A modest proposal for dropping poor arms in clinical trials.
This paper presents a simple procedure for clinical trials comparing several arms with control. Demand for streamlining the evaluation of new treatments has led to phase III clinical trials with more arms than would have been used in the past. In such a setting, it is reasonable that some arms may not perform as well as an active control. We introduce a simple procedure that takes advantage of negative results in some comparisons to lessen the required strength of evidence for other comparisons. We evaluate properties analytically and use them to support claims made about multi-arm multi-stage designs.
National Institute of Allergy and Infectious Diseases, Bethesda, MD, U.S.A.
Michael A
MA
Proschan
Lori E
LE
Dodd
D015982:Bias; D056808:Biostatistics; D001794:Blood Pressure; D017322:Clinical Trials, Phase II as Topic / Q000379:methods / Q000706:statistics & numerical data; D017326:Clinical Trials, Phase III as Topic / Q000379:methods / Q000706:statistics & numerical data; D018849:Controlled Clinical Trials as Topic / Q000379:methods* / Q000706:statistics & numerical data; D006801:Humans; D006973:Hypertension / Q000097:blood / Q000178:diet therapy / Q000503:physiopathology; D015233:Models, Statistical; D014280:Triglycerides / Q000097:blood
D014280:Triglycerides
comparisons with control; estimation bias; family-wise error (FWE) rate; multi-arm multi-stage (MAMS) design; multiple comparisons; sequentially rejective methods