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 In this section:

                Medical products for rare diseases and conditions

Medical products for rare diseases and conditions

                        


                        Designating an Orphan Product: Drugs and Biological Products
          FDA’s Overview of Catalyst Pharms., Inc. v. Becerra
          Rare Pediatric Disease (RPD) Designation and Voucher Programs
          Humanitarian Use Device (HUD) Designation Program
          Orphan Products Grants Program
          Pediatric Device Consortia Grants Program
          Personal Stories About Rare Diseases
          FDA's Rare Disease Day
          Education and Media Resources Home
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          Medical products for rare diseases and conditions
          Funding opportunities for rare disease researchMedical products for rare diseases and conditions Funding opportunities for rare disease research
  
  	
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    Print**Clinical Trials**

**Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)**Apply Here
Receipt Dates: October 22, 2024 (for new applications and resubmissions)
Resubmissions only 
Due Date(s): June 4, 2024; June 3, 2025 (for resubmissions only)
FOA Number: RFA-FD-23-001Users can also subscribe for automatic notifications ongrants.gov
 **Funding Opportunity Purpose:**
The purpose of this funding opportunity announcement is to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare diseases or conditions.  Additionally, through the funding of collaborative, efficient, and/or innovative clinical trials, FDA expects to increase the number of approved treatments for rare diseases and exert a broad and positive impact on rare disease drug development.

See instructions and helpful hints for applying for clinical trial grants.

**Natural History Studies:****Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01)**Apply Here 
Receipt Dates: February 13, 2024
FOA Number: RFA-FD-22-001Users can also subscribe for automatic notifications ongrants.gov
 **Funding Opportunity Purpose:**
The purpose of this funding opportunity announcement is to support efficient and innovative natural history studies that advance medical product development in rare diseases/conditions with unmet needs. Through the support of natural history studies with high quality and interpretable data elements, FDA expects to address critical knowledge gaps, remove major barriers to progress in the field, exert a significant and broad impact on a specific rare disease or multiple rare diseases with similar pathophysiology, and facilitate rare disease product development.

Seeinstructions and helpful hints for applying for natural history study grants.

**Rare Neurodegenerative Disease Grants Program Opportunities:** **Natural History and Biomarker Studies of Rare Neurodegenerative Diseases (U01)**Apply Here
Receipt Dates: May 6, 2024
FOA Number: RFA-FD-24-024Users can also subscribe for automatic notifications on grants.gov

**Funding Opportunity Purpose:**
The purpose of this funding opportunity announcement (FOA) is to support efficient natural history studies alone or in conjunction with the development and validation of clinical outcome assessments (COAs) and/or biomarker studies to address the unmet needs in rare neurodegenerative diseases for children and adults. Through the support of studies with high quality and interpretable data elements, FDA expects to address critical knowledge gaps, remove major barriers to progress in the field, exert a significant and broad impact on a specific rare neurodegenerative disease or multiple rare neurodegenerative diseases with similar pathophysiology, and facilitate rare disease product development.

See instructions and helpful hints for applying for natural history study grants.

                                                                                  Content current as of:
      12/12/2023
              
              
Topic(s)
			  ResearchMedical products for rare diseases and conditions

          
                        


                        Designating an Orphan Product: Drugs and Biological Products
          FDA’s Overview of Catalyst Pharms., Inc. v. Becerra
          Rare Pediatric Disease (RPD) Designation and Voucher Programs
          Humanitarian Use Device (HUD) Designation Program
          Orphan Products Grants Program
          Pediatric Device Consortia Grants Program
          Personal Stories About Rare Diseases
          FDA's Rare Disease Day
          Education and Media Resources
 
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