By Lynette Mary Murphy, MBS, BA, Dip BS, Dip OT. A thesis submitted in fulfilment of the requirements for the degree of DOCTOR OF PHILOSOPHY

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1 THE VALUE OF CLINICAL TRIALS: A NEW ZEALAND CASE STUDY By Lynette Mary Murphy, MBS, BA, Dip BS, Dip OT A thesis submitted in fulfilment of the requirements for the degree of DOCTOR OF PHILOSOPHY School of Accounting and Corporate Governance University of Tasmania Tasmania, Australia JULY 2012

2 STATEMENTS AND DECLARATIONS DECLARATION OF ORIGINALITY This thesis contains no material that has been accepted for a degree or diploma by the University or any other institution, except by way of background information and duly acknowledged in the thesis. To the best of my knowledge and belief the thesis contains no material previously published or written by another person except where due acknowledgement is made in the text of the thesis, nor does the thesis contain any material that infringes copyright. AUTHORITY OF ACCESS This thesis may be made available for loan and limited copying in accordance with the Copyright Act 1968 STATEMENT OF ETHICAL CONDUCT The research associated with this thesis abides by the international and Australian codes on human and animal experimentation, the guidelines by the Australian Government's Office of the Gene Technology Regulator and the rulings of the Safety, Ethics and Institutional Biosafety Committees of the University. This study has received ethical approval from the Tasmanian Social Science Human Research Ethics Committee reference number H10522, the New Zealand Northern Y Regional Ethics Committee reference number NTY/09/04/037 and the Manukau Institute of Technology Ethics Committee reference number E09/EXP/19 (nty/09/04/037). Date: 5 July 2012 Page 2 of 297

3 Objective: ABSTRACT The research question addressed in this thesis is - What is the value of conducting sponsored clinical trials in a publicly funded New Zealand hospital? Methods: The research design is a simultaneous parallel mixed method design that incorporates two strands (1) A quantitative analysis of economic outcomes and (2) a qualitative analysis of perceived value. In the first strand, quantitative methods draw on the data relating to two sponsored clinical trials. The data include Ministry of Health data, The Centre for Clinical Research and Effective Practice (CCRep) profit and loss statements, Counties Manukau District Health Board (CMDHB) annual reports and Chronic Care Management (CCM) data and results from a health outcome co-study. The second strand uses qualitative methods to explore the benefits and costs of sponsored clinical trials perceived by stakeholders. The study gathers data using focus groups, interviews and surveys and adopts a qualitative descriptive approach followed by a phenomenographical analysis. Results: The economics outcomes strand finds that CCRep, CMDHB and New Zealand society all derive financial benefits from these trials. The magnitude of the economic benefits differs depending on the perspective taken. Both CCRep and CMDHB have benefits that are positive but small. The largest and potentially the most controversial benefit is a benefit to New Zealand society of over 373,000 dollars. The qualitative results suggest that the benefits of conducting sponsored clinical trials within a publicly funded New Zealand hospital outweigh the costs in respect of all stakeholder groups. The results allow classification of the stakeholders into three layers: societal; where benefits and costs are filtered by political and social opinions; organisational; where benefits and costs are seen in terms of their influence on organisational functions and personal; where benefits and costs are seen as contributing to the psycho-social, cognitive, physical and behavioural needs of individuals. Conclusion: Public bodies must be mindful of the wider economic, social and cultural implications of their activities. This study demonstrates the value created from conducting clinical trials. The adoption of qualitative and quantitative methods to measure this value produces a more rounded analysis than would be the product of either approach on its own. Page 3 of 297

