New drugs with novel therapeutic characteristics. Have they been subject to randomized controlled trials?

OBJECTIVE To determine how many randomized controlled trials on the safety or efficacy of new drugs are published when these drugs are first marketed in Canada, and to determine the quality of the information in those trials.

DESIGN A MEDLINE search was conducted on each drug identified as having novel therapeutic characteristics and first marketed between 1990 and 2000.

MAIN OUTCOME MEASURES Number of trials dealing with the safety or efficacy of each drug published at the time the drug was marketed. Number of patients taking the study drug, length of the trial, and type of control.

RESULTS The number of trials varied substantially. For some drugs, there were more than 20 studies; for others only a single study. Many trials were small and short-term, and used placebo controls.

CONCLUSION Too few trials or inadequate trials on the safety and efficacy of new drugs are published when these drugs are first marketed in Canada. The lack of published trials means that physicians do not know whether results are generalizable to their patients, how to position the drug in relation to other treatments, or whether the drugs have long-term safety and efficacy.