Benefit-Risk Assessment in Pharmaceutical Research and Development

ISBN-13: 9781439867945 / Angielski / Twarda / 2013 / 220 str.

Andreas Sashegyi; James Felli; Rebecca Noel

Opis

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."

Recenzje

"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

Spis treści

Early Clinical Development: Pharmaceutical Benefit-Risk Assessment in Early Development. Full Clinical Development: Key Questions, Issues, and Challenges in Benefit-Risk Assessment in Full Clinical Development. The Clinical Aspects of Benefit and Risk. Quantifying Patient Preferences to Inform Benefit-Risk Evaluations. Benefit-Risk Modeling of Medicinal Products: Methods and Applications. Benefit-Risk Communication: Learning from Our Past and Creating Our Future. Regulatory Review and Policy: Policy Considerations and Strategic Issues Regarding Benefit-Risk. Systematic Approaches to Benefit-Risk Assessment. Post-Launch Assessment: Considerations and Strategies for Benefit-Risk Assessment in the Real World Setting. Benefit-Risk Assessment and the Payer Perspective. Epilogue. Glossary. Index.