Pharmacoepidemiology in safety evaluations of newly approved medications

In the recent extension of the Prescription Drug User Fee Act, the risk management programs that are to be funded by additional user fees combine ascertainment of risks in the initial postapproval phase with activities to confront these risks. Better recognition of risks, a prerequisite for success in managing risks, will require strategies to supplement the present system of spontaneous event reporting. This overview considers the merits of pharmacoepidemiology studies in examining gaps in drug safety resulting from the inherent limitations of clinical trials. After drugs become widely used following regulatory approval, many health outcomes potentially related to drug exposures can be studied in large health insurance claims databases using pharmacoepidemiology designs tailored to this venue. We clarify types of safety investigations particularly suited to examination in a large claims database, as well as the benefits and caveats of expanding postapproval follow-up through Prescription Drug User Fee Act funding.