Current PhD students
Rafael de Feria Cardet
Surrogate outcomes for the decision-making process in Prostate Cancer: an economic evaluation
Prostate cancer is the most commonly diagnosed cancer in men globally. In Australia, it is the most commonly diagnosed cancer in males, expecting to reach 19,508 new cases in 2019 and for every 5 men diagnosed, at least one will die at an advanced stage of the disease.
Improving mortality and morbidity from prostate cancer could be achieved with more effective treatments in early stage disease. However, for new treatments to become available in countries like Australia, they must undergo health technology assessment (HTA) which generally requires efficacy data from clinical trials to measure their impact on overall survival as the primary outcome of interest. Considering the natural history of prostate cancer, this may mean that studies require long follow-ups - on average 11.5 years – to show survival outcomes.
The use of surrogate outcomes – endpoints that strongly predict for survival - may play an important role for making treatments available for prostate cancer patients as they may potentially shorten the time it takes for drug approval. One potential surrogate is being free of metastatic disease at five years, identified in the literature as a strong predictor of longer-term survival.
If validated within the HTA process, it may become a useful tool to support decision-making and introduce treatments into practice sooner. Early access to cost-effective treatments may offset the higher costs of treating advanced or metastatic stages of prostate disease and potentially reduce productivity losses.
In collaboration with ANZUP and the Intermediate Clinical Endpoints in Cancer of the Prostate (ICECaP), this project will focus on how surrogate measures of outcomes, like five-year metastasis free survival, may be used to demonstrate to decision makers, how new treatments represent value for money. It will also consider the potential trade-offs of society when deciding to make a treatment available sooner based on surrogate outcomes. The main objectives of the projects are:
- To evaluate and validate an acceptable surrogate outcome as the primary indicator of effectiveness for its use in economic evaluation and health technology assessment according to PBAC guidelines.
- To develop an economic model capable of evaluating the comparative costs and benefits of bringing an intervention to clinical practice early based on a valid surrogate outcome compared to waiting for overall survival data.
This work is supported by untied funding received from Astellas Pharma Singapore Pty Ltd and Janssen Global, and is coordinated by the Australian and New Zealand Urogenital and Prostate (ANZUP) Cancer Trials Group.
Supervisors: A/Prof Richard De Abreu Lourenco, Prof Stephen Goodall, Prof Marion Haas
The impact of the chronic disease dental scheme
Australia’s Medicare provides universal health insurance for all Australians, including certain visa-class holders. While this scheme allows for a limited coverage of dental services, most dental services are funded privately through private insurance or through individual out-of-pocket payments. In 2007, the Federal Government introduced the the Chronic Disease Dental Scheme (CDDS) to provide subsidised dental services through the Medicare program to those with a chronic or complex condition. Ultimately, the expense associated with this program and criticisms in relation to its implementation resulted in its closure in 2013. This PhD will assess this program in terms of its impact on dental utilisation and dental health outcomes for the target population as well as to provide insight into the population who received the program.
Is there a need for a new framework to assess cost-effectiveness of cell and gene therapies in Australia?
There is debate as to whether the current approach to Health Technology Assessment (HTA) in Australia is appropriate to guide funding decisions for emerging cell and gene therapies. Decisions to publicly fund health technologies in Australia are based on an assessment of evidence on safety, clinical and cost-effectiveness, considered by expert advisory committees; the Pharmaceutical Benefits Advisory Committee (PBAC) for pharmaceuticals, and the Medical Services Advisory Committee (MSAC), for medical services. PBAC and MSAC Guidelines have an emphasis on data from randomised controlled trials (RCTs) to assess comparative health gain and incremental cost-effectiveness, with a reasonable level of certainty.
Due to the potentially curative nature of cell and gene therapies and the ethical issues associated with conducting an RCT for life-threatening conditions where there may be no effective alternative, evidence is likely to be generated from early phase, single arm studies. The evidentiary requirements to register therapies with the potential to be life-saving may also differ, with regulators often accepting of surrogate based outcomes, versus a preference for final outcomes (particularly overall survival) by HTA committees. While the lack of comparative, long-term evidence is a common issue for many therapies for the treatment of life-threatening diseases, cell and gene therapies present a unique set of challenges due to high, upfront costs as a result of the potential for one-off dosing and the requirement for extensive hospital infrastructure. There is a need for alternative approaches to dealing with uncertainty associated with modelling long-term cost-effectiveness of such therapies.
The primary aims of this research are to explore the impact of different modelling techniques to assess the cost-effectiveness of a cell and gene therapy, with a view to developing a best-practice approach for dealing with uncertainty. In addition, different payment arrangement options as a mechanism for dealing with uncertainty and high up-front budgetary implications will be explored.
This work is being undertaken as part of the UTS Industry Doctorate Program, in collaboration with CHERE and Novartis Pharmaceuticals.
Elena is a researcher in Health Economics with an interest in economic evaluation and the use of discrete choice experiments in the analysis and prediction of behaviour in the health care market.
During her PhD, Elena will be investigating the influence of brand premiums on consumers’ choice for prescription medicines in Australia. She will be applying discrete choice experiment methods and techniques to measure the effect of different factors on demand for medicines.
