Abstract: In the public hospital sector in Australia there is no dedicated scheme to offset costs associated with high cost medications (HCMs) to the institution or the public. (1) Concerns exist as to the equity of access and appropriate mechanisms to manage access to HCMs in public hospitals. (2) There are gaps in the literature as to how decisions are made, and in particular, decision-making processes by which ethical, clinical and economic considerations maybe taken into account. To date, limited work has been conducted regarding the use and funding of HCMs in public hospitals. There are no published data on perceptions, concerns and attitudes, among health care decision-makers or among the community-at-large about access to HCMs in public hospitals. The research reported in this thesis describes the decision-making process and criteria used by health care decision-makers to allocate resources to HCMs in public hospitals. The investigation triangulated quantitative and qualitative methods used to collect and analyse data. Four studies were conducted to describe the decision-making process and explore the perceptions, concerns and attitudes of health care decision-makers and the perceptions of members of the general public regarding access to HCMs in public hospitals. The first study, reported in Chapter Three, was a review of individual patient use (IPU) requests for non-formulary HCMs. This study showed that these requests had a significant impact on the capped expenditure of a public hospital. Subsequent to this review, a new policy and procedure for managing requests for HCMs for IPU was established. A high-cost drugs subcommittee (HCD-SC) operating under the auspices of the Drug and Therapeutics Committee (DTC) was created. The second study, reported in Chapter Four, described the operations of the newly formed HCD-SC. This study also evaluated the decision-making process using the ethical framework “accountability for reasonableness”. (3) Different factors were involved in decisions about access to HCMs and decisions were not solely based on effectiveness and cost. HCD-SC members considered it was important to have consistency in the way decisions were being made. The evaluation of this process allowed identification of good practices and gaps which were considered as opportunities for improvement. The third study, reported in Chapter Five, found that health care decision-makers in an Area Health Service echoed the concerns and agreed about the problems associated with access to HCMs expressed by the HCD-SC members. These studies concluded that the majority of decision-makers wanted an explicit, systematic process to allocate resources to HCMs. These studies also identified tensions between funding systems and hospital decision-making. According to participants there were no mechanisms in place to systematically capture, analyse and share the lessons learned between the macro level (ie. Federal, Pharmaceutical Benefits Scheme - PBS) and the meso level (ie. Institution, public hospital) regarding funding for HCMs. Furthermore, decision-makers considered there are strong incentives for cost-shifting between the Commonwealth and the States. Health care decision-makers also acknowledged the importance of public participation in decision-making regarding allocation of resources to HCMs in public hospitals. However the results of these studies showed that those decisions were not generally made in consultation with the community. Decision-makers perceived that the general public does not have good general knowledge about access to HCMs in public hospitals. A survey of members of the general public, reported in Chapter Six, was then conducted. The survey aimed to gather information about the knowledge and views of members of the general public about access to HCMs in public hospitals. Results of this fourth study showed that respondents had good general knowledge but were poorly informed about the specifics of funding of hospitals and HCMs in private and public hospitals. The results also offered support for the development of a process to involve community members in discussion on policy on the provision of treatment and services within health care institutions and specifically, to seek the views of members of the public on the provision of HCMs and expensive services within public hospitals. In summary, the research reported in this thesis has addressed the gaps in the literature as to how decisions are made, and in particular, the decision-making process and criteria used by health care decision-makers to allocate resources to HCMs in public hospitals. In a move towards more explicitness in decision-making regarding the allocation of scarce health care resources, the findings from these studies provide an evidence base for developing strategies to improve decision-making processes regarding access to HCMs the public sector Table of Contents STATEMENT OF ORIGINALITY ACKNOWLEDGEMENTS COMMUNICATIONS ARISING FROM THIS THESIS ABSTRACT LIST OF FIGURES LIST OF APPENDICES GLOSSARY OF ABBREVIATIONS PREFACE. 