Ther Innov Regul Sci. 2026 Feb 21. doi: 10.1007/s43441-025-00894-9. Online ahead of print.
ABSTRACT
Medicinal products have benefits and risks that must be carefully balanced to inform decision making. The structured benefit-risk (BR) framework is a powerful approach not only to standardize a holistic BR assessment, but also to incorporate the patient perspective and guide the decisions and discussions of sponsors and regulatory agencies throughout the continuum of drug development. Structured BR assessment has been usually conducted using a qualitative approach during the late development stage. The use of quantitative models that can be applied throughout the drug development process may provide more objective BR information to support scientific recommendations to optimize and inform decisions for critical external and internal development opportunities. A new Multidimensional Benefit-Risk Integrated Evaluation (MBRIE) quantitative model was developed using key attributes of the structured BR assessment. Each attribute was evaluated by assigning a rating score ranging from 1 to 3 (low), 4-6 (medium), 7-10 (high). Also, two dimensions for comparative purposes were considered: standard of care (SOC) and probability of development success (PODS) (likelihood or favorability for development success). Graphical outputs were used to visualize and compare the ranking scores for each of the attributes across the two dimensions. This analysis implements a structured quantitative BR assessment approach earlier in drug development and through the drug lifecycle. The MBRIE model may be an innovative tool to facilitate solutions by fostering a collaborative culture that points to the true objective to improve patient outcomes.
PMID:41723330 | DOI:10.1007/s43441-025-00894-9