4 ACKNOWLEDGEMENTS Many people have contributed in bountiful and generous ways to my journey that has culminated in this finished work. I am indebted to my supervisor, Dr William Maguire for his confidence in my capacity to achieve my goal, his rigorous academic critique, and his friendship, patience and generous donation of his time throughout the period of my candidature. I am grateful also to my co-supervisor Dr Willem Fourie for his expertise in his field and his willingness to provide the right support at the right time. My two supervisors provided in-depth knowledge in their respective fields, which combined to produce the type of supervision that I needed to complete this thesis. I have had wonderful guidance and support from the staff within the School of Accounting and Corporate Governance at the University of Tasmania. I also thank the Manukau Institute of Technology for their generous research grant, which allowed me the time I needed to complete this study. To my colleague Terry Quilt who not only provided technical website advice but also was a constant source of encouragement I say thank you. I am particularly indebted to the staff of the Centre for Clinical Research and Effective Practice (CCRep) especially Dr Stuart, Ryan general manager and Dr John Baker, clinical director and Irene Zing wh o gave up their time to answer my questions and to assist me in my search for, or collection of organisational data. I wish to thank all of the people who assisted me in the course of my fieldwork. Their preparedness to give up their time to participate by means of focus groups, interviews and survey completion and to assist me in my collection of data has made this thesis possible. There have also been innumerable people from many organisations, including government departments, pharmaceutical industries and research organisations that have provided or guided me in locating much of the necessary background data for this study. Finally, and above all others, I am deeply grateful to the patience and loving support of my husband, Patrick and children Samuel, Hannah and Leah and for their ability to provide balance and where necessary healthy distraction throughout the journey of my thesis. Page 4 of 297

5 CONTENTS Statements and Declarations... 2 Declaration of originality... 2 Authority of access... 2 Statement of ethical conduct... 2 Abstract... 3 Acknowledgements... 4 List of Abbreviations Introduction to the Study...11 The nature of value...11 Value and health...12 The empirical study...13 Micro, meso and macro level analysis...16 Study context...17 Motivation for the research...22 Contribution to public and social policy...22 Development of theory and method...26 Statement of reflexivity...27 Conclusion Literature Review...29 Value and its measurement...29 The concept and theory of value...29 Value in healthcare...36 Cost measurement in health care...39 Economic methods of measuring value...41 Merging perspectives on value...50 Stakeholder perspectives on value...52 Conclusion Review of the Empirical Research...59 The benefits and costs for trial participants...59 The benefits and costs for family and care givers...64 The benefits and costs for staff...65 The benefits and costs for pharmaceutical companies...69 The benefits and costs for DHBs...73 The benefits and costs for New Zealanders...80 The benefits and costs for the international community...82 The value of clinical trials: Theoretical models...83 Macro-level models...83 A societal perspective Page 5 of 297

6 4. Micro-level models...86 Conclusion...89 Research Methods...95 Study overview...96 Worldview...97 Methodology Research design Research methods Economic outcomes strand Economic evaluation Data collection Spreadsheet-based multiple account approach Research design Procedures Discount rate and sensitivity analysis Multiple stakeholder perceptions strand Data collection Sampling methods Trial participants Family members and care givers DHB staff members, Researchers The Counties Manukau Community Government bodies and politicians, Pharmaceutical industry Procedures used Data analysis Ethical issues Summary and conclusion Results from the Economic Outcomes Strand Micro-level: Benefit cost analysis from the perspective of CCRep Costs Revenue Cost and revenue streams Meso-level: Benefit cost analysis from the perspective of CMDHB Pharmaceuticals Laboratory tests Chronic Care Management costs (CCM) Page 6 of 297

7 Combined savings from cost avoidance Indirect costs funded by CMDHB Results of benefit cost analysis CMDHB Macro-level: Benefit cost analysis from the perspective of New Zealand society Conclusion Qualitative Results from the Multiple Stakeholder Perceptions Strand The research informants The benefits and costs for trial participants The benefits and costs for families and caregivers The benefits and costs to CMDHB researchers and staff The benefits and costs for pharmaceutical companies The benefits and costs for the DHB The benefits and costs for the New Zealand community The benefits and costs for the international community Emerging themes Phenomenographical analysis Perceptions across the stakeholder groups The distribution of benefits and costs Maori Researchers not involved in sponsored clinical trials Benefits for the rich and for pharmaceutical companies Ko Awatea Conclusion Conclusion Restatement of the problem Review of the emperical study Summary and discussion: Results from economic outcomes strand Summary and discussion: Results from multiple stakeholder perceptions strand Differences in perceptions The distribution of benefits and costs Contributions The application of BCA in accounting The measurement of value in healthcare The use of mixed methods Establishing the foundations for a social report Practical implications Strengths of the study Further research Internal validity External validity Conclusion Page 7 of 297