Accelerating Rare disease Cures (ARC) Program
CDER’s ARC Program | Center for Drug Evaluation and Research
CDER’s Accelerating Rare disease Cures (ARC) Program brings together CDER’s collective expertise and activities to provide strategic overview and coordination of CDER’s rare disease activities. The 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.
Vision: Speeding and increasing the development of effective and safe treatment options addressing the unmet needs of patients with rare diseases.
Mission: To drive scientific and regulatory innovation and engagement to accelerate the availability of treatments for patients with rare diseases.
Connect with us!
CDER_ARC_Program@fda.hhs.gov
- FDA issues draft guidance regarding confirmatory evidence of clinical trials
- Funding Opportunities for Rare Diseases at FDA
- CDER Continues to Advance Rare Disease Drug Development with New Efforts, Including the Accelerating Rare Disease Cures (ARC) Program
- Guidance: Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products
- 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
- FDA Voices: CDER Continues to Make Rare Diseases a Priority with Drug Approvals and Programming to Speed Therapeutic Development
- FDA approves first treatment for CD55-deficient protein-losing enteropathy (CHAPLE disease)
- FDA approves first treatment for Fibrodysplasia Ossificans Progressiva
- FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene
- FDA approves first treatment for activated phosphoinositide 3-kinase delta syndrome
- FDA approves first treatment for Friedreich’s ataxia
- FDA approves first enzyme replacement therapy for rare alpha-mannosidosis
- 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
- Advancing the Use of Complex Innovative Designs in Clinical Trials: From Pilot to Practice – March 5, 2024
- FDA and Duke-Margolis Public Workshop: Rare Disease Endpoint Advancement Pilot Program Workshop: Novel Endpoints for Rare Disease Drug Development – June 7-8, 2023
- FDA CDER & M-CERSI Workshop | Creating a Roadmap to Quantitative Systems Pharmacology-Informed Rare Disease Drug Development - May 11, 2023
- FDA CDER & JHU CERSI Workshop | Addressing Challenges in the Design and Analysis of Rare Disease Clinical Trials: Considerations and Tools - May 2-3, 2023
- FDA and Duke-Margolis Public Workshop: Translational Science in Drug Development: Surrogate Endpoints, Biomarkers, and More - May 24-25, 2022
- FDA CDER & NIH NCATS Regulatory Fitness in Rare Disease Clinical Trials Workshop - May 16-17, 2022
- 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
- New Drug Therapy Approvals- 2021
Featured CDER Rare Disease Projects and Activities
Featured CDER Rare Disease Projects and Activities
Funding Opportunities
Learn about available funding opportunities for rare disease product development research
Rare Disease Cures Accelerator
Learn about effort to support innovation and quality in rare disease drug development
Rare Disease Endpoint Advancement (RDEA) Pilot Program
Learn how the program supports novel endpoint efficacy development for drugs that treat rare diseases
Guidances for Rare Disease Drug Development
Review selected guidances that are relevant to rare disease drug development, organized by topic
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
