Curriculum: MS Drug Development Sciences

This course provides graduate students with an overview on legal and regulatory aspects relevant to drug and medical device development. This includes intellectual properties protection, global legal and regulatory requirements, protection of human subjects and ethics in clinical research and essential elements of management of global projects. Case studies will be included to underline how regulatory strategiesimpact product marketing and life cycle management. Throughout the course, students will integrate knowledge using specific industry examples with the goal to identify critical decision points in the development that impact success of a new drug or medical device on the market.

This course is an introduction to the discovery and early development (ie, prior to pre-clinical) of small molecule therapeutics and biopharmaceuticals such as proteins, peptides, vaccines, cell and gene therapies, and nucleotide based products. Introductions to the major classesand categories of drugs and biological will be provided including some principles of pharmacologysuch as drug targets/receptors and drug-receptor interactions. Methods and principles of drug/biologics discovery will be addressed from serendipitous observation to current approaches such as HTS, computational, combinatorial chemistry, and bioinformatics. Comparison of manufacturing methodology of drugs and biologics will be provided along with special clinical and regulatory considerations for biologic products. Additionally, emphasis will be given to major therapeutic areas (oncology, CNS, cardiovascular, Infectious Diseases/AIDS) in terms of approaches currently in use and in development along with addressing the unmet needs in each area.

The course is designed to teach students regulatory framework for the pre-clinical evaluation of the safety and efficacy of a molecular entity/lead compound identified in the drug discovery/early development phases for submission of the IND application. Major emphasis will be placed on the principles of pharmacokinetics/toxicokinetics and ADME processes(Absorption, Distribution, Metabolism and Excretion) so that safe (and efficacious) dosing regimens can be recommended for the early clinicaldevelopment phases. Differences between conventional (small molecules) vs. macromolecules and biologicals will be underscored.

This course is designed to provide an understanding of the studies conducted in support of the development of drug product (formulation/dosage form) and medical devices necessary to seek regulatory approval. An emphasiswill be placed on pre-formulation/formulation studies for optimizing lead compounds during earlystages of drug development, current good manufacturing practices (cGMP) and FDA/ICH guidelines for formulation components, processes and equipment, stability testing programs and overall quality assurance. Special aspects of formulation of biologicals and medical devices will also be discussed.

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.

This is the first of a two-course sequence. The overall objective is for the students to undertake a hypothesis driven research project or a comprehensive evaluation of a contemporary issue in the development of drugs/biologicals/devices. A committee comprising of three faculty members identified by the student will be constituted to guide the student in the undertaking. The project findings should demonstrate competency in multidisciplinary didactic coursework and scientific, regulatory and business aspects of new drug/drug product or device development.

This course follows Drug Development Project I. The overall objective of the effort in the two-course sequence is for the students to undertake a hypothesis driven research project or a comprehensive evaluation of a contemporary issue in the development of drugs/biologicals/devices. A committee comprising of three faculty members guides the student in the undertaking. The project findings should demonstrate competency in multidisciplinary didactic coursework and scientific, regulatory and business aspects of new drug/drug product or device development. At the end of the study, the student will be required to prepare a manuscript and also present a seminar as an "oral defense" that will be open to all students and faculty on campus.