Here is an interesting article on rare disease patients and families, written by By Peter L. Saltonstall, President and CEO of National Organization for Rare Disorders (NORD) about developments around the FDA Safety and Innovation Act.
Here is a short excerpt for a brief preview of what you’ll find in the article:
“Of particular importance to NORD and the 30 million Americans with rare diseases it represents are the provisions related to:
§ Accelerated patient access to new medical treatments
§ Resolution of conflict-of-interest provisions introduced in the previous PDUFA reauthorization
§ Accelerated development of “breakthrough therapies” that show early promise
§ Enhanced FDA consultation with rare disease medical experts
§ A rare pediatric disease priority review voucher incentive program and
§ The development of Humanitarian Use Devices (medical devices for small patient populations)”
“The stakes remain high for rare disease patients and patient organizations. Of the nearly 7,000 diseases considered rare (“rare” is defined as affecting fewer than 200,000 Americans), only about 250 have FDA-approved therapies.”For more information about NORD and more news on the FDA Safety and Innovation Act, visit the Web sites by clicking the links above.