REGISTRATION ENABLING CAMPAIGN FOR ACCELERATED DEVELOPMENT: A PPQ STRATEGY WITH MINIMAL EARLY INVESTMENTS TO ENABLE FAST TO MARKET DEVELOPMENT FOR A PROMISING MONOCLONAL ANTIBODY

摘要

Clinical development programs are increasingly designing trials and timelines with extra degrees of flexibility to allow for acceleration based on early positive data. It is becoming more common that a program in Phase I can plan for filing a Biological License Application in less then five years given a certain set of aggressive assumptions. This often places CMC activities on a critical and potentially risky path requiring a large resource commitment at an early stage of clinical development when the likelihood of commercial launch remains low. In this presentation we explore a case study for one such program that demonstrated both a low supply demand and limited clinical data, but also has the potential for a fast-to-market strategy gated to positive clinical results. We introduce the concept of a Registration Enabling Campaign (REC) conducted in parallel with a traditional Phase Ⅲ supply campaign that would supply the Registration Batches for a Biological License Application, but also eliminate the need for a traditional PPQ campaign. While this initially requires an early investment prior to Phase Ⅲ, it significantly reduces the resources required for a traditional qualification campaign that would be otherwise discarded for a low volume product. We will present the strategy for required prospective studies needed to support a Phase Ⅲ-REC, rationales for study deferrals and overall risks associated with analytical quality control systems that must be leveraged at a very early phase in development.