Assessment of benefit vs. risk of drug therapy: The potential for outcome analysis with flexible models

摘要

Pharmaceutical companies face twin pressures of improving the throughput of new products and responding to increased requirements by regulatory bodies for risk evaluation and mitigation both pre- and post-marketing. This paper gives a brief overview of flexible outcome models to improve the accuracy of these studies. The focus is on the discovery of niche populations in secondary analysis and monitoring of adverse events in observational studies of new products. Analytical results illustrate the power of the methods and they can be mapped onto Boolean filters to represent populations with particular benefit/risk ratios.