Drug Development for Rare Diseases e-bog
436,85 DKK
(inkl. moms 546,06 DKK)
A disease is defined as rare if the prevalence is fewer than 200,000 in the United States. It is estimated that there are more than 7,000 rare diseases, which collectively affect 30 million Americans or 10% of the US population. This diverse and complex disease area poses challenges for patients, caregivers, regulators, drug developers, and other stakeholders. This book is proposedto give an ov...
E-bog
436,85 DKK
Forlag
Chapman and Hall/CRC
Udgivet
13 februar 2023
Længde
222 sider
Genrer
MBGR1
Sprog
English
Format
pdf
Beskyttelse
LCP
ISBN
9781000820225
A disease is defined as rare if the prevalence is fewer than 200,000 in the United States. It is estimated that there are more than 7,000 rare diseases, which collectively affect 30 million Americans or 10% of the US population. This diverse and complex disease area poses challenges for patients, caregivers, regulators, drug developers, and other stakeholders. This book is proposedto give an overview of the common issues facing rare disease drug developers, summarize challenges specific to clinical development in small populations, discuss drug development strategies in the evolving regulatory environment, explain generation and utilization of different data and evidenceinside and beyond clinical trials, and use recent examples to demonstrate these challenges and the development strategies that respond to the challenges.Key Features: Rare disease. Drug development. Innovative clinical trial design. Regulatory approval. Real-world evidence.