Regulatory Considerations for Phases 2 and 3 in Drug Development

This mini-course is hosted by the Center of Excellence in Regulatory Science and Innovation (CERSI) co-led by UCSF and Stanford University.

In this mini-course, we will discuss regulatory considerations for Phases 2 and 3 of the drug development process in pharmaceutical industry. We will overview regulatory and design considerations for Phase 2 patient studies - exploring “Proof of Concept” drug effect in a defined patient population, relationship between dose and effectiveness effects, potential use of biomarkers, and safety evaluation. The use of Phase 2 data to design new Phase 3 protocols and how exceptional Phase 2 studies may be considered as a Pivotal study will be discussed. The description of FDA guidance on formal EOP2 meeting between FDA and sponsors regarding Phase 3 clinical protocol design and other key development aspects will be presented. We will discuss meeting objectives including, results of dose-response trials, identified major Phase 3 trial design aspects, such as, trial population, critical exclusions, choice of dose, primary and secondary trial endpoints, and major analyses (including any planned interim analyses and adaptive features, and major safety concerns). The attendees will review design features of Phase 3 studies, including types of controls, trial population, randomization and blinding, primary and secondary endpoints, and safety endpoints. Trial conduct and global considerations. Regulatory requirements for NDA/BLA. Course attendees will participate in group discussions of two case-studies.

Instructor: Ms. Carolyn Finkle

Carolyn Finkle has over 27 years scientific and industry experience including 17 years in management of drug and biologic development, quality and regulatory affairs for companies in the U.S. and Canada, including VP, Global Regulatory Affairs, Catalent Pharma Solutions, Senior Director, International Regulatory Affairs, AstraZeneca, Vice President, Global Product Development Strategy and VP North America Consulting, PAREXEL International, VP Regulatory Affairs at Celsion Corporation, VP Preclinical Development at TherImmune Research Corporation, VP Drug Development at GeminX Biotechnologies, Director of Preclinical Development at ConjuChem and Manager of Preclinical Development at BioChem Pharma (acquired by Shire). Carolyn Finkle holds a M.Sc. Chemistry from the University of Toronto, Canada and has a B.Sc. Chemistry from the University of Ottawa, Canada. She has worked in academic research at Stanford University, the University of Toronto, Max Bell Research Institute, Toronto General Hospital and Chiba University, Japan, prior to her industry managerial appointments. Carolyn Finkle currently serves as an Adjunct Lecturer and graduate course co-director of the Georgetown University Master’s Program in Clinical and Translational Research and Course Director of the Pharmaceutical Education and Research Institute (PERI), as well as, contributor to the UCSF American Course of Drug Development and Regulatory Sciences (ACDRS). Carolyn has co-authored multiple white papers, 1 book chapter on First-in-Human global regulatory requirements, over 18 abstracts and 12 papers for industry associations and scientific publications and chaired DIA sessions (global regulatory) at the annual meetings. She has
received numerous awards and scholarships throughout her career.