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.

\n Format: Hardback
\n Length: 306 pages
\n Publication date: 12 November 2020
\n Publisher: Taylor & Francis Ltd
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Reviews critical issues such as endpoint selection, sample size requirement, and complex innovative design in regulatory review and approval processes for rare diseases. This comprehensive analysis enhances the understanding of statistical concepts and methods, which are essential in these assessments. It sheds light on controversial statistical matters that arise during regulatory review and approval, providing clarity and guidance. The report makes recommendations to accurately and reliably evaluate rare diseases regulatory submissions, leveraging innovative study designs and statistical methods. These include n-of-1 trial design, adaptive trial design, and master protocols such as platform trials, which have the potential to accelerate drug development for rare diseases. Additionally, the report provides insights into current regulatory guidance on rare diseases drug development, including gene therapy, which is a rapidly evolving field. By addressing these critical issues, the report contributes to improving the regulatory framework for rare diseases and advancing the development of treatments for patients with these challenging conditions.

\n Weight: 626g\n
Dimension: 161 x 242 x 26 (mm)\n
ISBN-13: 9780367502102\n \n