Senate accelerates rare disease treatments for 15 million children
This compromise was led by Sens. Johnny Isakson (R-GA), Robert Casey (D-PA) and Lamar Alexander (R-TN). As a result, the program has been extended until the end of this year; previously, it was scheduled to expire at the end of September.
Members of the National Organization for Rare Disorders (NORD) organize the Rare Pediatric Disease PRV. It represents the U.S. Food and Drug Administration’s (FDA) program to encourage research and development concerning rare pediatric disease treatments.
Before passing this program, the senators tried to pass the Advancing Hope Act (S 1878) to reauthorize the program for another six years. This vote was unsuccessful, but the recent decision to lengthen the program included the policy changes in S 1878.
“The Advancing Hope Act changes the statutory definition for rare pediatric diseases because it takes into account morbidity and incidence rather than overly relying on prevalence data,” Peter Saltonstall, president and CEO of NORD, said. “NORD has pushed for this important change for nearly two years so that all deserving rare pediatric diseases are included.”