The High Cost of Hope: When the Parallel Interests of Pharma and Families Collide

September 7, 2018

(Kaiser Health News) – Manufacturers selling precious, lifesaving medicines and patients share an uneasy alliance. They need each other but have clashing priorities, especially when it comes to drugs treating rare diseases such as cystinosis. Cystinosis families say they are deeply grateful for Procysbi. The medicine would not be on the market without Raptor — which performed clinical trials — and Horizon Pharma, which acquired the rights two years ago, they acknowledge. But they also feel they’ve been used. What began as a desperate search for life-extending medicine, cystinosis parents say, has become a story of corporations profiteering off their children.

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