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 Takes Exciting Steps Toward Establishing the Rare Disease Innovation Hub
- FDA Rare Disease Innovation Hub
- 9 Things to Know About CDER’s Efforts on Rare Diseases
- FDA Voices: FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients
- CDER and CBER initiate the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program
- LEADER 3D: Learning and Education to Advance and Empower Rare Disease Drug Developers (PDF - 683 KB)
- Rare Diseases: Considerations for the Development of Drugs and Biological Products
- FDA Creates New Advisory Committee for Evaluation of Genetic Metabolic Disease Treatments
- CDER soliciting comments regarding clinical trial innovation
- FDA issues draft guidance regarding confirmatory evidence of clinical trials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 Approves New Drug to Treat Niemann-Pick Disease, Type C
- FDA Approves First Treatment for Niemann-Pick Disease, Type C
- FDA approves first drug for WHIM syndrome, a rare disorder that can lead to recurrent, life-threatening infections
- 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
- Opportunities to Improve Dose-Finding and Optimization for Rare Disease Drug Development - October 29, 2024
- Advancing Rare Disease Therapies Through an FDA Rare Disease Innovation Hub - October 16, 2024
- Natural History Studies and Registries in the Development of Rare Disease Treatments - May 13, 2024
- Advancing the Use of Complex Innovative Designs in Clinical Trials: From Pilot to Practice – March 5, 2024
- Public Meeting on Advancing the Development of Therapeutics Through Rare Disease Patient Community Engagement - December 14, 2023
- FDA CDER and CBER & Duke-Margolis Center for Health Policy | 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
2024 Annual Report
Read about how the program is driving innovation through scientific and regulatory advancement
Introduction Video
Watch Dr. Kerry Jo Lee, Associate Director for Rare Diseases, share more about the vision and mission of the program
LEADER 3D Initiative
Find educational resources for rare disease drug development
Funding Opportunities
Learn about available funding opportunities for rare disease product development
Rare Disease Endpoint Advancement Pilot Program
Learn how the program supports novel efficacy endpoint development
Rare Disease Cures Accelerator
Learn about effort to support innovation and quality in rare disease drug development
Featured CDER Rare Disease Projects and Activities
Guidances for Rare Disease Drug Development
Review select guidance documents that are relevant to rare disease drug development, organized by topic
Lysosomal Diseases C-Path Pre-Consortium
Learn about partnership to address unmet needs for patients living with lysosomal diseases and how to get involved
CDER Rare Diseases Team
Learn about CDER’s Rare Diseases Team and how it supports the development of drugs for patients with rare diseases
Act for ALS
Learn about FDA’s Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis (ALS)