International Journal of Technology Assessment in Health Care

Special Section: The Rational Use of Therapeutic Drugs

The Rational Use of Therapeutic Drugs in the 21st Century: Important Lessons from Cumulative Meta-analyses of Randomized Control Trials

Joseph Laua1 and Thomas C. Chalmersa2

a1 New England Medical Center

a2 MetaWorks, Inc.


The use of randomized control trials to assess the usefulness of therapeutic drugs over the last half century has brought significant benefits to patient care. The full potential benefits, however, have been only partially fulfilled because available data are frequently poorly used. Meta-analysis has emerged as an important tool for combining clinical evidence. Several examples are presented that compared the results of cumulative meta-analysis of randomized control trials with clinical expert recommendations. These comparisons demonstrated that clinical expert recommendations are often not synchronized with accumulating evidence, and this lack of recognition often resulted in delays in the acceptance of effective drugs and the slow abandonment of possibly harmful therapeutic practices. The problems of inappropriate therapeutic drug use will only intensify as new drugs are introduced and new uses for established drugs are proposed. The rational use of therapeutic drugs can be achieved only through the routine use of meta-analysis on high-quality clinical data. Some suggestions are made to improve the quality of the original research and the ways of assembling meta-analyses and disseminating their results.