Many practitioners in the pharmaceutical industry are still largely unfamiliar with benefit-risk assessment, despite its growing prominence in drug development and commercialization. Helping to alleviate this knowledge gap, Benefit-Risk Assessment in Pharmaceutical Research and Development provides a succinct overview of the key considerations relevant to benefit-risk assessment across the pharmaceutical R&D spectrum, from early clinical development to late-stage development to regulatory review to post-launch assessment.

The book first presents interpretations of benefit and risk in the context of a molecule moving from preclinical evaluation into its early testing in humans. It next considers benefit and risk characterization and assessment during a molecule’s journey from its clinical evaluation in humans through its submission to regulators for marketing approval. Throughout these sections, the book offers insight into the role of benefit-risk assessment in heightening understanding among key stakeholders by shaping questions and guiding discussions among scientists, physicians, developers, and regulatory agencies. The book also focuses on a molecule’s entry into the marketplace as a drug available for consumption by people. It explores the role of benefit-risk assessment as the relevance of carefully collected clinical efficacy and safety metrics fades in the wake of real-world use and evidence of effectiveness and safety.

Bringing together the expertise of 15 contributors from academia and the industry, this book offers an easy-to-read guide to the various facets of benefit-risk assessment in the major stages of pharmaceutical R&D. Suitable for those in both technical and managerial roles, it enables readers to communicate more effectively across their development chain as well as rationally and thoughtfully embed benefit-risk assessment into their R&D processes.

Bringing together the expertise of 15 contributors from academia and the industry, this book offers an easy-to-read guide to the various facets of benefit-risk assessment in the major stages of pharmaceutical R&D, from early clinical development to late-stage development to regulatory review to post-launch assessment. Suitable for those in both technical and managerial roles, it enables readers to communicate more effectively across their development chain as well as rationally and thoughtfully embed benefit-risk assessment into their R&D processes.

Early Clinical Development
Pharmaceutical Benefit-Risk Assessment in Early Development
Bennett Levitan and James Cross

Full Clinical Development
Key Questions, Issues, and Challenges in Benefit-Risk Assessment in Full Clinical Development
John Ferguson and Marilyn Metcalf

The Clinical Aspects of Benefit and Risk
Marilyn Metcalf

Quantifying Patient Preferences to Inform Benefit-Risk Evaluations
F. Reed Johnson, A. Brett Hauber, and Jing Zhang

Benefit-Risk Modeling of Medicinal Products: Methods and Applications
Lawrence D. Phillips

Benefit-Risk Communication: Learning from Our Past and Creating Our Future
Marilyn Metcalf

Regulatory Review and Policy
Policy Considerations and Strategic Issues Regarding Benefit-Risk
Timothy Franson and Philip Bonforte

Systematic Approaches to Benefit-Risk Assessment
Rebecca Noel

Post-Launch Assessment
Considerations and Strategies for Benefit-Risk Assessment in the Real-World Setting
Alicia Gilsenan and Elizabeth Andrews

Benefit-Risk Assessment and the Payer Perspective
Joseph Johnston, Ralph Swindle, James Felli, and Don Buesching

Epilogue

Glossary

Index