Curriculum: Clinical Trials Design and Research Graduate Certificate

This course will provide an understanding on the early clinical drug development activities for newchemical entity that has received IND approval. Concepts of early clinical pharmacology studies, typically conducted in a Phase I setting such as single and multiple dose pharmacokinetics, dose escalation studies to determine the safety and tolerability of drug, mass balance and drug metabolism studies and bioavailability studies will be taught. Emphasis will then be placed on Phase II trials as proof-of-principle, and on the dose-ranging requirements for Phase II. Trial designs including cross-over and parallel group will be discussed. The importance of Phase I/II trials for making dose selections for Phase III will be explored, as will the use of Phase II trials as a go/no-go decision point. The importance of exposure-response (PK/PD) information in guiding the go/no-go decision will also be discussed. The use of biomarkers and pharmacogenomic information in decision making will be addressed. Activities which run in parallel to PhaseI/II trials, including formulation development and clinical supplies and toxicology studies will be discussed.

The course is designed to teach the statistical principles involved in designing clinical researchstudies to support new drug development programs. Statistical analysis techniques commonly used willbe discussed as well as the newer approaches beingconsidered. Statistical topics addressed in regulatory guidelines will be discussed. Emphasis will be placed on the use of statistical software such as JMP. Hands on exercises using the latest version of JMP will be conducted in various classes.

This course will provide a comprehensive background in the design and conduct of large, multicenter Phase III clinical trials of investigational compounds. Emphasis will be placedon the conduct of studies that include patients with varying demographics (age, gender, genetic background and disease state such as renal and hepatic). Issues pertaining to clinical operations, project management, human subjects protections and data safety monitoring will be discussed. An introduction to principles of population pharmacokinetics and pharmacodynamics will be included. The course will include conceptsof post marketing surveillance of approved drugs for drug safety and idiosyncratic reactions, global approaches for pharmacovigilance and compliance with ICH guidelines for recording and dissemination of adverse events. Issues pertainingto pharmacoepidemiology and clinical utilization/effectiveness of the drug will also bediscussed.

This course will provide students a background in project management issues in the conduct of global clinical trials, financial aspects of drug development and pharmacoeconomics of approved drug/drug products. The first module will focus on logistic and strategic issues of project management encountered in modern drug development. It includes exposure to principles of project management of complex programs involving: pharmaceutical development, operations, and regulatory affairs. In addition, the corporate challenges involved with portfolio management and optimization will be presented focusing on tools used for optimum decision making. This knowledge base will be applied towards selective case presentations with the goal to identify critical decision points in the process that dramatically impact the successful launch of a new product. The second module will emphasize the business and financial aspects of drug development, including biotechnology and other innovative small pharmaceutical companies that derive their funding primarily from venture capital funding, business strategies, outsourcing and merger and acquisitions. Valuation of the new chemical/molecule as it evolves in the drug development pipeline will be discussed.