Over the past decade pharmaceutical companies and regulatory agencies have been reviewing the benefit-risk assessment of medicines with a view to developing a structured systematic standardized approach. In view of this a universal framework together with an appropriate documentation system for recording benefit-risk decisions has been developed and applied and is a valuable addition to the Benefit-Risk Toolbox. The opportunity to examine the implementation of this approach by several regulatory authorities places this book in a unique position with the potential to positively contribute to the ongoing debate in this area.
The field of benefit-risk assessment continues to evolve at a rapid pace due to political and societal pressure, as is also reflected in the recent FDA PUDFA agreement as well as in the EMA 2015 Roadmap. Therefore, this is not an attempt to provide a comprehensive and complete account of the current benefit-risk assessment environment; rather, the strength of this book is that it provides an evaluation of some current approaches to the benefit-risk assessment of medicines and outlines the development of a new universal framework. It also demonstrates how a number of mature agencies have implemented this universal framework together with the methodology for documenting their benefit-risk decisions. In addition, it also reviews current publicly available documents together with their strengths and weaknesses in communicating benefit-risk decisions to stakeholders.
Table of Contents
Prologue.- Preface.- Overview.- Approaches to utilising decision-making framework.- Benefit-risk assessment of medicines by pharmaceutical companies and regulatory authorities.- Development of a universal benefit-risk framework and template.- Implementation of the benefit-risk assessment template by mature agencies.- Implementation of the benefit- risk summary template by a maturing agency: A case study.- Communicating benefit-risk decisions by US FDA, EMA, TGA and Health Canada.- Conclusions and future directions.- References.
James Leong, BSc Pharm Hons, MClinPharm, PhD, BCPS is a Board certified pharmacist
and Head of Education at the Centre of Regulatory Excellence, Duke-NUS Graduate
Medical School Singapore. His role focuses on identifying the educational needs
for the various stakeholders involved in regulatory affairs in the Asia Pacific
region and establishing educational roadmaps, priorities anddeliverables. He
received his PhD in regulatory sciences from Cardiff University, UK.
Sam Salek PhD RPh FFPM MRPSGB MCMS FESCP is Professor of Pharmacoepidemiology at the University of Hertfordshire, Hatfield, visiting Professor at the Estate of Hessen, Germany, and Director of the Institute for Medicines Development, Cardiff, UK. Professor Salek’s research focuses on mainly four areas including: development and application of patient-reported outcome measures; drug safety evaluation and pharmacovigilance; pharmacoeconomics and economic evaluation of healthcare; and pharmaceutical regulation and medicines development in the mature and emerging markets. He has published 19 books and 600 journal articles and abstracts. Professor Salek is member of five Editorial Boards and an active member of several societies.
Stuart Walker BSc, PhD, MFPM FRSC FIBiol, FRCPath, is the Founder of the Centre for Medicines Research International, the Centre for Innovation in Regulatory Science and Professor of Pharmaceutical Medicine, University of Wales, Cardiff. During his research career in academia and industry, he has supervised many PhD programmes in clinical development, regulatory policies, the Benefit/Risk Assessment of Medicines and Health Technology Assessment, co-authored 280 research papers and edited twenty-five books. He is frequently involved in the organisation of international meetings on key issues that concern the Regulatory Review of Medicines and has lectured extensively throughout Europe, the United States, the Asia-Pacific Region, Latin America and the Middle East.