Global benefit-risk assessment in designing clinical trials and some statistical considerations of the method.
When characterizing a therapy, the efficacy and the safety are two major aspects under consideration. In prescribing a therapy to a patient, a clinician puts the two aspects together and makes a decision based on a consolidated thought process. The global benefit-risk (GBR) measures proposed by Chuang-Stein et al. (Stat. Med. 1991; 10:1349-1359) are useful in facilitating the thinking, and creating the framework for making statistical comparisons based on benefit-risk point of view. This article describes how a GBR linear score was defined and used as the primary outcome measure in a clinical trial design. The robustness of the definitions of 'benefit' and 'risk' are evaluated using different criteria. The sensitivity of the pre-specified weights is also analyzed using alternative weights; one of those was determined by the relative to an identified distribution integral transformation approach (Biometrics 1958; 14:18-38). Statistical considerations are illustrated using pooled data from clinical trials studying antidepressant. The pros and cons for using GBR assessments in the setting of clinical trials are discussed.
Pritchett
Yili Lu
YL
Abbott Laboratories, Abbott Park, IL 60064, USA. yili.pritchett@abbott.com
Tamura
Roy
R
D002986:Clinical Trials as Topic / Q000379:methods / Q000706:statistics & numerical data; D006801:Humans; D016032:Randomized Controlled Trials as Topic / Q000379:methods* / Q000706:statistics & numerical data*; D012107:Research Design / Q000706:statistics & numerical data*; D018570:Risk Assessment / Q000379:methods / Q000706:statistics & numerical data