1979/05/31 by Marvin Zelen · 7 citations
Mathematics · Decision Sciences · Economics, Econometrics and Finance · Medicine · #Statistical Methods in Clinical Trials #Meta-analysis and systematic reviews #Health Systems, Economic Evaluations, Quality of Life #Medicine #Randomized controlled trial #Treatment and control groups #Clinical trial #Random assignment #Treatment effect #Physical therapy #Medical physics #Surgery #Internal medicine
paper · doi:10.1056/nejm197905313002203
openalex publication_date 1979/05/31 · openalex created_date 2025/10/10 · openalex updated_date 2026/08/04
This paper proposes a new method for planning randomized clinical trials. This method is especially suited to comparison of a best standard or control treatment with an experimental treatment. Patients are allocated into two groups by a random or chance mechanism. Patients in the first group receive standard treatment; those in the second group are asked if they will accept the experimental therapy; if they decline, they receive the best standard treatment. In the analyses of results, all those in the second group, regardless of treatment, are compared with those in the first group. Any loss of statistical efficiency can be overcome by increased numbers. This experimental plan is indeed a randomized clinical trial and has the advantage that, before providing consent, a patient will know whether an experimental treatment is to be used.