Drug Development for Rare Diseases 1st Edition

Grab attention with a clear roadmap to one of medicine’s most urgent frontiers: Drug Development for Rare Diseases, 1st Edition by Yang Bo is an essential guide for scientists, clinicians, regulators, and industry leaders navigating orphan drug discovery and development worldwide.

This concise, practical volume balances scientific rigor with real-world strategy. You’ll find clear explanations of translational research, biomarker-driven design, adaptive trial methods, and manufacturing considerations specific to rare disease therapeutics. The book also examines regulatory pathways across major regions — including FDA, EMA, and Asia-Pacific frameworks — offering a global perspective valuable to multi-regional development teams and policy-minded readers.

What makes this edition particularly useful is its focus on actionable insights: risk-benefit assessment for small populations, patient-centric trial design, leverage of natural history studies, and commercialization strategies for orphan drugs. Case-based discussions and comparative regulatory examples make complex topics accessible, helping readers translate science into viable programs.

Ideal for researchers, biotech professionals, clinical development teams, and postgraduate students, this book helps you accelerate decision-making, reduce development risk, and align programs with regulatory and payer expectations. Its pragmatic tone and up-to-date guidance make it a practical reference for work in the US, EU, China, and other global markets.

For anyone committed to advancing therapies for rare diseases, Drug Development for Rare Diseases, 1st Edition by Yang Bo is a go-to resource — authoritative, globally aware, and built for application. Add it to your professional library to support smarter, patient-focused drug development.

Note: eBooks do not include supplementary materials such as CDs, access codes, etc.