Drug development for rare diseases / edited by Bo Yang, Yang Song, Yijie Zhou.
Series: Publisher: Boca Raton : CRC Press, ©2023Edition: First edition.Description: 221 p.Content type: text Media type: computer Carrier type: online resourceISBN: 9780367518349; 9780367532116.Subject(s): Rare Diseases -- drug therapy | Drug Development | Orphan Drug Production | Clinical Trials as Topic | United StatesGenre/Form: Print books.| Current location | Call number | Status | Date due | Barcode | Item holds |
|---|---|---|---|---|---|
| On Shelf | RM301.25 .Z3625 2023 (Browse shelf) | Available | AU00000000019819 |
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| RM301.25 .S73 2015 Statistical methods in drug combination studies / | RM301.25 .V47 2022 Making medicine : surprising stories from the history of drug discovery / | RM301.25 .Y3625 2023 Data science, AI, and machine learning in drug development / | RM301.25 .Z3625 2023 Drug development for rare diseases / | RM301.27 .G65 2014 Bad pharma : how drug companies mislead doctors and harm patients / | RM301.27 .K39 2015 Statistical thinking for non-statisticians in drug regulation / | RM301.27 .V47 2015 Pharmacy research : a how-to guide for students, residents, and new practitioners / |
Includes bibliographical references and index.
Challenges for Rare Disease Drug Development / By Glen Laird -- Regulatory Landscape / By Joanne Palmisano -- Clinical Trial Design Considerations for Rare Diseases / By Lanju Zhang, Xiaolong Luo, Lingyun Liu, Ina Jazic, Bingming Yi, Ntin Nair, Xiaopeng Miao, Glen Laird and Xioayan Liu -- Statistical Analyses Methodologies and Considerations for Rare Disease Data / By Lina Titievsky and Nataliya Volkova -- Real World Evidence vs. Clinical Trials / By Jingjin Ye, Lian Ma, Amy Barone, Nicholas Richardson, Emily Wearne, Kerry Jo Lee and Charu Gandotra -- Clinical Development of Pediatric Program within Rare Diseases / By Brenda Cirincione and Mark Peterson -- Case Studies of Rare Disease Drug Development / By Yimeng Lu, Chenkun Wang, Tu Xu, Jason Yuan and Tina Liu.
"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 proposed to 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 evidence inside and beyond clinical trials, and use recent examples to demonstrate these challenges and the development strategies that respond to the challenges"--