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Benefit-Risk Appraisal of Medicines: A Systematic Approach to Decision-making / Edition 1

Benefit-Risk Appraisal of Medicines: A Systematic Approach to Decision-making / Edition 1


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Benefit-risk assessment is at the centre of the approval process for every new medicine. The ability to assess the risks of a new medicine accurately and to balance these against the benefits the medicine could bring is critical for every regulatory authority and pharmaceutical company. Despite this there are very few tried and tested evaluative models currently available.

The authors of this book have developed a new, pioneering tool for the assessment of benefits and risks for new medicines in development. This model utilises a multi-criteria decision analysis which involves selecting, scoring and weighting key benefit and risk attributes and leads to an overall appraisal of benefits and risks of medicines.

Benefit-Risk Appraisal of Medicines establishes the background and criteria required to assess benefit and risk in general and reviews the current practices by regulatory authorities and the pharmaceutical industry, including those models currently available. It outlines the development and evaluation of the authors’ new model and analyses the implications of its implementation.

  • Describes an innovative, systematic model which leads to transparent and responsible benefit-risk decision making
  • Contributes important ideas to the debate on benefit-risk appraisal
  • Provides a future framework for benefit-risk appraisal of medicines

Benefit-Risk Appraisal of Medicines covers the entire process from the discovery of new medicines to their marketing and is ideal for all those who work in the pharmaceutical industry and regulatory authorities,, as well as post-graduate students of pharmaceutical medicine and clinical pharmacology.

Product Details

ISBN-13: 9780470060858
Publisher: Wiley
Publication date: 11/16/2009
Pages: 294
Product dimensions: 6.80(w) x 9.80(h) x 0.90(d)

About the Author

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.

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Table of Contents

Foreword vii

Preface ix

1 Concept and Scope of Benefit-Risk Evaluation of Medicines 1

1.1 Historical background 1

1.2 The regulatory systems for assessing medicines 3

1.3 Benefit-risk assessment: definitions 4

1.4 Views and perceptions of benefits and risks of medicines 6

1.5 Stages and concepts in benefit-risk assessment 11

1.6 Benefit-risk assessment: the current regulatory environment 19

1.7 Benefit-risk assessment in other disciplines 19

1.8 Specific methods and models for benefit-risk assessment 21

1.9 Discussions with stakeholders on the concepts and models for benefit-risk evaluation 30

2 Criteria for a Benefit-Risk Model: a Conceptual Framework 31

2.1 Introduction 31

2.2 Regulatory guidelines on benefit and risk criteria 32

2.3 Identification, definition and rationale of relevant benefit and risk criteria 35

2.4 Verification of the list of benefit and risk criteria by means of a survey 59

3 Review of the Current Benefit-Risk Assessment Models 63

3.1 Background 63

3.2 Evaluation of the existing benefit-risk assessment models 65

3.3 Review of models in single clinical trials and for specific medicines 86

3.4 Conclusion 93

3.5 Newer models 96

4 Defining a Systematic Approach to Decision Making 99

4.1 Introduction 99

4.2 Objectives and features of the ideal model for benefit-risk assessment 100

4.3 The use of decision-analysis techniques for the development of the new model 101

5 Development and Application of a Benefit-Risk Assessment Model Based on Multi-Criteria Decision Analysis 111

5.1 Introduction 111

5.2 Conceptualization of the new model 111

5.3 Reasons for using decision analysis techniques in the new model 113

5.4 The use of MCDA in the new model 114

5.5 Development of the new model 116

5.6 Applicability of the new model 133

5.7 Summary 148

5.8 Review of the MCDA model 149

6 A Future Framework for Benefit-Risk Appraisal of Medicines 151

6.1 Background 151

6.2 Development of a benefit-risk framework for regulatory review of new medicines 151

6.3 Prerequisites of a benefit-risk framework for the registration of a new medicine 161

6.4 Current status of benefit-risk assessment among companies and agencies 164

6.5 Constructing a benefit-risk framework 172

6.6 Conclusion 184


Appendix 1 Summary Reports of the CMR International Institute for Regulatory Science March 2004 and June 2005 Workshops on Benefit-Risk 185

Appendix 2 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) 207

Appendix 3 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) 233

Appendix 4 Commentaries on 'A Quantitative Approach to Benefit-risk Assessment of Medicines' Pharmacoepidemiology and Drug Safety, 2007, 16 251

Appendix 5 Forum on Benefit: Risk Decision Analysis - Summary of Discussions and Recommendations - MHRA (September 2008) 261

References 271

Index 285

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