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Shein-Chung Chow

Innovative Methods for Rare Disease Drug Development

Innovative Methods for Rare Disease Drug Development

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  • More about Innovative Methods for Rare Disease Drug Development

Reviews critical issues in rare diseases drug development, such as endpoint selection, sample size requirement, and complex innovative design. Provides better understanding of statistical concepts and methods for regulatory review and approval. Clarifies controversial statistical issues. Makes recommendations for accurate and reliable evaluation of rare diseases regulatory submissions. Proposes innovative study designs and statistical methods, including n-of-1 trial design, adaptive trial design, and master protocols. Offers insight into current regulatory guidance on gene therapy for rare diseases.

Format: Paperback / softback
Length: 306 pages
Publication date: 30 May 2022
Publisher: Taylor & Francis Ltd


Reviews critical issues such as endpoint selection, sample size requirement, and complex innovative design in regulatory submissions for rare diseases. Provides a better understanding of statistical concepts and methods that can be used in regulatory review and approval. Clarifies controversial statistical issues in regulatory review and approval. Makes recommendations to accurately and reliably evaluate rare diseases regulatory submissions. Proposes innovative study designs and statistical methods for rare diseases drug development, including n-of-1 trial design, adaptive trial design, and master protocols such as platform trials. Offers insight regarding current regulatory guidance on rare diseases drug development, such as gene therapy.

Reviews critical issues such as endpoint selection, sample size requirement, and complex innovative design in regulatory submissions for rare diseases. Provides a better understanding of statistical concepts and methods that can be used in regulatory review and approval. Clarifies controversial statistical issues in regulatory review and approval. Makes recommendations to accurately and reliably evaluate rare diseases regulatory submissions. Proposes innovative study designs and statistical methods for rare diseases drug development, including n-of-1 trial design, adaptive trial design, and master protocols such as platform trials. Offers insight regarding current regulatory guidance on rare diseases drug development, such as gene therapy.

Weight: 590g
Dimension: 234 x 156 (mm)
ISBN-13: 9780367502904

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