Many decision makers desire credible information about the clinical and economic effects of new interventions. The sentiment of many speakers was that randomized trials, although providing the greatest assurance of internal validity, fall short of providing the data needed for economic analyses to be useful in the real world. The sample size of trials may be insufficient to detect significant differences in economic end points. Second, the medical resources actually used in trials are often driven by the research protocol in response to regulatory concerns, which can limit the generalizability of the findings when applied to real-world situations. Most technology must be adopted before all long-term consequences can be established with certainty.
Controversy remains about how best to overcome the limitations of randomized trials.
Different constituencies, despite their varied agendas, have expressed concern that existing methods of randomized trials do not provide the quality and timeliness of evidence that they and the public need for making real-world decisions. It is encouraging, however, that these groups are striving to work together to develop competent and reliable standards, perhaps considering new solutions, for estimating the clinical and economic consequences of new therapies. John Hornberger, MD, MS Roche Global Pharmacoeconomic Research Palo Alto, CA 94304 Louis Garrison, PhD Stanford University School of Medicine Stanford, CA References 1. Food and Drug Administration Modernization Act of 1997. Am J Publ Health.