U.S. Sen. Roger Wicker (R-MS) on March 10 introduced a bipartisan, bicameral bill that would amend the Federal Food, Drug, and Cosmetic Act to accelerate development of therapies across the spectrum of rare diseases and conditions that affect millions of Americans.
Sen. Wicker cosponsored the Speeding Therapy Access Today (STAT) Act of 2021, S. 670, with bill sponsor U.S. Sen. Amy Klobuchar (D-MN), which also would facilitate patient access to such therapies, according to the congressional record bill summary.
If enacted, S. 670 would improve rare disease coordination, stakeholder engagement, and policy development within the Food and Drug Administration (FDA) by expanding existing authority to create a Rare Disease Center of Excellence, and would create a Rare Disease and Condition Drug Advisory Committee, according to a bill summary provided by Sen. Wicker’s office.
“Millions of Americans are living with one or more rare diseases, the majority of which do not have FDA approved treatments,” Sen. Wicker said. “Creating a Rare Disease Center of Excellence would accelerate the development of therapies for rare diseases and disorders at the FDA while putting patient input first. This would be an important step forward as we work to find cures for all diseases.”
S. 670 also would fund regulatory science and related activities to support the development of therapies to treat very small rare disease populations, the summary says.
Rep. Klobuchar noted that 30 million Americans are impacted by one of 7,000 known rare diseases, but nearly 93 percent of those diseases have no treatment that has been approved by the FDA.
Julia Jenkins, executive director of the EveryLife Foundation for Rare Diseases, endorsed the bill and thanked the lawmakers for their efforts. “We are grateful for their leadership, and we look forward to working with them to ensure their innovative vision and approach to catalyzing treatments and cures for Americans with rare diseases becomes a reality,” she said.
U.S. Reps. Gus Bilirakis (R-FL) G.K. Butterfield (D-NC) on March 10 introduced companion legislation, H.R. 1730, in the U.S. House of Representatives.