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

Siobhan Dickinson

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.

Supervisors: Prof Jane Hall, Prof Kees van Gool, Dr Maryam Naghsh Nejad

Maria (Mish) Farris

The cell and gene (C&G) therapy area of pharmaceutical development is in a period of rapid and ongoing innovation. These new therapeutics may offer substantial benefits to patients and often target previously difficult to treat or untreatable diseases. The curative potential of these therapies gives them the scope to dramatically improve the health status and quality of life of patients and caregivers.

Given the possible benefits that a potentially curative single administration therapy can provide, C&G therapies can be expected to improve patient quality of life in many ways such as lowering the number of hospitalisations or chronic care and may increase patients’ expected longevity. A curative treatment can also lead to considerable savings (“cost-offsets”) in terms of long-term social care costs, as well as benefits for the immediate family, caregivers, and wider society. C&G therapies therefore present the possibility of substantial clinical and economic benefits.

The benefits from any potentially curative new therapies—in terms of savings in long-term social care costs, as well as financially positive effects for immediate family members, caregivers and wider society—could be transformative. Furthermore, the ‘health catastrophe’ element of diseases that C&G therapies typically treat may make them considered to be more valuable in societal opinion.

However such specialised therapies are generally associated with relatively high costs compared with existing therapies, and may lack, long-term comparative clinical evidence or the relative benefits from treatment on the caregiver and more broadly. This presents a challenge for the Australian health care system that applies a rigorous Health Technology Assessment (HTA) process, requiring high-level, comparative clinical evidence and an assessment of cost-effectiveness versus existing treatments, to make decisions on whether to nationally fund new therapies.

Incorporating broader value elements that are deemed important by decision-makers and by society would allow for more informed decision choices. Considering these elements (for C&G therapies but also for all other therapies) in an explicit manner would provide greater transparency and consistency in the appraisal.

The primary aim of this research is to research different modelling techniques to assess the cost-effectiveness of gene therapies, with a view to developing a best-practice approach to incorporate the broader value of gene therapies, to facilitate Government funding decisions.

This work is being undertaken as part of the UTS Industry Doctorate Program, in collaboration with CHERE and Novartis Pharmaceuticals.

Supervisors: A/Prof Richard De Abreu Lourenco, Prof Stephen Goodall, Dr Mark Schulz (Novartis)

Amy Gye

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.

Supervisors: A/Prof Richard De Abreu Lourenco, Prof Stephen Goodall, Dr Mark Schulz (Novartis)

Yiting Luo

Testing the method of valuing paediatric Health-Related Quality of Life (HRQoL) measures

The health-related quality of life (HRQoL) describes an individual’s or a group of people’s physical and mental health and it can be used to generate the quality-adjusted life-years (QALYs) which is a measure of health outcomes for use in economic evaluation.  QALYs incorporate both quantity and quality of life and the measurements of QALYs help address clinical and public policy questions through their application in cost-effectiveness analyses (CEAs).

Preference-based measures of HRQoL for adults have been well discussed and studied. However, paediatric HRQoL measures represent some challenges when children are required to complete the valuation tasks because the extent to which children are able to understand the tasks can be impacted by their ages, development stages, educational abilities, and experience of ill health. This PhD will use both qualitative and quantitative methods to explore how children/adults value  paediatric preference-based measures.

Supervisors: Prof Rosalie Viney, Prof Deborah Street, A/Prof Brendan Mulhern

Elena Meshcheriakova

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

Kittiphong Thiboonboon

Kittiphong is investigating an impact of a tax on sugar-sweetened beverages (SSBs) on economic and health outcomes in Thailand. He also has an interest that whether the tax will improve inequity in health across populations in Thailand. In his study, he will examine lesson learnt from countries that have already implemented the tax and how that could be applicable to the case in Thailand. As part of his study, he aims to build a microsimulation model to observe health and economic impact affected by the tax in Thailand.

Supervisors: Prof Stephen Goodall, A/Prof Richard De Abreu Lourenco, Dr Paula Cronin