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
- LEADER 3D: Learning and Education to Advance and Empower Rare Disease Drug Developers (PDF - 683KB)
- 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- FDA issues draft guidance regarding confirmatory evidence of clinical trials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 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
- 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
![ARC Anniversary Update](https://public4.pagefreezer.com:443/content/FDA/15-03-2024T15:32/https://www.fda.gov/files/styles/medium_3/public/OND_ARC_AnniversaryUpdate_1600x900_2306122.png?itok=OrLRyv5z)
Anniversary Update
Read about how the program is driving innovation through collaboration and engagement with rare disease stakeholders
![Kerry Jo Lee, M.D.](https://public4.pagefreezer.com:443/content/FDA/15-03-2024T15:32/https://www.fda.gov/files/styles/medium_3/public/ARC_IntroVideoThumb_1920x1080_230602_1340.png?itok=ErUnuAjO)
Introduction Video
Watch Dr. Kerry Jo Lee, Associate Director for Rare Diseases, share more about the vision and mission of the program
![Rare Diseases spelled in wood blocks](https://public4.pagefreezer.com:443/content/FDA/15-03-2024T15:32/https://www.fda.gov/files/styles/medium_3/public/d11_cover_image_edited_resized_3.png?itok=eEPUBRgu)
LEADER 3D Public Report of External Engagement Analysis
Read a summary of recommendations from the drug development community for continued efforts to expand outreach
![Pharmacist talking with mother and child](https://public4.pagefreezer.com:443/content/FDA/15-03-2024T15:32/https://www.fda.gov/files/styles/medium_3/public/OND_ARDWeb_RDEA.png?itok=vnXH7X0Y)
Rare Disease Endpoint Advancement Pilot Program
Learn how the program supports novel efficacy endpoint development for drugs that treat rare diseases
![Hand with lightbulb and hand with money symbol](https://public4.pagefreezer.com:443/content/FDA/15-03-2024T15:32/https://www.fda.gov/files/styles/medium_3/public/ARC_WebBanners_FundingOpportunities_1600x900px.png?itok=kdUXl0kv)
Funding Opportunities
Learn about available funding opportunities for rare disease product development
![Rare Diseases spelled in wood blocks](https://public4.pagefreezer.com:443/content/FDA/15-03-2024T15:32/https://www.fda.gov/files/styles/medium_3/public/d11_cover_image_edited_resized_3.png?itok=eEPUBRgu)
Rare Disease Cures Accelerator
Learn about effort to support innovation and quality in rare disease drug development
Featured CDER Rare Disease Projects and Activities
![Graphic with teal and dark blue background. Text on the left of the graphic in white letters reads Guidance for Industry.](https://public4.pagefreezer.com:443/content/FDA/15-03-2024T15:32/https://www.fda.gov/files/styles/medium_3/public/standard_guidance_graphic.png?itok=4LzZoS3w)
Guidances for Rare Disease Drug Development
Review select guidance documents that are relevant to rare disease drug development, organized by topic
![Lysosomal Disease](https://public4.pagefreezer.com:443/content/FDA/15-03-2024T15:32/https://www.fda.gov/files/styles/medium_3/public/OND_ARDWeb_Lysosomal%20Disease%20C-Path.png?itok=oZCtFqE3)
Lysosomal Diseases C-Path Pre-Consortium
Learn about partnership to address unmet needs for patients living with lysosomal diseases and how to get involved
![Man lecturing while people sit at desk](https://public4.pagefreezer.com:443/content/FDA/15-03-2024T15:32/https://www.fda.gov/files/styles/medium_3/public/OND_ARDWeb_Rare%20Disease%20Team.png?itok=UBML6AcT)
CDER Rare Diseases Team
Learn about CDER’s Rare Diseases Team and how it supports the development of drugs for patients with rare diseases
![Brain](https://public4.pagefreezer.com:443/content/FDA/15-03-2024T15:32/https://www.fda.gov/files/styles/medium_3/public/OND_ARDWeb_Act%20for%20ALS.png?itok=chWFRDUl)
Act for ALS
Learn about FDA’s Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis (ALS)