CMC Strategy Forum North America Fall 2020

In response to increasing demands to expedite the availability of various therapeutic modalities globally, health authorities have developed accelerated regulatory pathways to reduce development timelines towards product licensure with the goal of patient benefit. Due to the complexity and unique nature of each program and product modality, some solutions must be worked out between sponsors and regulatory agencies on a case-by-case basis. This session will provide an overview of currently available regulatory pathways for accelerated product development, as well as tools for effective management of post-approval changes specified in ICH Guideline Q12. Health authorities’ representatives will share their experience with review of requests and submissions under expedited programs and will provide examples and case studies for different therapeutic modalities to illustrate applicability of expedited programs to real cases, success, CMC challenges, and deficiencies with submissions’ packages seen in regulatory reviews. Topics for discussion will include comparability assessment, process validation strategies, specification setting, expectations for regulatory submissions, and some emerging approaches in the context of accelerated programs to facilitate a common understanding of CMC challenges and their resolution.

Innovative regulatory pathways have accelerated product development and approval for many innovative medicines for patients with an unmet medical need. These pathways have broken down barriers to speed up access for new therapies and have had a direct impact on public health. However, the shortened timelines and pressure to accelerate development creates unique challenges for industry. One of the biggest challenges is to develop, characterize and validate the commercial manufacturing processes and analytical testing methods under compressed clinical development timelines and to provide sufficient data and information to support line of sight from clinical to final intended commercial materials. This session will explore industry perspectives for accelerated programs with focus on process validation, comparability, stability and shelf-life considerations, and post-approval change management plans.

In response to the competitive landscape, unmet medical needs and increased patient demand, pharmaceutical companies have taken phase appropriate GMP approaches to accelerate drug development to market. In addition, increased interactions between regulatory agencies and industry also allowed for clarity and acceptability of these expedited approaches. This session focuses on industry and regulatory perspectives of phase appropriate GMP considerations for process and analytical development and validation, including considerations for different modalities (e.g. antibody-drug conjugates, vaccines, cell/gene therapies). Furthermore, the session will also cover innovative approaches such as setting of phase appropriate specifications, use of pools of clones for toxicological studies, clearance of process- and product-related impurities, phase appropriate raw materials testing, and inspection and commercial readiness.