Supervisors: Prof Stephen Goodall, Prof Deborah Street, Prof Rosalie Viney
The cost effectiveness of advanced heart failure management and mechanical circulatory assist therapy
Heart failure is a chronic condition that affects 1.5-2.0% of Australians. It is estimated to cost the economy more than $1 billion annually. For those at the most severe end of the heart failure spectrum, re-hospitalisations are common and costs are compounded by expensive treatment options including transplantation and mechanical circulatory support through the implantation of left ventricular assist devices (LVADs).
The technology is advancing quickly with newer continuous flow devices showing fewer complications and better survival, and the demand for LVAD therapy is likely to rise as the population ages and a significant proportion face the reality of living with advanced heart failure.
No studies have examined cost effectiveness of LVADs in the Australian context, and we know very little about the actual health resource use of patients with advanced heart failure (particularly in the 12 months prior to LVAD implantation).
This study will assess the cost effectiveness of LVAD therapy in an Australian health care setting and explore the development of utility measures that will capture the experience of living with an implantable mechanical support device.
The retrospective component will compare the costs generated by patients with LVADs with the costs for those who receive optimum medical management only. The prospective cohort study will capture the journey of patients from first presentation to a quaternary hospital through four possible treatment pathways, using clinical, frailty and utility outcome measures to generate a comprehensive cost effectiveness evaluation of mechanical heart failure therapy.
(Enrolled Faculty of Health, UTS)
Modelling the cost-effectiveness of strategies to treat end-stage heart failure using discrete event simulation
Economic evaluation is performed to help make better resource allocation decisions within the health care system. Health technology assessment guidelines and methods had first been established in the context of pharmaceutical reimbursement. Its application to medical devices presents some challenges including the lack of quantity and quality of evidence (e.g limited randomised controlled trials and reliance on observational data). Other challenges include the learning curve associated with medical devices, particularly, implantable devices and the incremental improvements of devices over time. In addition to this, operational factors such as restricted supply (either due to high cost or restricted indication) all influence the cost effectiveness of implantable medical devices.
The PhD will explore these complexities using a case-study of treatment options for patients with end-stage heart failure (ESHF). In the past, patients have relied on heart transplants. However, with decreasing donor hearts available, clinicians are looking towards other treatment strategies including mechanical circulatory support in the form of ventricular assist devices (VADs). VADs represent an opportunity for patients with ESHF to be given a VAD whilst waiting for a donor heart to become available. Overseas, VADs are becoming long-term option for patients not being considered for heart transplants, that is, until death.
This PhD aims to conduct an economic evaluation of the treatment strategies of ESHF in Australia. The PhD will explore the application of advanced modelling methods to address the resource capacity constraints associated with life-saving treatments in ESHF. This project will also review methods to incorporate observational data into measures of effectiveness. The project will use individual patient data on costs and outcomes from St. Vincents Hospital.
Supervisors: Prof Stephen Goodall, Dr Naomi van der Linden1, Prof Christopher Hayward2
1. AstraZeneca, The Netherlands
2. St Vincent's Hospital
Health economics of anxiety and depression in cancer patients
Anxiety and depression are the third largest causes of disability in Australia, and rates of anxiety and depression are significantly higher in cancer patients than in the general population. It is estimated that over 20% of adults with cancer have a mood disorder. Anxiety and depression are often undetected and underestimated in busy cancer services, and high unmet need for psychosocial care is persistent in cancer patients. This is surprising, given the large evidence-base that interventions for anxiety and depression in patients with cancer are effective in the short and long-term. In Australia, patterns of referral, treatment and follow-up of detected anxiety and depression vary greatly across services.
The Psycho-Oncology Co-operative Research Group (PoCoG) has recently developed a Clinical Pathway for the management of anxiety and depression in adults with cancer, the first of its kind internationally. The Anxiety and Depression Pathway Program (ADAPT), funded through a Translation Program Grant awarded by the Cancer Institute NSW, is a randomised cluster controlled trial assessing the efficacy of core versus enhanced strategies for the implementation of a clinical care pathway for anxiety and depression in cancer patients.
The aim of this PhD research program is to determine the economic impact of anxiety and depression in adult cancer patients to health care systems. There is currently limited literature regarding the costs and/or heath care resource use among cancer patients diagnosed with anxiety and depression. Using the ADAPT study, this research will analyse the costs associated with health service use pre and post implementation of the ADAPT pathway, as well as evaluating the cost effectiveness of different implementation strategies.
The program of work is divided into four projects, with the following objectives:
1. To undertake a literature review of evidence around anxiety and depression in adults with cancer, including health service use, cost effectiveness of interventions, and cost effectiveness of alternative strategies to implement interventions.
2. To analyse the impact of anxiety and depression on health service use by using linked administrative data and baseline ADAPT study data.
3. To compare the cost effectiveness of two approaches (core vs enhanced) for the implementation of the Australian Clinical Pathway for the Identification and Management of Anxiety and Depression (the ADAPT study) using health economic methods
4. Measuring preferences of cancer patients for the identification and management of anxiety and depression using discrete choice experiment methods.
Alice’s PhD focuses on quality of life and in understanding consumer/patient preferences
Alice is currently in the second year of the Health Economics PhD Program at UTS and has been awarded the UTS Chancellors Research Scholarship. Alice’s PhD focuses on quality of life and in understanding consumer/patient preferences. She is also interested in the design and application of discrete choice experiments (DCEs) in health economics. Currently, she is conducting a DCE in collaboration with a team of clinicians in order to gain an understanding of patient preferences for the assessment of peripheral neuropathy that may result from chemotherapy treatment for cancer.