Background: Assessing the benefit-risk profile of a medicinal product is a complex but fundamental activity that sponsors and regulators must perform throughout the product's lifecycle. In order to improve the transparency and consistency of the decision-making process, regulators and sponsors alike are increasingly applying a structured approach to benefit-risk assessment. However, to our knowledge, there has been little practical guidance in the published literature regarding how to embed such a process organizationally. This paper seeks to address this gap.
Methods: Using a case study approach, we describe (1) how to integrate a lifecycle approach to structured benefit-risk assessment within a biopharmaceutical company; (2) key issues to anticipate during implementation, and (3) best practices and lessons learned to date.
Results: Based on our experience, key prerequisites for successful implementation included the selection of a structured benefit-risk assessment (SBRA) framework; application of a "core" approach to conducting SBRA with an accompanying template; development of a supporting standard operating procedure; and cross-functional team training. Common implementation challenges encountered were (1) facilitating cross-functional team adoption of SBRA nomenclature and analytic methods, including the use of a value tree and effects table, and (2) applying the SBRA framework to different products with heterogeneous data sources.
Conclusion: Conducting transparent, systematic benefit-risk evaluations is an emerging "best practice" for medicinal product lifecycle management. Our experience using such an approach resulted in improvements in the consistency, quality, conciseness and strategic value of our benefit-risk assessments, and increased transparency and harmonization in the communication of the product benefit-risk profile.
Keywords: European Medicines Agency (EMA); US Food and Drug Administration (FDA); benefit-risk framework; medicinal product; risk management plan; structured benefit-risk assessment.