In the first two decades of the 21st century, significant advances have been made in the science of drug development resulting in an array of new modalities entering the clinic. Regulators and sponsors alike are subject to increasing pressures to expedite the availability of these drugs globally for the treatment of unmet medical needs. Beginning with the United States and the European Union, health authorities from many countries have now implemented accelerated regulatory pathways that are based on the potential for significant patient benefit. The clinical successes of these drugs have resulted in significantly reduced development timelines, thus generation of CMC information for marketing authorizations is on the critical path to approval. Additionally, companies face significant challenges when managing post-approval changes globally. Solutions are often worked out on a case-by-case basis between each sponsor and regulatory agency since each program and modality present unique challenges. However, common topics and questions do exist from a CMC perspective. In concert with CMC challenges presented when managing expedited programs, the interpretation of phase-appropriate GMP guidance from health authorities remains a subject open to interpretation from industry experts. Interaction between regulatory agencies and industry representatives can assist with the development of harmonized risk-based approaches and develop tools to address these challenges, thus facilitating a common understanding of CMC challenges, and implementing strategies to ensure phase-appropriate GMP compliance.

This three-day CMC Forum will address pitfalls, risk mitigation levers, and risk-based approaches for common CMC issues, and discuss regulatory tools currently available to accelerate development in various countries. The first and second day of this forum will focus on CMC strategies for expedited program development, while the third day will focus on phase-appropriate GMPs and practical considerations for expedited programs. Specific topics and questions will be discussed via the use of case studies.