Innovative Methods for Rare Disease Drug Development - Chapman & Hall/CRC Biostatistics Series

Reviews critical issues (e.g., endpoint/margin selection, sample size requirement and complex innovative design). Provides better understanding of statistical concepts and methods which may 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. Provides insight regarding current regulatory guidance on rare diseases drug development such as gene therapy.

ISBN:	9780367502904
Publisher:	Taylor & Francis
Imprint:	Chapman & Hall/CRC
Published date:	30 May 2022
DEWEY:	615.19
DEWEY edition:	23
Language:	English
Number of pages:	306
Weight:	488g
Height:	154mm
Width:	233mm
Spine width:	22mm

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