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Drug Of The Week: A Blockbuster Orphan Drug

posted by Alicia Caramenico

on February 5, 2016

The latest Drug of the Week installment calls attention to a growing problem that’s putting pharmaceutical profits ahead of patient access: abuse of the Orphan Drug Act.

Instead of encouraging the development of treatments that will help patients with rare diseases, pharmaceutical companies are seeking “orphan” status to develop blockbuster drugs used to treat other common medical conditions. They reap the lucrative benefits of orphan status – subsidies, tax credits, and waivers – while generating billions of dollars in profits.

This exploitation is what enables Rituxan to be considered an orphan drug while also being the 12th best-selling medication in the United States.

Originally approved as an orphan drug for follicular B-cell non-Hodgkin’s lymphoma, Rituxan is now used to treat several hematologic cancers including chronic lymphocytic leukemia, several autoimmune diseases including rheumatoid arthritis, and organ rejection following kidney transplantation. More approved indications mean more sales – which is how orphan drug Rituxan has become one of the top-selling drugs of all time.