1. THE AUSTRALIAN HEALTH CARE SYSTEM AND HIGH COST MEDICATIONS (HCMS) 1.1 Introduction 1.2 The Australian Health Care System 1.3 Pharmaceuticals 1.3.1 Availability of Pharmaceuticals in Australia 1.3.2 Registration 1.3.3 Subsidy 1.3.3.1 The Pharmaceutical Benefits Scheme (PBS 1.3.3.1.1 Section 85 1.3.3.1.2 Section 100 Highly Specialised Drug Program (S100-HSD 1.3.4 Other Categories of Access 1.3.4.1 Orphan Medications 1.3.4.2 Lifesaving Medications 1.3.4.3 Unapproved medications 1.3.4.3.1 Special Access Scheme (SAS 1.3.4.3.2 Clinical Trials (CTN and CTX Schemes) 1.3.4.3.3 Importation for personal use 1.3.5 Hospitals 1.3.5.1 Access to pharmaceuticals in private hospitals 1.3.5.2 Access to pharmaceuticals in public hospitals 1.4 High Cost Medications (HCMs 1.4.1 Definitions of HCMs in Australia 2. DECISION-MAKING AND PRIORITY SETTING IN HEALTH CARE 2.1 Introduction 2.2 Levels of Decision-making priority setting in health care 2.2.1 Macro – Government level 2.2.2 Meso – Hospital or Institution 2.2.2.1 Hospital Drug and Therapeutics Committees (DTCs) 2.2.2.2 Australian Hospital Drug and Therapeutics Committees 2.2.3 Micro- Individual 2.3 Decision-making priority setting in health care and the general public 2.4 Decision-making priority setting in health care and ethics. 3. THE FINANCIAL IMPACT OF APPROVAL OF MEDICATIONS FOR INDIVIDUAL PATIENT USE (IPU) IN A PUBLIC HOSPITAL 3.1 Introduction 3.2 Aim 3.2.1 Methods 3.2.1.1 Setting 3.2.2 Data Collection 3.2.3 Data Analysis and Statistics 3.3 Results 3.4 Discussion 4. ALLOCATING RESOURCES TO HIGH COST MEDICATIONS (HCMS) IN PUBLIC HOSPITALS. THE ROLE OF A HIGH COST DRUG SUBCOMMITTEE (HCD-SC 4.1 Introduction 4.2 Aim 4.3 Methods 4.3.1 Selection of methods 4.3.1.1 Quantitative research 4.3.1.2 Qualitative research 4.3.1.3 Case study 4.3.1.3.1 Triangulation in Case Studies 4.3.1.3.2 Semi-structured Interviews in Case Studies 4.3.1.3.3 Direct Observations in Case Studies 4.3.1.3.4 Document Review in Case Studies 4.3.2 Data Collection 4.3.2.1 Ethical considerations 4.3.3 Data analysis 4.3.3.1 Demographic data 4.3.3.2 Transcription and thematic analysis 4.3.3.3 Validity 4.3.3.4 Reliability 4.4 Results 4.4.1 Document Review and Observations 4.4.2 Interviews 4.5 Discussion 5. DECISION-MAKERS’ VIEWS ABOUT ACCESS TO HIGH COST MEDICATIONS (HCMS) IN PUBLIC HOSPITALS. 5.1 Introduction 5.2 Aims and objectives 5.3 Methods 5.3.1 Selection of methods 5.3.1.1 Grounded Theory 5.3.1.2 In-depth interviews 5.3.2 Setting 5.3.3 Sampling 5.3.4 Recruitment 5.3.5 Data collection 5.3.5.1 Interview Process 5.3.5.2 Demographic characteristics 5.3.6 Data analysis 5.3.6.1 Qualitative data 5.3.6.2 Validity and reliability 5.3.6.3 Quantitative data 5.3.7 Ethics 5.3.8 Funding 5.4 Results 5.4.1 Study participants 5.4.2 Themes 5.4.2.1 High Cost Medications 5.4.2.2 Cost 5.4.2.3 The decision-making process 5.4.2.3.1 Consistency and Transparency 5.4.2.4 Criteria 5.4.2.5 Problems and concerns 5.4.2.6 Solutions 5.5 Discussion 5.5.1 High Cost Medications 5.5.2 The decision-making process 5.5.2.1 Consistency and Transparency 5.5.3 Criteria 5.5.4 Problems and concerns 5.5.5 Solutions 6. ACCESS TO HIGH COST MEDICATIONS (HCMS) IN PUBLIC HOSPITALS. PERCEPTIONS OF MEMBERS OF THE GENERAL PUBLIC. 6.1 Introduction 6.2 Aim 6.3 Methods 6.3.1 Selection of Method 6.3.1.1 Self-administered surveys 6.3.2 Sample selection 6.3.2.1 Sample size 6.3.3 Eligibility criteria 6.3.4 Questionnaire development 6.3.5 Pilot testing 6.4 Data Collection and Analysis 6.4.1 Quantitative data 6.4.2 Qualitative data 6.5 Ethics 6.6 Results 6.6.1 Demographics 6.6.2 Health and health services 6.6.3 Knowledge about access to HCMs in public hospitals 6.6.4 Factors & choices 6.7 Discussion 6.7.1 Knowledge 6.7.2 Factors and choices 6.7.3 Demographics 7. SUMMARY AND FUTURE DIRECTIONS REFERENCES LIST OF APPENDICES Access to High Cost Medicines in Australian Hospitals (Tiếp cận với thuốc Chi phí cao trong các bệnh viện Úc)
