Case Studies
Regulatory Affairs and Quality Assurance

CMC Regulatory Strategy to Accelerate Entry into Phase

Benefits of Case:
•Effective CMC regulatory strategy and execution to quickly coordinate interactions with FDA to allow rapid and cost effective advancement to Phase 3 with buy-in from agency on proposed strategy
•Complete, detailed and integrated Phase 3 program that supported starting Phase 3 with a Phase 2 formulation and bridging to a Phase 3 formulation prior to an NDA submission
•Allowed acceleration into Phase 3 clinical trials with a formulation strategy that permitted a commercial dosage form to be developed concurrently with Phase 3 trials

Background and Challenge:
The Client was a Phase 2 company having just completed and achieved favorable results from the proof of concept clinical trials on its drug candidate.  The drug candidate was a novel approach to an unmet medical need in a multi-billion dollar market.  The Client was actively pursuing several options for starting Phase 3:

• Allowing an additional 12 months to initiate Phase 3 while waiting for a commercial dosage form to be developed
• Starting Phase 3 with a sub-optimal dosage form and developing an optimum dosage form after the NDA submission
• Approaching the FDA with a novel strategy for developing a commercial dosage form during Phase 3 trial

PharmaDirections Strategy: 
• Identify the path to the NDA and a commercial dosage form using a unique regulatory approach
• Understand the Client’s risk tolerance
• Utilize deep knowledge of FDA expectations and commercial product development to suggest a strategy to the FDA that meet their requirements while meeting the client’s cost and time constraints

The Objectives:
• Regulatory strategy to present to the FDA for concurrence and guidance on a plan to develop a commercial dosage form during Phase 3 and bridge to this product
• Detailed plan, schedule and budget for drug development and formulation activities to meet the commercial product development timeline during Phase 3
• Manufacturing strategy and costs for analytical and clinical bridging studies• Designate outsourced services and time saving scenarios
• Preparation of documents and submissions to present the formulation development and clinical plan for FDA review

Value for the Client:

• Reduced time for initiating Phase 3 studies from 18 months to 6 months
• Allowed milestone of submitting NDA to be one year earlier
• Kept Phase 3 clinical trials on track while allowing sufficient time to develop an optimized commercial product

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