.” Why? It’s a twofold answer:

Competition. Zolgensma is the second treatment approved for SMA, following Biogen’s Spinraza. Now that there’s competition in the marketplace, that will lead to lower prices over the long term.

Related article:  CRISPRcon talks designer babies and scientific colonialism

Long-term value. The first two years of treatment with Spinraza cost around 50% of one Zolgensma infusion, but Spinraza treatments must continue for life at a cost of $375,000 each year. The four initial loading doses of Spinraza in the first year of treatment total $750,000. Over a 10-year period, the cost-effectiveness of Zolgensma is clear.

Read full, original post: I have spinal muscular atrophy. Critics of the $2 million new gene therapy are missing the point

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