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

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

Authors

Tom Brody Consultant for Second Genome, Inc. (Crohn's disease), Rigel Pharmaceuticals (platelet disorder), PrimeGen Biotech (stem cell therapy), Regeneron Pharmaceuticals (broadly neutralizing antibodies for HIV), and Biogen IDEC (multiple sclerosis). Dr. Tom Brody received his PhD from the University of California at Berkeley in 1980, and conducted postdoctoral research at University of Wisconsin-Madison and also at U.C. Berkeley. His 20 research publications concern the metabolism and pharmacology of folates, cloning an anti-cancer gene (XPE gene), and the structure of an antibody (natalizumab) used for treating multiple sclerosis. The author has 15 years of pharmaceutical industry experience, acquired at Schering-Plough, Cerus Corporation, and 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. At an earlier time, he wrote two editions of Clinical Trials, published by Elsevier, Inc. The author has 16 years of training and experience in the Code of Federal regulations, as it applies to pharmaceuticals and clinical trial design.