Orphan Drug Development for Rare Diseases
vii
Foreword
Understanding development and regulatory processes is crucial to advancing therapies for rare diseases,
which involves complex and unique incentives, requirements, market considerations, and opportunities
for patient engagement. This new book, Orphan Drug Development for Rare Diseases, will quickly become
a go-to resource for anyone looking to understand requirements and regulatory pathways for drugs and
biologics intended to treat rare diseases.
A comprehensive overview, the book is divided into seven chapters by geographic region—US, EU, UK,
Japan, Australia, Canada, and Rest of the World—providing an in-depth look at orphan drug development
in every major global market. Each chapter includes current information on history, incentives, benefits,
tools, step-by-step processes for seeking and obtaining orphan designation and marketing authorization for
orphan drugs, opportunities for patient engagement, case studies, and market access considerations.
Orphan Drug Development for Rare Diseases will be a valuable resource to help guide development
and regulatory engagement, uncover areas of global harmonization, and provide a working understanding
of this dynamic area for anyone interested in how drugs for rare diseases are developed. It will be helpful
for academic institutions, pharmaceutical professionals involved in drug development, and regulatory
professionals at all levels—from those just learning about drug development to those looking to engage in
new frontiers in finding cures and treatments for rare diseases.
Eva A. Temkin, JD
Partner
King &Spalding
vii
Foreword
Understanding development and regulatory processes is crucial to advancing therapies for rare diseases,
which involves complex and unique incentives, requirements, market considerations, and opportunities
for patient engagement. This new book, Orphan Drug Development for Rare Diseases, will quickly become
a go-to resource for anyone looking to understand requirements and regulatory pathways for drugs and
biologics intended to treat rare diseases.
A comprehensive overview, the book is divided into seven chapters by geographic region—US, EU, UK,
Japan, Australia, Canada, and Rest of the World—providing an in-depth look at orphan drug development
in every major global market. Each chapter includes current information on history, incentives, benefits,
tools, step-by-step processes for seeking and obtaining orphan designation and marketing authorization for
orphan drugs, opportunities for patient engagement, case studies, and market access considerations.
Orphan Drug Development for Rare Diseases will be a valuable resource to help guide development
and regulatory engagement, uncover areas of global harmonization, and provide a working understanding
of this dynamic area for anyone interested in how drugs for rare diseases are developed. It will be helpful
for academic institutions, pharmaceutical professionals involved in drug development, and regulatory
professionals at all levels—from those just learning about drug development to those looking to engage in
new frontiers in finding cures and treatments for rare diseases.
Eva A. Temkin, JD
Partner
King &Spalding