Klobuchar, Wicker, Bilirakis, Matsui Announce New FDA Rare Disease Innovation Hub

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

The Rare Disease Innovation Hub will serve as a single point of connection and engagement within the FDA to spur and support the development of treatments and products for rare diseases. The Hub 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). Klobuchar, Wicker, Bilirakis, and Matsui have led the Rare Disease Congressional Caucus in calling on the FDA to improve and centralize the process of reviewing rare disease therapies.

"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. "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 the experience of patients living with rare diseases."

"The FDA approval process for new treatments is a complicated and time-consuming process, leaving patients with rare diseases in an untenable spot and their voices unheard. Not only will this hub give them better access to resources and medication options, but it will also provide them with hope. Milestones like this one make me proud to be a co-chair of the caucus," said Wicker.

"The Rare Disease Innovation Hub is an important step forward in providing hope to the millions of Americans suffering with a rare disease," said 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 of 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."

"For the millions of Americans with rare diseases, any step towards receiving the most advanced medical care is a huge step in the right direction," said Matsui. "Rare disease patients face countless challenges to receiving the care they need, from a long diagnostic journey to the lack of FDA-approved medications specific to their condition. The Hub would provide alignment within the FDA in the critical points that impact patients the most, helping rare diseases get the attention they need to deliver life-changing solutions for patients. As Co-Chair of the Congressional Rare Disease Caucus, I know the Hub will ensure that the future is bright for patients with rare and ultra-rare diseases and their families."

"The EveryLife Foundation is grateful for the unwavering dedication of our Rare Disease Congressional Caucus leadership in working alongside scientific and policy leaders, as well as the thousands of rare disease community members whose experiences informed their collective advocacy," said Michael Pearlmutter, CEO of the EveryLife Foundation for Rare Diseases. "The FDA Rare Disease Innovation Hub is a positive and meaningful step forward in addressing the regulatory science challenges unique to rare disease therapy development and will be instrumental in bringing approved therapies to patients faster."

"The FDA's Rare Disease Innovation Hub is an exciting step forward to help speed up the development of new treatments for rare diseases," said Pamela Gavin, CEO of the National Organization for Rare Disorders (NORD). "NORD is grateful to the Rare Disease Congressional Caucus for their long-standing leadership in ensuring the FDA's regulatory process can capitalize on this new era of science and technology. The more than 30 million Americans living with a rare disease are desperate for treatment options and this initiative provides new hope. NORD has been a steadfast partner to the FDA for decades and hopes the Hub is a continuation of this collaboration. This program further prioritizes patient input into the process at FDA, and we are thankful for the strategic thinking and leadership behind it."

In May 2023, Klobuchar, Wicker, Bilirakis, and Matsui led the Rare Disease Congressional Caucus in calling on the FDA to bring more reliability and consistency to the process of reviewing rare disease therapies. Following the lead of patients and advocates who had detailed how FDA divisions and offices can differ in how they treat applications for rare diseases, the lawmakers urged FDA to improve the reliability and consistency in how it reviews applications for rare disease products.

In March 2021, then-RDCC co-chairs Klobuchar, Wicker, Bilirakis, and former Representative G. K. Butterfield (D-NC) introduced the Speeding Therapy Access Today (STAT) Act of 2021, calling for the creation of an FDA Center of Excellence for Rare Diseases. The fiscal year 2023 omnibus included a provision of the STAT Act creating a grant program focused on regulatory science pertaining to products used to treat individuals with rare diseases.

In June 2020, Klobuchar, Wicker, Bilirakis, and Butterfield led the Rare Disease Congressional Caucus in calling on the FDA led a bipartisan group of lawmakers in calling on the FDA to create a Center of Excellence for Rare Diseases.

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