Clinical Trials

Study Design, Endpoints and Biomarkers, Drug Safety, and FDA and ICH Guidelines


  • Tom Brody, PhD, University of California at Berkeley, The author is a former employee of Schering-Plough Corporation. He has ten years of pharmaceutical industry experience, acquired at Schering-Plough, Cerus Corporation, and Athena Neurosciences (Elan Pharmaceuticals), and has contributed to FDA submissions for the indications of multiple sclerosis, melanoma, head and neck cancer, liver cancer, pancreatic cancer, and hepatitis C.

Clinical Trials: Study Design, Endpoints and Biomarkers, Drug Safety, and FDA and ICH Guidelines is a practical guidebook for those engaged in clinical trial design. This book details the organizations and content of clinical trials, including trial design, safety, endpoints, subgroups, HRQoL, consent forms and package inserts. It provides extensive information on both US and international regulatory guidelines and features concrete examples of study design from the medical literature. This book is intended to orient those new to clinical trial design and provide them with a better understanding of how to conduct clinical trials. It will also act as a guide for the more experienced by detailing endpoint selection and illustrating how to avoid unnecessary pitfalls. This book is a straightforward and valuable reference for all those involved in clinical trial design.
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Pharmaceutical scientists and pharmacologists, medical writers and physicians, nurses and pharmacists who plan and conduct clinical trials.


Book information

  • Published: November 2011
  • ISBN: 978-0-12-391911-3


"A solid guide to designing clinical trials for medical scientists, especially those working in oncology, immune disease, and infectious disease…Three chapters address quality-of-life topics."  -Selected for The First Clinical Research Bookshelf, "Essential Reading for Clinical Research Professionals," Journal of Clinical Research Best Practices, September 2012, Vol. 8, No. 9

Table of Contents

1. The Origins of Drugs
2. Study Schema
3. Run-in Period
4. Inclusion and Stratification, Part I
5. Inclusion and Stratification, Part II
6. Blinding, Randomization, and Allocation
7. Placebo
8. Intent to Treat Versus Per Protocol
9. Statistics
10. Introduction to Endpoints for Clinical Trials in Pharmacology
11. Oncology Endpoint: Objective Response
12. Oncology Endpoint: Overall Survival and Progression-free Survival
13. Oncology Endpoint: Time to Progression
14. Oncology Endpoint: Disease-free Survival
15. Oncology Endpoint: Time to Distant Metastasis
16. Neoadjuvant Therapy Versus Adjuvant Therapy
17. Hematological Cancers
18. Biomarkers and Personalized Medicine
19. Endpoints for Immune Diseases
20. Endpoints for Infections
21. Health-related Quality of Life for Oncology
22. Health-related Quality of Life for Immune Disorders
23. Health-related Quality of Life and Infections
24. Drug Safety
25. Mechanism of Action, Part I
26. Mechanism of Action, Part II: Cancer
27. Mechanism of Action, Part III: Immune Diseases
28. Mechanism of Action, Part IV: Infections
29. Consent Forms
30. Package Inserts
31. Regulatory Approval
32. Patents