Quantitative Decisions in Drug Development

This book focuses on important decision points and evidence needed for making decisions at these points during the development of a new drug. It takes a holistic approach towards drug development by incorporating explicitly knowledge learned from the earlier part of the development and available historical information into decisions at later stages. The second edition reiterates the need for making evidence-based Go/No Go decisions in drug development discussed in the first edition. It substantially expands several topics that have seen great advances since the publication of the first edition, such as three adaptive trials conducted in recent years, the use of historical data in the design and analysis of clinical trials, and extending decision criteria to the cases when the primary endpoint is binary. The book offers useful insights to statisticians, clinicians, regulatory affairs managers, and decision-makers in the pharmaceutical industry who have a basic understanding of the drug-development process and the clinical trials conducted to support drug-marketing authorization.

Format: Hardback
Length: 342 pages
Publication date: 04 September 2021
Publisher: Springer Nature Switzerland AG

This comprehensive book delves into crucial decision points and the evidence required for making informed choices during the development of a new drug. It adopts a holistic approach to drug development, integrating knowledge gained from earlier stages and leveraging historical information to inform decisions at later stages. Additionally, the book shares valuable lessons learned from select examples published in the literature since the first edition.

The second edition of this text emphasizes the importance of making evidence-based Go/No Go decisions in drug development, a topic explored in the first edition. It significantly expands upon various topics that have witnessed significant advancements since the first edition. Noteworthy additions include three adaptive trials conducted in recent years, which provide valuable learning opportunities. The book also discusses the use of historical data in clinical trial design and analysis, as well as extending decision criteria to cases where the primary endpoint is binary.

To illustrate the additional materials, the book employs real-life trial examples, accompanied by post-trial reflections offered by the authors. The book begins with an overview of product development and regulatory approval pathways, setting the stage for subsequent chapters. It then delves into how prior knowledge can be effectively incorporated into study design and decision-making at different stages of drug development. Historical controls are considered a key component of prior knowledge, and the book provides guidance on how to assess their relevance and usefulness.

To assist in decision-making, the book discusses appropriate metrics and frameworks for assessing the potential of a drug candidate. It introduces the concept of the positive predictive value, commonly used in the field of diagnostics, and applies it to drug development. This helps readers understand the likelihood of a drug candidate progressing to the next development stage.

Throughout the book, case studies and real-world examples are used to reinforce the concepts and principles discussed. These examples help readers apply the knowledge gained to practical situations and gain a deeper understanding of the challenges and complexities involved in drug development.

In conclusion, this second edition of the book is an invaluable resource for researchers, scientists, and professionals involved in drug development. It provides a comprehensive and up-to-date overview of the key decision points and evidence required for successful drug development. By adopting a holistic approach and leveraging prior knowledge, readers can make informed decisions that contribute to the advancement of medical science and the improvement of patient care.

Weight: 676g
Dimension: 164 x 245 x 25 (mm)
ISBN-13: 9783030797300
Edition number: 2nd ed. 2021