Pasco Reporter

WASHINGTON—U.S. Senators Amy Klobuchar (D-MN) and Roger Wicker (R-MS), along with U.S. Representatives Gus Bilirakis (R-FL) and Doris Matsui (D-CA), co-chairs of the bipartisan Rare Disease Congressional Caucus (RDCC), have welcomed the Food and Drug Administration's (FDA) announcement of a new Rare Disease Innovation Hub.

The Rare Disease Innovation Hub will act as a centralized point within the FDA to support the development of treatments for rare diseases. It will be co-led by the Directors of the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER). The RDCC leaders have been advocating for improved processes in reviewing rare disease therapies.

“The Rare Disease Innovation Hub is an important step forward in providing hope to the millions of Americans suffering with a rare disease,” said Congressman Bilirakis. “As Co-Chairs of the Rare Disease Caucus, we have long called on the FDA to improve its policies, streamline processes, and strengthen intercenter collaboration efforts to accelerate the development of treatments and cures for rare diseases. I am glad to see the agency do the right thing for the rare disease community by recognizing the unique challenges of rare development and the need for a new innovative approach to review therapies. I look forward to working closely with my colleagues in the Caucus and with the FDA as it implements this new Hub to ensure its success for patients.”

“Rare diseases are not a rare problem. Over 30 million Americans live with a rare disease but most have no FDA-approved treatment available,” said Klobuchar, co-chair of the Rare Disease Congressional Caucus. “Too often, applications for new and innovative products for ultra-rare diseases get bogged down or lost due to misunderstanding or needless red tape. The new Rare Disease Innovation Hub is a welcome step forward to provide a more transparent process for applicants by consistently incorporating expertise and best practices across the agency as well as experience from patients living with rare diseases.”

“For millions of Americans with rare diseases, any step towards receiving advanced medical care is significant,” said Congresswoman Matsui. “Rare disease patients face countless challenges in receiving care, from long diagnostic journeys to lack of FDA-approved medications specific to their condition. The Hub would provide alignment within critical points at FDA that impact patients most, helping deliver life-changing solutions. As Co-Chair of the Congressional Rare Disease Caucus, I know this initiative will ensure a brighter future for patients with rare diseases.”

“The EveryLife Foundation is grateful for our Rare Disease Congressional Caucus leadership’s dedication alongside scientific and policy leaders, as well as thousands from our community whose experiences informed their advocacy,” said Michael Pearlmutter, CEO of EveryLife Foundation for Rare Diseases. “The FDA Rare Disease Innovation Hub addresses regulatory science challenges unique to therapy development in this field and will help bring approved therapies faster."

“The FDA’s Rare Disease Innovation Hub is an exciting step forward,” said Pamela Gavin, CEO of National Organization for Rare Disorders (NORD). “NORD appreciates RDCC’s leadership ensuring that FDA’s regulatory process can leverage modern science and technology advances. With over 30 million Americans living with a rare disease desperate for treatment options, this initiative provides new hope."

In May 2023, Klobuchar, Wicker, Bilirakis, and Matsui led RDCC in urging FDA improvements in reviewing rare disease therapies following feedback on inconsistent application reviews across divisions.

In March 2021, then-RDCC co-chairs introduced Speeding Therapy Access Today (STAT) Act calling for an FDA Center of Excellence for Rare Diseases; fiscal year 2023 omnibus included related grant program provisions.

In June 2020, they led calls urging creation of such a center.

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