Session (CTI)

PURPOSE: This workshop aims to equip participants with the skills to calculate and interpret maximum acceptable risk (MAR) and minimum acceptable benefit (MAB) estimates. By understanding these measures, attendees will enhance their ability to integrate patient-preference information into regulatory reviews. The workshop aligns with recent FDA guidance, which emphasizes evaluating health technologies based on patient risk tolerance to improve the assessment of treatment benefits and risks. DESCRIPION: MAR and MAB are widely used to quantify patients’ risk tolerance and provide critical insights into their perspectives on new treatments. However, these estimates are influenced by factors such as patients’ baseline risk, how risks are presented, and the inclusion of opt-out options. Extensions of MARs and MABs, such as simultaneous MAR thresholds (SMARTs), enable the evaluation of tolerance for multiple risks collectively, providing a more comprehensive assessment of real-world risk tolerance.During the workshop, participants will explore extensions of basic MAR and MAB calculations and learn how to integrate them into benefit-risk assessments. They will use an interactive online calculator to analyze how varying assumptions impact risk-tolerance measures. Through this tool, participants will generate MARs, MABs, and SMARTs across different scenarios and engage in guided discussions with presenters on applying these measures to real-world regulatory contexts.By the end of the workshop, attendees will be equipped to effectively incorporate patient-preference data into regulatory decision-making, aligning with FDA expectations.Dr. Janssen will introduce MARs/MABs and moderate a discussion on the need to extend these estimates under various scenarios. Dr. Gonzalez will show how to use SMARTs to assess multiple risks and guide attendees in generating SMARTs. Dr. Boeri will present compensating variation, an alternative method for calculating MAR that is useful when an opt-out option is present.