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The Orphan Drug Act (ODA), enacted 40 years ago, has significantly advanced the development of treatments for rare diseases, yet 95% of known rare diseases still lack FDA-approved therapies. While the ODA has faced criticism for high drug prices, advocates argue that the focus should be on broader drug pricing policies rather than reducing the incentives provided by the ODA. Continued advocacy from patients and families is crucial to maintain the integrity of the ODA and support ongoing investment in rare d

Author

shailendra45yadav2002

Language

EN