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