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.
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To learn more about CDER’s ARC Program and its role in CDER’s rare disease activities, please read CDER Launches New Accelerating Rare disease Cures (ARC) Program.
- FDA Launches AATD Pre-Consortium Partnership with the Critical Path Institute
- Rare Disease Endpoint Advancement Pilot Program
- FDA and NIH Launch Public-Private Partnership for Rare Neurodegenerative Diseases
- FDA initiates the Lysosomal Diseases Pre-Consortium at the Critical Path Institute
- Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis
- 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
- 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
Rare Disease Endpoint Advancement (RDEA) Pilot Program
Learn how the program supports novel endpoint efficacy development for drugs that treat rare diseases
Lysosomal Diseases C-Path Pre-Consortium
Learn about partnership to address unmet needs for patients living with lysosomal diseases and how to get involved
Act for ALS
Learn about FDA’s Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis (ALS)
CDER Rare Diseases Team
Learn about CDER’s Rare Diseases Team and how it supports the development of drugs for patients with rare diseases
LEADER 3D Docket Now Open for Comments
Provide feedback on development and dissemination of educational materials on rare disease drug development
CDER Patient-Focused Drug Development
Learn about CDER's work to better incorporate the patient’s voice in drug development and evaluation