© 2013 – Chapman and Hall/CRC
220 pages | 34 B/W Illus.
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.
"I think this text makes a strong case for the increasing importance of formal (and potentially quantitative) B-R assessment … readers new to B-R will get a well-rounded perspective of the key considerations and challenges to performing these assessments from thorough discussion of the overarching principles, different perspectives, and work to date. The authors identify several gaps, such as the emerging need to include patient perspective into the equation, and acknowledge that there are no universally accepted statistical methods at this time. Whether you are completely new to B-R or whether you are trying to implement quantitative methods at the company or project level, this book will be useful for statisticians charged with supporting these assessments now and in the future."
—Pharmaceutical Statistics, 2014
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
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
Regulatory Review and Policy
Policy Considerations and Strategic Issues Regarding Benefit-Risk
Timothy Franson and Philip Bonforte
Systematic Approaches to Benefit-Risk 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