INTRODUCTION

Clinical trials are the backbone of the development and advancement of therapeutic interventions. The pace of development of new agents, the costs of data management, inclusion of quality-of-life assessments, symptom-control assessments and the evaluation of biologic correlates all present significant challenges for the future development of new therapeutic regimens. As these therapeutic regimens become increasingly targeted and our understanding of lymphoma deepens, the identification and assessment of relevant endpoints, be they clinical (e.g. physical examination, radiologic) or biologic (e.g. immunologic, genetic, metabolic etc.), also becomes increasingly complex. In turn, the more complex our endpoints and the more targeted our regimens, the more challenges are presented when designing and conducting clinical trials and analyzing and interpreting their results. While the field of clinical trial design is too broad and widely discussed to adequately give full discussion in this chapter, the reader is referred to more general references on this area. Given the complexity of issues when designing, monitoring, interpreting and analyzing data for a clinical trial, statistician input and collaboration is of paramount importance. This chapter focuses on the principles behind and details of clinical trials from the clinician perspective. As such, we discuss the fundamental considerations when designing trials as well as outline the types of trials typically conducted in clinical research.

BACKGROUND

The practice and science of clinical trials and research is in its relative infancy. Until 1750, the thinking was that of Galen, who attained an authority that remained unchallenged.