ix
Foreword
This continues to be a remarkable time for science, medicine and public health. The rapid pace of
scientific discovery—from the so-called “omics” revolution and synthetic biology, to newly evolv-
ing cellular and gene therapies, to advances in technology such as gene-editing, nanotechnology,
3-D printing and, of course, information technology and data science—holds the promise of
truly revolutionary new therapies and interventions to prevent, treat and cure disease. Before us
is the opportunity to advance human health and well-being in ways not previously understood or
even imagined.
But, while translating new discoveries into real-world products can make an enormous
difference for individual and population health, the process is a complex and difficult one. It is
an activity fraught with challenges, requiring many steps and partners. The painstaking work to
develop, test, manufacture and distribute a new medical product successfully requires a vast range
of expertise, skills and dedication. Moreover, it demands coordinated work across disciplines,
sectors and, increasingly, across nations.
The task is not easy, but this volume provides a wonderful roadmap for those who are part
of this extraordinary enterprise seeking to realize the potential of modern medicine and public
health through enhanced regulatory science and strategy. Organized around a set of thoughtful,
authoritative and well-written chapters, the book lays out the essential elements of an inte-
grated regulatory approach. Written by a group of distinguished authors, each chapter is a clear
exposition of an important topic, ranging from the ABCs of regulatory strategy and the whys
and wherefores of interacting with regulators, to more technical aspects of the medical product
challenge such as preclinical and clinical development, expedited pathways, orphan designation,
pediatric products and product formulation, manufacturing and control. Of note, this book takes
a systematic approach, spanning the earliest stages of research and development to necessary
assessments and modifications in the postmarket period, as well as embracing globalization’s
realities for regulatory strategy and systems.
No matter how steeped one is in drug development and regulation, this book can provide
both updated information and new insights. And, for those still being initiated into this exciting
and essential realm of professional activity, this book can serve as a vital handbook for knowledge
and action.
Given the array of unmet medical care and public health needs before us, this is not an
academic exercise. There is a pressing responsibility to make sure the opportunities in science and
technology today will result in the safe, effective and high-quality medical therapeutics people
so hope for and deserve. Every effort must be made to ensure the best possible products are
delivered as swiftly and surely as possible, but never forgetting the scientific rigor and regulatory
oversight that ultimately determine failure or success.
Margaret A. Hamburg, MD
Former FDA Commissioner
Foreword
This continues to be a remarkable time for science, medicine and public health. The rapid pace of
scientific discovery—from the so-called “omics” revolution and synthetic biology, to newly evolv-
ing cellular and gene therapies, to advances in technology such as gene-editing, nanotechnology,
3-D printing and, of course, information technology and data science—holds the promise of
truly revolutionary new therapies and interventions to prevent, treat and cure disease. Before us
is the opportunity to advance human health and well-being in ways not previously understood or
even imagined.
But, while translating new discoveries into real-world products can make an enormous
difference for individual and population health, the process is a complex and difficult one. It is
an activity fraught with challenges, requiring many steps and partners. The painstaking work to
develop, test, manufacture and distribute a new medical product successfully requires a vast range
of expertise, skills and dedication. Moreover, it demands coordinated work across disciplines,
sectors and, increasingly, across nations.
The task is not easy, but this volume provides a wonderful roadmap for those who are part
of this extraordinary enterprise seeking to realize the potential of modern medicine and public
health through enhanced regulatory science and strategy. Organized around a set of thoughtful,
authoritative and well-written chapters, the book lays out the essential elements of an inte-
grated regulatory approach. Written by a group of distinguished authors, each chapter is a clear
exposition of an important topic, ranging from the ABCs of regulatory strategy and the whys
and wherefores of interacting with regulators, to more technical aspects of the medical product
challenge such as preclinical and clinical development, expedited pathways, orphan designation,
pediatric products and product formulation, manufacturing and control. Of note, this book takes
a systematic approach, spanning the earliest stages of research and development to necessary
assessments and modifications in the postmarket period, as well as embracing globalization’s
realities for regulatory strategy and systems.
No matter how steeped one is in drug development and regulation, this book can provide
both updated information and new insights. And, for those still being initiated into this exciting
and essential realm of professional activity, this book can serve as a vital handbook for knowledge
and action.
Given the array of unmet medical care and public health needs before us, this is not an
academic exercise. There is a pressing responsibility to make sure the opportunities in science and
technology today will result in the safe, effective and high-quality medical therapeutics people
so hope for and deserve. Every effort must be made to ensure the best possible products are
delivered as swiftly and surely as possible, but never forgetting the scientific rigor and regulatory
oversight that ultimately determine failure or success.
Margaret A. Hamburg, MD
Former FDA Commissioner