Clinical Trials: Study Design, Endpoints and Biomarkers, Drug Safety, and FDA and ICH Guidelines

Clinical Trials, Second Edition, offers those engaged in clinical trial design a valuable and practical guide. This book takes an integrated approach to incorporate biomedical science, laboratory data of human study, endpoint specification, legal and regulatory aspects and much more with the fundamentals of clinical trial design. It provides an overview of the design options along with the specific details of trial design and offers guidance on how to make appropriate choices. Full of numerous examples and now containing actual decisions from FDA reviewers to better inform trial design, the 2nd edition of Clinical Trials is a must-have resource for early and mid-career researchers and clinicians who design and conduct clinical trials.

Contains new and fully revised material on key topics such as biostatistics, biomarkers, orphan drugs, biosimilars, drug regulations in Europe, drug safety, regulatory approval and moreExtensively covers the "study schema" and related features of study designIncorporates laboratory data from studies on human patients to provide a concrete tool for understanding the concepts in the design and conduct of clinical trialsIncludes decisions made by FDA reviewers when granting approval of a drug as real world learning examples for readers

1. The Origins of Drugs 2. Introduction to Clinical Trial Design 3. Run-in Period 4. Inclusion/Exclusion Criteria, Stratification and Subgroups – Part I 5. Inclusion and Stratification Criteria – Part II 6. Randomization, Allocation, and Binding 7. Intent to Treat Analysis vs. Per Protocol Analysis 8. Biostatistics – Part I 9. Biostatistics – Part II 10. Introduction to Endpoints for Clinical Trials in Pharmacology 11. Endpoints in Clinical Trials on Solid Tumors – Objective Response 12. Oncology Endpoints: Overall Survival and Profession-Free Survival 13. Oncology Endpoints:  Time to Progression 14. Oncology Endpoint: Disease-Free Survival 15. Oncology Endpoint: Time to Distant Metastasis 16. Neoadjuvant Therapy vs. Adjuvant Therapy 17. Hematological Cancers 18. Biomarkers and Personalized Medicine 19. Endpoints in Immune Diseases 20. Endpoints in Clinical Trials on Infections 21. Health-Related Quality of Life 22. Health-Related Quality of Life Instruments for Immune Disorders 23. Health-Related Quality of Life Instruments and Infections 24. Drug Safety 25. Mechanisms of Action, Part I 26. Mechanisms of Action, Part II – Cancer 27. Mechanisms of Action, Part III – Immune Disorders 28. Mechanisms of Action, Part IV- Infections 29. Consent Forms 30. Package Inserts 31. Regulatory Approval 32. Patents