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FDA CDER’s Accelerating Rare disease Cures (ARC) Program

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In 2022, the FDA’s Center for Drug Evaluation and Research (CDER) launched the Accelerating Rare disease Cures (ARC) Program to help bridge the gap between the urgent needs of patients and the complexities of rare disease drug development.

ARC’s mission is to accelerate the availability of safe and effective therapies by promoting scientific innovation, enhancing understanding of regulatory policies, sharing insights gathered from all rare disease programs across CDER, and fostering engagement with patients, their advocates, and other rare disease stakeholders. By harnessing the collective knowledge and efforts of CDER, ARC has implemented several initiatives to actively expand the agency’s knowledge base in rare disease space while providing a strategic framework of CDER’s rare disease activities.

ARC Program | Year One Initiatives

In the first year of the ARC Program (May 2022-May 2023), CDER prioritized enhancing engagement and collaboration with rare disease stakeholders, recognizing their essential role in advancing the development of treatments. Thus, CDER cultivated a robust network of patients, their advocates, drug developers, and researchers and launched the Learning and Education to Advance and Empower Rare Disease Drug Developers (LEADER 3D) initiative to better understand the challenges in bringing rare disease treatments to market. LEADER 3D prioritized engaging stakeholders who design and conduct drug development programs and identifying complex regulatory topics that would benefit from expanded or additional educational materials.

Other patient-focused and scientific efforts of the ARC Program included:

  • Conducting patient listening sessions to share and gather valuable insights and perspectives on rare diseases while simultaneously providing patients, families, and their caregivers with the opportunity and tools to gain deeper knowledge of the drug development and regulatory process. 
  • Collaborating with CDER’s Patient-Focused Drug Development Program (PFDD) to expand resources tailored to patients.
  • Collaborating with other crucial FDA resources such as the Rare Disease Endpoint Advancement (RDEA) Pilot Program.
  • Participating in conferences, workshops, and external speaking engagements to share regulatory perspective and knowledge on topics, such as real-word data (RWD) in rare disease drug development and complex innovative design approaches.

ARC Program | Year Two Goals

In the second year, the ARC Program is set to broaden its scope to provide more scientific and regulatory guidance and tools to promote rare disease drug development. This entails a greater focus on translational medicine approaches for individual rare diseases and will include ongoing regulatory research in confirmatory evidence and surrogate biomarkers. Simultaneously, the ARC program will work to improve frameworks, including complex innovative methodologies such as Bayesian statistical approaches, which have the potential to increase trial efficiencies in rare disease research. Additionally, ARC will support platforms that enable rigorous use of data from natural history studies and other real-world data (RWD).

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