Launched in May 2022, CDER’s Accelerating Rare disease Cures (ARC) Program harnesses CDER’s collective expertise and activities to provide strategic overview and coordination of CDER’s rare disease activities. CDER’s ARC Program is governed by leadership from across CDER's Office of the Center Director, Office of New Drugs, and the Office of Translational Sciences. The program is managed by CDER's Rare Diseases Team.
Speeding and increasing the development of effective and safe treatment options addressing the unmet needs of patients with rare diseases.
CDER’s ARC Program drives scientific and regulatory innovation and engagement to accelerate the availability of treatments for patients with rare diseases.
- FDA Voices: CDER Continues to Make Rare Diseases a Priority with Drug Approvals and Programming to Speed Therapeutic Development
- FDA approves drug for treatment of seizures associated with rare disease in patients two years of age and older
- FDA approves drug for adults with rare form of bone marrow disorder
- FDA approves treatment for anemia in adults with rare inherited disorder
- FDA approves treatment for adults with rare type of anemia
- FDA Approves Treatment for Rare Blood Disease
- FDA approves first drug to decrease urine protein in IgA nephropathy, a rare kidney disease
- Guidances: Annotated Listing for Rare Diseases
- New Drug Therapy Approvals- 2021
- Emergency IND Application Timeline
- FDA’s Office of Orphan Products Development
- FDA Patient Engagement Opportunities
- For Physicians: How to Request Single Patient Expanded Access (“Compassionate Use”)
- Oncology Center of Excellence Rare Cancers Program
- Physician's Checklist for an IND Application for Emergency Treatment
- Rare Disease Cures Accelerator