Regulation of Regenerative Medicines: A Global Perspective
vii
Foreword
Regenerative medicines have become paradigm-shifting treatment options for rare diseases, liquid tumor
cancers, and even more common disorders, such as severe burns in the US, Europe, Asia-Pacific, and
beyond. Each new discovery in gene therapy, cell therapy, and tissue engineering can change the conven-
tional wisdom about what is possible in human medicine—from durably treating a previously intractable
disease to possibly preventing it from occurring in the first place.
Science often advances faster than regulatory frameworks can adapt, particularly when those frameworks
are designed for simple molecules. If the nascent field of regenerative medicine is going to become the stan-
dard for a greater portion of humanity, regulatory frameworks around the world need to evolve rapidly.
This is not a challenge solely for regulators or regulatory affairs experts. Making the future of medi-
cine a reality will require a nimble partnership between regulators and therapeutic developers to nurture
the science, convert discoveries into treatments, and change the course of patients’ lives. Regulation of
Regenerative Medicines: A Global Perspective seeks to provide the foundational knowledge for this partner-
ship, including the history of the past and the possibilities of the future.
Throughout Regulation of Regenerative Medicines: A Global Perspective, preeminent regulatory experts
share definitive accounts of the recent history, current state of play, and the future of regenerative medi-
cine regulation. The book also explains how regenerative medicines differ from small molecules and other
biologics, the distinctive regulatory considerations for gene therapies and cell therapies, the similarities
and divergences in regulatory approaches across major geographies, and the requirements and evolving
guidance for the manufacture of these complex therapies.
The differences between regenerative medicines and traditional therapies are numerous. These medi-
cines have different starting and critical materials, distinctive manufacturing processes, and often require
unique clinical trial frameworks, long-term follow-up to demonstrate durability and safety, more rigorous
storage and distribution requirements, and difficult benefit-risk trade-offs given patient populations who
often have high unmet medical needs.
For the past 12 years, the Alliance for Regenerative Medicine has served as the global voice of this
rapidly evolving sector. During this brief period, the sector has moved from largely theoretical to increas-
ingly established—with several approved products, record-breaking investment, and a robust pipeline of
more than 2,200 clinical trials worldwide. The sector holds immense promise not only for monogenetic
rare diseases, but also for complex, polygenic diseases that afflict wide swaths of the population.
In the next 12 years, the scientific possibilities will only accelerate. This book will help build the
regulatory frameworks of the future and ensure that science’s greatest advancements have a transformative
impact on patients’ lives.
Janet Lynch Lambert
Chief Executive Officer
Alliance for Regenerative Medicine
vii
Foreword
Regenerative medicines have become paradigm-shifting treatment options for rare diseases, liquid tumor
cancers, and even more common disorders, such as severe burns in the US, Europe, Asia-Pacific, and
beyond. Each new discovery in gene therapy, cell therapy, and tissue engineering can change the conven-
tional wisdom about what is possible in human medicine—from durably treating a previously intractable
disease to possibly preventing it from occurring in the first place.
Science often advances faster than regulatory frameworks can adapt, particularly when those frameworks
are designed for simple molecules. If the nascent field of regenerative medicine is going to become the stan-
dard for a greater portion of humanity, regulatory frameworks around the world need to evolve rapidly.
This is not a challenge solely for regulators or regulatory affairs experts. Making the future of medi-
cine a reality will require a nimble partnership between regulators and therapeutic developers to nurture
the science, convert discoveries into treatments, and change the course of patients’ lives. Regulation of
Regenerative Medicines: A Global Perspective seeks to provide the foundational knowledge for this partner-
ship, including the history of the past and the possibilities of the future.
Throughout Regulation of Regenerative Medicines: A Global Perspective, preeminent regulatory experts
share definitive accounts of the recent history, current state of play, and the future of regenerative medi-
cine regulation. The book also explains how regenerative medicines differ from small molecules and other
biologics, the distinctive regulatory considerations for gene therapies and cell therapies, the similarities
and divergences in regulatory approaches across major geographies, and the requirements and evolving
guidance for the manufacture of these complex therapies.
The differences between regenerative medicines and traditional therapies are numerous. These medi-
cines have different starting and critical materials, distinctive manufacturing processes, and often require
unique clinical trial frameworks, long-term follow-up to demonstrate durability and safety, more rigorous
storage and distribution requirements, and difficult benefit-risk trade-offs given patient populations who
often have high unmet medical needs.
For the past 12 years, the Alliance for Regenerative Medicine has served as the global voice of this
rapidly evolving sector. During this brief period, the sector has moved from largely theoretical to increas-
ingly established—with several approved products, record-breaking investment, and a robust pipeline of
more than 2,200 clinical trials worldwide. The sector holds immense promise not only for monogenetic
rare diseases, but also for complex, polygenic diseases that afflict wide swaths of the population.
In the next 12 years, the scientific possibilities will only accelerate. This book will help build the
regulatory frameworks of the future and ensure that science’s greatest advancements have a transformative
impact on patients’ lives.
Janet Lynch Lambert
Chief Executive Officer
Alliance for Regenerative Medicine