Benefit-Risk Appraisal of Medicines

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.

Autorentext

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.

Zusammenfassung
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.

Inhalt

Foreword.

Preface.

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.

Appendices.

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).

References.

Index.