Bill offers hope for patients with rare diseases


WASHINGTON — A bill passed in the Senate will provide measures to speed up the development of treatments for people who desperately need them.

The Senate voted Tuesday to pass S. 3187, the Food and Drug Administration Safety and Innovation Act, which now awaits President Barack Obama's signature. The House approved a similar bill last week.

Among other features, the bill enhances accelerated patient access to new medical treatments; encourages the development of medical devices for small patient populations; provides for accelerated development of therapies that show early promise; enhances consultation with medical experts on rare diseases; and creates a priority review voucher incentive system for rare children's diseases.

The legislation won praise from a group focused on rare diseases.

"Only about 250 of the nearly 7,000 diseases considered rare in the United States have therapies," National Organization for Rare Disorders president and CEO Peter Saltonstall said. "But treatments are desperately needed because most rare diseases are serious, many are life-threatening and about two-thirds of the patients are children."

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