Benefit-risk assessment is a fundamental element of drug development with the aim to strengthen decision making for regulators, payors, providers, and patients. Customized benefit-risk assessment can provide useful information for proactive intervention in health care settings, which could save lives, reduce litigation, improve patient safety and health care outcomes, and lower overall health care costs. We have been focusing on post approval benefit-risk assessment methodologies.
We argue that a structured benefit-risk assessment is not simply a statistical exercise, but should be a multi-disciplinary effort involving experts in clinical science, patient advocacy groups, safety assessment, decision science, health economics, epidemiology and statistics. Multiple stakeholders, each with their own objectives and perspectives, are involved in making treatment decisions. Instead of ignoring the different perspectives and insisting upon one set of benefits and risks and one way to measure their impact, we believe we need to embrace the difference and help all stakeholders make consistent, well-thought out decisions.