• Orphan Products Grants Overview
• Sponsor-Investigator Roles and Responsibilities in Clinical Investigations for Drug and Biological Orphan Products
• Video Discussion on Natural History Studies
• Workshop on Natural History Studies in Rare Diseases: Meeting the Needs of Drug Development and Research
• How Rare Disease Patients Can Move Scientific Discovery Forward
• Natural History Video for Grant Applicants
• Patient Affairs Staff: Patients Matter Video Series

• CDER: About the Center for Drug Evaluation and Research
• CBER: About the Center for Biologics Evaluation and Research
• CDRH: About the Center for Devices and Radiological Health
• CFSAN: About the Center for Food Safety and Applied Nutrition

•  CBER: Guidance, Compliance & Regulatory Information (Biologics)
•  CDER: Guidances (Drugs)
•  CDRH: Guidance Documents (Medical Devices and Radiation-Emitting Products

• Application Guide: How to Apply NIH Resources
• FDA’s Role: ClinicalTrials.gov Information

IND

• Rare Diseases Early Drug Development and the Role of Pre IND Meetings
• IND Application Information and Procedures
• Information for Sponsor-Investigators Submitting Investigational New Drug Applications (INDs)
• Small Business and Industry Assistance: Frequently Asked Questions on the Pre-Investigational New Drug (IND) Meeting

Highlighted Guidances

• E6(R2) Good Clinical Practice: Integrated Addendum to ICH E6(R1)
• Good Clinical Practice
• Rare Diseases: Common Issues in Drug Development
• Rare Diseases: Early Drug Development and the Role of Pre-IND Meetings
• Rare Diseases: Natural History Studies for Drug Development