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A Drug Was Approved for a Fatal Childhood Disease--But Costs $700,000

The FDA approved a new $700,000 drug to treat CLN2, a form of Batten disease, a neurodegenerative condition typically fatal before a child exits their teen years. As a result of the Orphan Drug Act, this and other rare disease drugs are subject to skyrocketing prices based on legal monopolies and corrupt sales practices by the drug companies.

A Drug Was Approved for a Fatal Childhood Disease--But Costs $700,000

[DIGEST: Bloomberg, Fortune, fda.gov, BizJournals, Biospace; FDA)

The FDA approved a new drug for a form of Batten disease, a rare inherited neurodegenerative condition which rarely allows children to live past their teens. But the drug retails at over $700,000 per year. This, and other drugs that treat rare diseases, are protected by what is known as the Orphan Drug Act. Originally created to encourage research and production of treatments for rare diseases, the Act has inadvertently produced monopolies that allow prices to skyrocket out of control, and corruption among drug companies as they try to push more product onto a tiny market.

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