Course Description
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, liver diseases and rare 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. Topics include the paradigm changing impact of HIV on drug regulation and approval, biomarker qualification programs, expedited approval mechanisms, the Orphan Drug Act, and innovation in addressing unmet medical needs of great public health significance. 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 overarching objective of improving global health”. 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. Moreover, innovations in drug development offer great opportunities for treatments to address unmet medical needs worldwide. Corresponding innovations in regulation of drug products are essential to evaluate their efficacy and safety and provide a more efficient path to ensure access for patients who need them.
Instructor Information
Dr. Miller is a leading expert in the process of advancing regulatory science and facilitating a development path through collaborative engagement of stakeholders from both sides of the Atlantic. She has extensive experience in addressing regulatory issues in several key disease areas, working closely with experts from the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in these projects.
Dr. Miller is the Director of the Forum for Collaborative Research, a public and private partnership addressing cutting edge science and policy issues through a process of stakeholder engagement and deliberation. Dr. Miller is also Professor (Adjunct) at the UC Berkeley School of Public Health. She developed and teaches this course based on case studies from the Forum's rich history in facilitating drug development.
Dr. Miller mentors interns and fellows pursuing regulatory, biotech, and translational medicine careers. Dr. Miller has published over 100 peer-reviewed publications on treatment and regulatory strategies.
Course Objectives
By the end of this course, students will be expected to be able to:1. Understand the concept of regulatory science and how it does (or not) keep pace with innovation in the biotech sector.
2. Describe and discuss the policies underlying current drug and biomarker regulation and the impact on clinical trial design and statistics, benefit-risk analysis, drug safety, medical ethics, as well as global health and foreign policy.
- Understand the historical progression to the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA) and the 21st Century Cures Act, including mechanisms for expedited approval.
4.Understand how regulatory science plays out in the international context.
Weeks at a Glance
Week 1: Introduction to Regulatory Science and Forum for Collaborative Research modelWeek 2: Ethics in the Regulatory Context
Week 3: Standards of Evidence: Efficacy and Safety
Week 4: Surrogate markers; Expedited Approval Mechanisms
Week 5: Long-term Safety: REMS and Pharmacovigilance
Week 6: New Frontiers – HBV
Week 7: New Frontiers – Liver disease: NASH
Week 8: Deconstructing the Regulatory Path for Diagnostics
Week 9: Diagnostics and Global Health
Week 10: New Frontiers – HIV Prevention
Week 11: New Frontiers – Primary Sclerosing Cholangitis
Week 12: New Frontiers – Rare Diseases – Orphan Drug Act
Week 13: Final Group Presentations
