Incorporating Patient Perspectives into Structured Benefit-Risk Assessment: A Drug Development Framework Recommendation.

Abstract

Regulatory agencies have been promoting the incorporation of the patient perspective into benefit-risk assessment to better align regulatory decisions with patients' needs and priorities. Currently, benefit-risk assessments for regulatory submissions primarily capture the patient perspective through patient-reported outcome data from clinical trials. However, there is a push for a more systematic approach to capturing the patient perspective in benefit-risk assessment and decision-making throughout the drug development life cycle. Although different guidelines and frameworks have been developed, consensus on how to systematically incorporate the patient perspective into structured benefit-risk (sBR) assessment remains elusive. In 2023, Sullivan et al. published an sBR assessment framework that was developed to enhance systematic and collaborative decision-making throughout a drug life cycle. Here we propose how this sBR assessment framework could be expanded, committing to a patient-centered approach by considering the patient perspective at every step of drug development. These recommendations aim to put patients at the center of drug development, ultimately leading to better treatment outcomes and improved lives.