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



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

Filip Mussen, Sam Salek, Stuart Walker

ISBN: 978-0-470-74812-1 August 2009 304 Pages

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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.



1 Concept and Scope of Benefit–Risk Evaluation of Medicines.

1.1 Historical backgroun.

1.2 The regulatory systems for assessing medicines.

1.3 Benefit–risk assessment: definitions.

1.4 Views and perceptions of benefits and risks of medicines.

1.5 Stages and concepts in benefit–risk assessment.

1.6 Benefit–risk assessment: the current regulatory environment.

1.7 Benefit–risk assessment in other disciplines.

1.8 Specific methods and models for benefit–risk assessment.

1.9 Discussions with stakeholders on the concepts and models for benefit–risk evaluation.

2 Criteria for a Benefit–Risk Model: a Conceptual Framework.

2.1 Introduction.

2.2 Regulatory guidelines on benefit and risk criteria.

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

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

3 Review of the Current Benefit–Risk Assessment Models.

3.1 Background.

3.2 Evaluation of the existing benefit–risk assessment models.

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

3.4 Conclusion.

3.5 Newer models.

4 Defining a Systematic Approach to Decision Making.

4.1 Introduction.

4.2 Objectives and features of the ideal model for benefit–risk assessment.

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

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

5.1 Introduction.

5.2 Conceptualization of the new model.

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

5.4 The use of MCDA in the new model.

5.5 Development of the new model.

5.6 Applicability of the new model.

5.7 Summary.

5.8 Review of the MCDA model.

6 A Future Framework for Benefit–Risk Appraisal of Medicines.

6.1 Background.

6.2 Development of a benefit–risk framework for regulatory review of new medicines.

6.3 Prerequisites of a benefit–risk framework for the registration of a new medicine.

6.4 Current status of benefit–risk assessment among companies and agencies.

6.5 Constructing a benefit–risk framework.

6.6 Conclusion.


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

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).

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).

Appendix 4 Commentaries on ‘A Quantitative Approach to Benefit–risk Assessment of Medicines’ Pharmacoepidemiology and Drug Safety, 2007, 16.

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