FDA offers an update and reveals its intentions towards the ARC program

The Accelerating Rare Disease Cures (ARC) Program, a collaborative initiative launched by the Center for Drug Evaluation and Research (CDER) a year ago, is making significant strides in its mission to bring more therapies to market for the thousands of rare diseases that currently have no treatment.

The ARC Program is a joint effort between several offices within CDER, including the Office of the Center Director, Office of New Drugs, Office of Communications, and Office of Translational Sciences. In its first year, the program has successfully promoted creative scientific design, helped stakeholders understand regulatory policies, and engaged with patients, advocates, and other community members.

One of the key initiatives of the ARC Program is the Learning and Education to Advance and Empower Rare Disease Drug Developers (LEADER 3D). Through LEADER 3D, the program plans to create and disseminate educational materials to better understand the challenges in rare disease development. These challenges include a poor understanding of the disease process, research funding, clinical trial design and participation, and access to sufficient patient data.

To address these challenges, the ARC Program has fortified stakeholder partnerships and engaged with external experts. For instance, CDER 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 ARC Program is also working with the National Organization for Rare Disorders to create an advanced treatment development education series for patients.

Moreover, the ARC Program is collaborating with the Critical Path Institute on three projects having to do with treatment development for inherited metabolic disorders, neurodegenerative diseases, and alpha-1 antitrypsin deficiency. The program will share knowledge gained by using novel endpoints for potential rare disease therapies in the FDA's RDEA pilot program.

In addition, the ARC Program aims to further empower stakeholders, including patients, patient advocates, scientists, and biopharmaceutical companies, to harness their collective expertise and experiences to promote progress. To this end, the program will join patient listening sessions with rare disease advocates and partner with CDER's Patient-Focused Drug Development Program to create patient materials.

The LEADER 3D initiative has also led to identifying complex regulatory topics that require further exploration and participation in public workshops. The ARC Program's ultimate goal is to bring more rare disease therapies to market, as about 95% of the 7,000 known rare disorders have no treatment.

By working together, the ARC Program and its partners hope to accelerate the development of treatments for rare diseases, improving the lives of countless individuals affected by these conditions.