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        Center for Drug Evaluation and Research | CDER

Accelerating Rare disease Cures (ARC) Program Home
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          Center for Drug Evaluation and Research | CDER
          Accelerating Rare disease Cures (ARC) ProgramCenter for Drug Evaluation and Research | CDER Accelerating Rare disease Cures (ARC) Program
  			CDER’s ARC Program | Center for Drug Evaluation and Research
	
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    PrintCDER’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

CDER Rare Disease News

What’s New in Rare Disease
	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	FDA issues draft guidance regarding confirmatory evidence of clinical trials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

Rare Disease Drug Approvals
	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

Upcoming and Recent Events
	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

Other Rare Disease Resources
	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
Sub-Topic Paragraphs
          Featured CDER Rare Disease Projects and ActivitiesARC Anniversary Update

                    Anniversary Update
            Read about how the program is driving innovation through collaboration and engagement with rare disease stakeholders
          
          Kerry Jo Lee, M.D.

                    The ARC Program Introduction Video
            Watch Dr. Kerry Jo Lee, Associate Director for Rare Diseases, share more about the vision and mission of the program
          Featured CDER Rare Disease Projects and ActivitiesHand with lightbulb and hand with money symbol

                    Funding Opportunities
            Learn about available funding opportunities for rare disease product development research
          
          Person typing on laptop showing graphics

                    Rare Disease Cures Accelerator
            Learn about effort to support innovation and quality in rare disease drug development
    
          Pharmacist talking with mother and child

                    Rare Disease Endpoint Advancement (RDEA) Pilot Program
            Learn how the program supports novel endpoint efficacy development for drugs that treat rare diseases
    
          Guidance for Industry

                    Guidances for Rare Disease Drug Development
            Review selected guidances that are relevant to rare disease drug development, organized by topic
    
          Brain

                    Act for ALS
            Learn about FDA’s Action Plan for Rare Neurodegenerative Diseases including Amyotrophic Lateral Sclerosis (ALS)
    
          Man lecturing while people sit at desk

                    CDER Rare Diseases Team
            Learn about CDER’s Rare Diseases Team and how it supports the development of drugs for patients with rare diseases
          Featured CDER Rare Disease Projects and ActivitiesLysosomal Disease

                    Lysosomal Diseases C-Path Pre-Consortium
            Learn about partnership to address unmet needs for patients living with lysosomal diseases and how to get involved
          
          A woman is sitting in an audience composed of several people and she is raising her hand.

                    CDER Patient-Focused Drug Development
            Learn about CDER's work to better incorporate the patient’s voice in drug development and evaluation

                                                                                  Content current as of:
      01/19/2024Accelerating Rare disease Cures (ARC) Program
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