Drugmakers are manipulating the Orphan Drug Act -- which offers lucrative financial incentives in order to motivate pharmaceutical companies to develop rare disease drugs -- to maximize profits and to protect niche markets, says a Kaiser Health News investigation.
KHN’s investigation found that about a third of orphan approvals by the FDA since the program began in 1983 have been either for repurposed mass market drugs or drugs that received multiple orphan approvals.
Also, the report found that the seven-year exclusivity granted to orphan drug approvals is being used as a pricing tool. "Drugmakers can charge whatever they want by shielding their medicine from competition. The market exclusivity granted by the Orphan Drug Act can be a vital part of the protective shield that companies create. What’s more, manufacturers can return to the FDA with the same drug again and again, each time testing the drug against a new rare disease," says the report.
Read the full KHN article