The Academic NDA: Justification, Process, and Lessons Learned.

Area of Science:

• Molecular Imaging
• Radiopharmaceutical Development
• Regulatory Science

Background:

• The development pathway for novel PET imaging agents often faces challenges in achieving regulatory approval.
• Academic institutions may lack the infrastructure and expertise for navigating complex drug approval processes.
• ⁶⁸Ga-DOTATOC represents a clinically valuable PET radiopharmaceutical that required a dedicated regulatory strategy.

Purpose of the Study:

• To detail the University of Iowa's experience in obtaining Food and Drug Administration (FDA) marketing approval for the PET imaging agent ⁶⁸Ga-DOTATOC.
• To provide a comprehensive overview of the new-drug application (NDA) process from an academic perspective.
• To encourage greater community understanding and support for academic-led drug development and regulatory filings.

Main Methods:

• A retrospective review of the 4-year process for the ⁶⁸Ga-DOTATOC new-drug application (NDA).
• Description of the clinical trials conducted to support the application.
• Overview of the structured content and requirements of the FDA NDA document.
• Analysis of the interactions and experiences with the FDA regulatory body.

Main Results:

• The University of Iowa successfully achieved marketing approval for ⁶⁸Ga-DOTATOC in August 2019.
• The FDA demonstrated efficiency, reasonableness, and clear communication throughout the rigorous review process.
• The experience highlighted the FDA's ability to balance support for innovation with ensuring safety and efficacy standards.

Conclusions:

• The academic new-drug application (NDA) process, while rigorous, is navigable with adequate preparation and engagement.
• Sharing information and fostering support within the molecular imaging community can streamline future academic filings.
• Establishing infrastructure and understanding for academic NDAs is crucial for bringing valuable PET imaging agents to market.