FDA shares update, vision about its ARC program

Accelerating Rare disease Cures Program enters its second year

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by Mary Chapman |

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A year after it was established by the Center for Drug Evaluation and Research (CDER), the Accelerating Rare disease Cures (ARC) Program is seeking to build on efforts to bridge the gap between patients’ pressing needs and the challenges of developing rare disease treatments.

The U.S. Food and Drug Administration’s (FDA’s) CDER launched ARC with the overarching goal of bringing more rare disease therapies to market, since about 95% of the 7,000 known rare disorders have no treatment. The CDER-wide program is a collaboration of the Office of the Center Director, Office of New Drugs, Office of Communications, and Office of Translational Sciences, and is supported by other FDA offices and centers.

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In its first year, the program promoted creative scientific design, helping stakeholders understand regulatory policies, and engaging with patients, advocates, and other community members. According to CDER, it has helped empower patients, patient advocates, scientists, and biopharmaceutical companies to harness their collective expertise and experiences to promote progress.

To date, ARC has fortified stakeholder partnerships and engaged with external experts to help find solutions to challenges in rare disease development. Such impediments may include a poor understanding of the disease process, research funding, clinical trial design and participation, and access to sufficient patient data.

To better understand these challenges, the program launched the Learning and Education to Advance and Empower Rare Disease Drug Developers (LEADER 3D), which led to identifying complex regulatory topics that require further exploration and participation in public workshops.

“ARC’s emphasis on engagement and collaboration in our first year is affirmation of the essential role that all stakeholders must play to foster the development of safe and effective therapies,” Kerry Jo Lee, MD, associate director for rare diseases, Office of New Drugs, CDER, said on a webpage about ARC. “Through deliberate outreach, we have cultivated a robust network with patients and patient advocates, drug developers, and researchers.”

Empowering rare disease stakeholders

In its second year, ARC seeks to further empower stakeholders by:

  • creating and disseminating educational materials through the LEADER 3D initiative
  • partnering with CDER’s Patient-Focused Drug Development Program to create patient materials
  • sharing knowledge gained by using novel endpoints for potential rare disease therapies in the FDA’s Rare Disease Endpoint Advancement (RDEA) pilot program.
  • joining patient listening sessions with rare disease advocates.

CDER is working with the National Organization for Rare Disorders to create an advanced treatment development education series for patients. CDER also has partnered with the RDEA pilot program, which supports effectiveness goals or clinical outcome development, and the timely federal approval of rare disease treatments. The pilot program, which runs through Sept. 30, 2027, is for sponsors who have an active therapy development program or a natural history study of a rare disease such as Angelman syndrome, which is thought to affect one in 15,000 people.

CDER also is working with the Critical Path Institute, under the ARC program, on three projects having to do with treatment development for inherited metabolic disorders, neurodegenerative diseases, and alpha-1 antitrypsin deficiency.