Clinical Development of Biological Response Modifiers

Abstract

OBJECTIVE: To present perspectives on selected issues that frequently arise during the clinical development of biological response modifier (BRM) therapies.DATA SOURCES: The perspectives and opinions presented herein were developed over several years of reviewing and consulting on the clinical development of BRM therapies at the United States Food and Drug Administration.CONCLUSIONS: BRM therapies encompass a broad spectrum of products used to treat a wide variety of diseases. Due to this diversity. most principles of clinical trial design and conduct applicable to the majority of BRMS are those that are applicable to all therapies. Nevertheless, the clinical development of BRM therapies often raises specific issues and problems in the areas of selecting animal models, defining the study population, adverse reactions, dosing and defining end-points. Over 10 years’ experience in testing biotechnology derived BRMS in clinical trials has created a database from which we can draw valuable generalizations for guidance in future studies.