Registration enabling campaign for accelerated development: A PPQ strategy with minimal early investments to enable fast to market development for a promising monoclonal antibody
May 6-11, 2018
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.
Jessica Wuu and Kristen Koterba Douglas, "Registration enabling campaign for accelerated development: A PPQ strategy with minimal early investments to enable fast to market development for a promising monoclonal antibody" in "Cell Culture Engineering XVI", A. Robinson, PhD, Tulane University R. Venkat, PhD, MedImmune E. Schaefer, ScD, J&J Janssen Eds, ECI Symposium Series, (2018). http://dc.engconfintl.org/ccexvi/241