Gaps in the evaluation and monitoring of new pharmaceuticals: proposal for a different approach.

© 2003 Canadian Medical Association or its licensors The marked increase in spending on drugs 1 has led payers such as provincial governments to restrict funding for many drugs to specific clinical indications that are thought to be cost-effective. Some have argued that this unreasonably deprives patients of access to beneficial drugs. In this article we argue for a new approach to drug evaluation in Canada that combines the strengths of randomized trials and observational studies, and places more emphasis on the use of drug evaluation after marketing for decision-making. At least 4 different types of clinical studies are required to inform rational drug policy: (1) randomized trials to determine efficacy and safety (which are required for licensing), (2) real-world randomized trials to determine effectiveness and safety in regular practice, (3) observational studies that use administrative databases and (4) targeted primary data collection (Table 1). Currently, most randomized trials are done to determine efficacy and safety under ideal conditions, whereas the other designs, which are less frequently used, attempt to determine a drug’s pattern of use and effectiveness under real-world conditions. For some drugs, the results of randomized trials of efficacy will be so straightforward and the possibility of real-world use outside the conditions of the trial so small that no other study designs will be required. However, for other drugs there may be concern about the impact of the drug upon clinically important outcomes (e.g., if surrogate outcomes were used in the efficacy trials) or concern that the drug Gaps in the evaluation and monitoring of new pharmaceuticals: proposal for a different approach