Program Scope

This training program covers the entire orphan drug development and approval process from rare disease discovery through preclinical development, clinical trials, NDA submission and FDA review, and post approval lifecycle management including maintenance of seven year orphan drug exclusivity.

You will learn the Orphan Drug Act of 1983, the step by step process for obtaining orphan drug designation from the FDA Office of Orphan Products Development, the full range of benefits including seven year market exclusivity, fifty percent tax credits, PDUFA fee waivers, and grant funding. You will then dive deeply into each component of the New Drug Application organized into the five CTD modules, with specific adaptations and flexibilities for orphan drug products including small batch manufacturing, single arm clinical trials, natural history controls, surrogate endpoints, and accelerated approval pathways.

A fictional gene therapy for Duchenne Muscular Dystrophy called TRT-GEN-001 is used as a running case study. Duchenne Muscular Dystrophy is a rare, X linked genetic disorder affecting approximately fifteen thousand to twenty thousand people in the United States.

Program Objectives

Upon completion of this training program, participants will be able to:

• Understand the complete legal framework of the Orphan Drug Act
• Prepare and submit a complete request for orphan drug designation to the Office of Orphan Products Development
• Design clinical trials for small patient populations including natural history studies and single arm trials
• Utilize accelerated approval pathways based on validated surrogate endpoints
• Prepare each section of an NDA for a rare disease drug with limited patient exposure data
• Navigate the unique regulatory flexibilities available for orphan drugs
• Maintain seven year market exclusivity after approval including annual reporting to the Office of Orphan Products Development
• Manage post approval requirements including confirmatory trials for accelerated approval, REMS if required, and postmarketing safety surveillance

Target Audience

• Regulatory Affairs Specialists working on gene therapies, enzyme replacement therapies, antisense oligonucleotides, and other orphan products
• Clinical Research Associates conducting trials in rare disease populations with geographically dispersed patients
• Medical Writers preparing orphan drug designation requests, natural history study reports, and NDA summaries
• CMC Associates for biologics and gene therapies including viral vector manufacturing and potency assay development
• Nonclinical Scientists working with rare disease animal models
• Quality Assurance Professionals for small batch manufacturing
• Project Managers for orphan drug development programs with extended timelines
• Legal and Patent Professionals for orphan drug exclusivity strategies and patent life extension
• Patient Advocacy Group Liaisons

Certification Details

Learners who successfully complete the program will receive a dated, traceable certificate that provides verifiable proof of their achievement for professional records.