2018 Spring Semester **Thursday 6:30-8:30pm**
SYNOPSIS
In this interdisciplinary course, we review the history and evolution of the US Food and Drug Administration (the most influential public health and scientific regulatory agency worldwide, overseeing more than 25% of the US economy) and other major regulatory agencies in the world. We will discuss the concept of regulatory science and how it affects health policy and public health. We present the principles and process of drug development and illustrate the evolution of regulatory policies through the Forum for Collaborative Research’s experience in HIV, HCV, HBV, CMV, and liver diseases. We demonstrate the interdisciplinary nature of the regulatory agencies’ mandate (basic sciences, statistics, toxicology, pharmacology, clinical medicine, ethics, policy, law, political science, economic, foreign policy), and their responsiveness to public health needs. Real-time examples such as maintaining quality of generic drug products distributed through PEPFAR, development of regulatory policy on the use of next-generation sequencing platforms, ethical issues in HIV and HBV “cure” research, qualification of biomarkers for fatty liver diseases, serve as case studies to illustrate the benefits and contribution of a stakeholder engagement process of deliberation. Biotech and pharmaceutical industry experts will participate as guest lecturers to illustrate the ongoing need for interaction between the agency, academia, industry and public health community. The course will be of interest to students in public health, law, medicine, business or policy interested in biotech and the pharmaceutical industry.
CONTEXT
Regulatory science concerns “the development and use of tools, standards and approaches to more efficiently develop products and to more effectively evaluate product safety, efficacy and quality”. With the increasing role of regulatory agencies as knowledge stewards and broker's (data sets), comes the expansion of the regulatory mission, from solely protection, to the protection and promotion of the health of the public. Innovation to address the 21st century’s evolving regulatory landscape, in the context of new technologies, new understanding of diseases and a sharpened lens on safety -- is an exciting new area of science, one that requires a new generation of trained regulatory experts and professionals in academic, government, industry and public health sectors.
No other disease offers a better insight into the drug development process and the role of collaborative interactions between regulators, industry, academic research networks and community than HIV infection -- now a chronic disease thanks to the approval of more than 20 different antiretroviral agents in 7 drug classes, focused efforts to end the epidemic and collaborative efforts towards an HIV cure. Involvement of the HIV community led the way to significant changes in the approval process, which benefit all drugs. The HIV experience serves as a model for the development of HCV drugs and treatment of its worldwide epidemic. HCV affects more than 3 million people in the US and up to 170 million worldwide, its morbidity and mortality burden superseding that of HIV. Development of direct acting antivirals occurred at an unprecedented pace, with the first two drugs approved in 2011, two additional drugs reaching the market in 2014, and over 20 agents in phase 2 and/or 3 trials. The resulting treatment paradigm shift presents regulatory challenges and opportunities requiring multiple levels of interaction within the agency, as well as between agency and stakeholders. The multi-stakeholder model is now being applied to the development of new drugs and vaccines to prevent/treat cytomegalovirus (CMV – dubbed the “stealth virus”) disease in transplantation settings, and new therapeutic approaches for the prevention/treatment of liver fibrosis and other consequences of fatty liver disease (affecting 30% of adults and 10% of children/adolescents in the US). “If the prevalence of NASH continues to increase at this current rate and effective treatments are not found, about 25 million Americans will have the disease by 2025, and five million will need new livers” (NY Times, June 13, 2014).
INSTRUCTOR
Veronica Miller, PhD, the Executive Director of the Washington-based Forum for Collaborative Research, and Professor (Adjunct) or Infectious Diseases Vaccinology at UC Berkeley’s School of Public Health. She is an authority in HIV clinical research with over 20 years of experience in HIV clinical and drug development research. She has led numerous projects advancing HIV, HCV, HBV, CMV and liver fibrosis drug development in collaboration with the FDA, the European Medicines Agency, pharmaceutical and diagnostic industry, community and academia.
DRAFT COURSE OUTLINE
(Please see bCourses or Forum Educational Activities: Regulatory Science, Drug Development, Public Health & Health Policy (PH 236) for updated/detailed course schedule).
Session 1 (Jan 18): Introduction: Regulatory Science, Drug Development and Forum Model
Instructor: Veronica Miller
Session 2 (Jan 25): Standards for Efficacy
Instructor: Veronica Miller
Session 3 (Feb 1): Ethics (Case study: HIV and HBV Cure)
Instructor: Veronica Miller + Guests (TBN)
Session 4 (Feb 8): Biomarker Qualification Process
Instructor: Veronica Miller
Session 5 (Feb 15): Surrogate Markers, Expedite Approval Mechanisms (Antiviral Case Studies)
Instructor: Veronica Miller + Guests (TBN)
Session 6 (Feb 22): Diagnostics
Instructor: Veronica Miller
Session 7 (Mar 1): Rare Diseases and Orphan Drug Act
Instructor: Veronica Miller + Guests (TBN)
Session 8 (Mar 8): Benefit Risk (Case Studies: PrEP, Cure)
Instructor: Veronica Miller + Guests (TBN)
Session 9 (Mar 15): Drugs for Children
Instructor: Veronica Miller + Guests (TBN)
Session 10 (Mar 22): New Frontiers: Fatty Liver Disease
Instructor: Veronica Miller + Guests (TBN)
Session 11 (Apr 5): Pharmacovigilance
Instructor: Veronica Miller + Guests (TBN)
Session 12 (Apr 12): Regulation and Global Health
Instructor: Veronica Miller + Guests (TBN)
Sessions 13 (April 19): Student Presentations
Instructor: Veronica Miller
Sessions 14 (April 26): Student Presentations
Instructor: Veronica Miller
