Benefit-Risk Appraisal of Medicines A Systematic Approach to Decision-making

by Mussen, Filip; Salek, Sam; Walker, Stuart

• ISBN13: 9780470060858
• ISBN10: 0470060859
• Edition: 1st
• Format: Hardcover
• Copyright: 2010-02-01
• Publisher: Wiley

Summary

Written by renowned experts in the field, this book establishes the criteria required to assess benefit-risk in general and reviews the current practice of benefit-risk assessment by drug regulatory authorities and the pharmaceutical industry. It outlines how the new MCDA model was developed and evaluated, and discusses the implications of its implementation into the practice of drug evaluation. Comparisons are also made with existing benefit-risk models such as the 'Principle of Threes' and TURBO models. Discusses a very topical subject in the wake of the withdrawal of the Cox-2 inhibitors and other high-profile withdrawals Includes risk-management plans New MCDA model is relevant for the assessment of both, new compounds being presented for licence and the ongoing evaluation of existing drugs on the market MCDA model allows for all available efficacy and safety data to be evaluatedRisk/Benefit Assessment in Medicine is an invaluable resource for researchers, clinical trial managers, pharmacovigilance and epidemiology staff in the pharmaceutical industry. The book is also useful for drug regulatory authorities, and pharmaceutical medicine or medicinal chemistry departments at universities.

Author Biography

Dr Sam Salek, Reader in Pharmacoepidemiology, Director - WSP Centre for Socioeconomic Research; Director - Postgraduate Course in Pharmaceutical Medicine, Cardiff University, UK.

Mr Filip Mussen, Johnson & Johnson Pharmaceuticals Research and Development, Belgium.

Prof Stuart Walker, President and Founder, International Institute for Regulatory Science, UK.

Table of Contents

Foreword	p. vii
Preface	p. ix
Concept and Scope of Benefit-Risk Evaluation of Medicines	p. 1
Historical background	p. 1
The regulatory systems for assessing medicines	p. 3
Benefit-risk assessment: definitions	p. 4
Views and perceptions of benefits and risks of medicines	p. 6
Stages and concepts in benefit-risk assessment	p. 11
Benefit-risk assessment: the current regulatory environment	p. 19
Benefit-risk assessment in other disciplines	p. 19
Specific methods and models for benefit-risk assessment	p. 21
Discussions with stakeholders on the concepts and models for benefit-risk evaluation	p. 30
Criteria for a Benefit-Risk Model: a Conceptual Framework	p. 31
Introduction	p. 31
Regulatory guidelines on benefit and risk criteria	p. 32
Identification, definition and rationale of relevant benefit and risk criteria	p. 35
Verification of the list of benefit and risk criteria by means of a survey	p. 59
Review of the Current Benefit-Risk Assessment Models	p. 63
Background	p. 63
Evaluation of the existing benefit-risk assessment models	p. 65
Review of models in single clinical trials and for specific medicines	p. 86
Conclusion	p. 93
Newer models	p. 96
Defining a Systematic Approach to Decision Making	p. 99
Introduction	p. 99
Objectives and features of the ideal model for benefit-risk assessment	p. 100
The use of decision-analysis techniques for the development of the new model	p. 101
Development and Application of a Benefit-Risk Assessment Model Based on Multi-Criteria Decision Analysis	p. 111
Introduction	p. 111
Conceptualization of the new model	p. 111
Reasons for using decision analysis techniques in the new model	p. 113
The use of MCDA in the new model	p. 114
Development of the new model	p. 116
Applicability of the new model	p. 133
Summary	p. 148
Review of the MCDA model	p. 149
A Future Framework for Benefit-Risk Appraisal of Medicines	p. 151
Background	p. 151
Development of a benefit-risk framework for regulatory review of new medicines	p. 151
Prerequisites of a benefit-risk framework for the registration of a new medicine	p. 161
Current status of benefit-risk assessment among companies and agencies	p. 164
Constructing a benefit-risk framework	p. 172
Conclusion	p. 184
Appendices
Summary Reports of the CMR International Institute for Regulatory Science March 2004 and June 2005 Workshops on Benefit-Risk	p. 185
Office of Health Economics Briefing: Challenges and Opportunities for Improving Benefit-risk Assessment of Pharmaceuticals from an Economic Perspective - James Cross and Louis Garrison (August 2008)	p. 207
Reflection Paper on Benefit-risk Assessment Methods in the Context of the Evaluation of Marketing Authorisation Applications of Medicinal Products for Human Use - Committee for Medicinal Products for Human Use (March 2008)	p. 233
Commentaries on 'A Quantitative Approach to Benefit-risk Assessment of Medicines' Pharmacoepidemiology and Drug Safety, 2007, 16	p. 251
Forum on Benefit: Risk Decision Analysis - Summary of Discussions and Recommendations - MHRA (September 2008)	p. 261
References	p. 271
Index	p. 285
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