The FDA Safety and Innovation Act
The FDA Safety and Innovation Act
...was signed this week thanks to the APF and other rare disease organizations.
This is a monumental step toward the development of safe and effective treatments for millions of Americans with rare diseases. Specific to the rare disease community, the Act provides the following:
- accelerated patient access to new medical treatments
- the development of Humanitarian Use Devices (medical devices for small patient populations)
- accelerated development of "breakthrough therapies" -- those that show early promise
- enhanced FDA consultation with rare disease medical experts
- a rare pediatric disease priority review voucher incentive program
- resolution of conflict-of-interest issues that kept voices of rare disease medical experts from being heard
This contains the most groundbreaking measures for rare disease patients and their families since the Orphan Drug Act of 1983. This legislation represents great progress for people with rare diseases, who often struggle to access treatments for their particular disorder, like porphyria. APF Executive Director, Desiree Lyon Howe, said about the new act, "Our thirty years as advocates enabled the APF to identify and articulate the unique needs of people with rare diseases for interested legislators." Desiree was one of the pioneers and supporters of the Orphan Drug Act and has been involved in rare disease legislation for 30 years.
Remember.... Research is the key to your cure!
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