In honor of Rare Disease Day on February 28, 2025, we will publish a series of posts throughout the month on As Prescribed and Health Law Scan, focusing on issues impacting the rare disease community.
In response to calls from industry stakeholders for increased innovation, coordination, and tailored regulatory approaches to the development of treatments in rare disease, in recent years the US Food and Drug Administration (FDA) has established a number of rare disease-focused programs within the agency. Building on other recent FDA initiatives such as the Center for Drug Evaluation and Research (CDER) Accelerating Rare disease Cures (ARC) Program and the Center for Biologic Evaluation and Research (CBER) Rare Disease Program, FDA expanded its rare disease toolkit to include its Rare Disease Innovation Hub in 2024 to serve as a point of collaboration between CDER and CBER with the overarching goal of enhancing collaboration across centers to improve patient outcomes and addressing common challenges in drug development for rare diseases.
The Rare Disease Innovation Hub recently published its strategic agenda, providing stakeholders with insight into where we might see FDA focus in the rare disease space in 2025 and beyond. The Hub’s strategic agenda describes goals to strengthen coordination, improve regulatory science, and create a more centralized communication channel for stakeholders in the rare disease community in order to facilitate development of treatments for rare diseases.
Two Programs and a Hub
The Hub joins other recent developments in the space, including CDER’s ARC Program and CBER’s Rare Disease Program, as well as the perennial FDA focal point for rare disease, the Office of Orphan Products. While stakeholders have called for greater attention in this space from FDA, it remains to be seen whether this proliferation of programs within the agency will meet the needs of rare disease development programs and effectively collaborate within the already complex structure of the agency. While CDER’s ARC Program and CBER’s Rare Disease Programs are focused on coordinating activities within each respective agency center, the Hub may be aimed as a platform to enhance needed cross-center collaboration.
Per FDA, the Hub plans to focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood. The Hub is co-led by the directors of CDER and CBER in close collaboration with FDA’s Center for Devices and Radiological Health (CDRH), Oncology Center of Excellence, Office of Orphan Products Development, and Office of Combination Products.
Key Goals of the Hub
The Hub’s strategic agenda describes three key goals and corresponding actions the Hub plans to take in 2025. These goals were established based on feedback the agency received from the rare disease community through public meetings. The key goals that FDA describes for the Hub are (1) further advancing regulatory science for rare disease therapies, (2) enhancing and strengthening coordination between medical product centers, and (3) creating a centralized point of contact for external stakeholders.
- Advancing regulatory science for rare disease therapies: Per FDA, the Hub plans to develop multipartner education and engagement opportunities for drug developers, FDA reviewers and staff, patient organizations, federal agencies, and researchers to educate on novel approaches to therapy development for rare diseases. The Hub will launch a pilot for a Rare Disease Innovation, Science, and Exploration (RISE) workshop series, aiming to address scientific and regulatory challenges common across rare diseases. The first workshop will focus on designing clinical trials where the population is a small and diminishing population of eligible trial participants. Potential topics include the use of alternatives to placebo (e.g., historical controls and natural history studies) and utilization of adaptive trial designs. Future workshops will be selected based on proposals submitted by the rare disease community.
- Enhancing coordination between medical product centers: FDA plans for the Hub to work to put in place best practices that promote drug review information sharing opportunities and facilitates communication between CDER and CBER reviewers. In addition, the Hub will build on the launch of the Rare Disease Policy and Portfolio Council (RDPPC), a senior level forum to promote cross-center dialogue on rare disease product development.
- Creating a centralized point of contact for stakeholders: Reacting to stakeholder feedback regarding the challenges associated with navigating the variety of FDA programs dedicated to rare disease drug development coupled with a lack of a clear point of entry for those programs, FDA plans for the Hub to develop a website providing stakeholders with information on FDA’s rare disease programs to help guide them to through the agency’s programs. The Hub also plans to continue direct engagement with stakeholders through public meetings.
Looking Ahead
In 2025 and beyond, the Hub is poised to enhance stakeholder engagement to ensure the agency is attuned to the needs of the rare disease community. Transparency, innovation, and effective coordination within the agency on these matters will likely be key to—and key measures of—the success of this new FDA program. As the initiative unfolds, stakeholders should continue to engage with the agency and provide input to help shape the future of rare disease drug development at FDA. Rare disease applicants and stakeholders should stay attuned to FDA’s evolving efforts to support rare disease drug development to be poised to make the most of critical agency interactions.
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