Clinical trials: planning and analysis.
With specific reference to studies of growth hormone therapies, this chapter re-examines some basic principles in clinical research and considers some of the more complex aspects of trial design. The statistical structure which underpins trial design is characterized with specific attention given to samples size, subgroup analyses, multicentre trials and the role and implementation of randomization. Topics related to outcomes in studies of growth hormone therapy are discussed, including the possible value of surrogate responses, longitudinal outcomes and their analysis, and multiple outcomes. Additional design issues addressed are multiarmed trials, factorial designs and study monitoring. Brief comments are offered on the interpretation of negative trials and on non-inferiority trials.
Vernon T
VT
Farewell
Richard J
RJ
Cook
D003627:Data Interpretation, Statistical; D004700:Endocrine System Diseases / Q000188:drug therapy*; D006130:Growth Disorders / Q000188:drug therapy*; D019382:Human Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D016032:Randomized Controlled Trials as Topic / Q000379:methods* / Q000706:statistics & numerical data*
D019382:Human Growth Hormone