Maarten IJzerman

University of Melbourne | MSD · Melbourne School of Population Health

Background: Clinical indications for ibrutinib reimbursement in Australia should consider the inclusion of patients with chronic lymphocytic leukemia (CLL) harboring prognostically unfavorable TP53/IGHV genomic aberrations. This study assessed the cost effectiveness of five first-line treatment strategies in CLL for young (aged ≤ 65 years), fit pa...

Background: Patients treated with immune checkpoint inhibitors (ICI) are at risk of adverse events (AEs) even though not all patients will benefit. Serum tumor markers (STMs) are known to reflect tumor activity and might therefore be useful to predict response, guide treatment decisions and thereby prevent AEs. Objective: This study aims to comp...

Background This systematic review and meta-analysis aimed to evaluate existing evidence on the relationship between diagnostic and treatment intervals and outcomes for colorectal cancer. Methods Four databases were searched for English language articles assessing the role of time before initial treatment in colorectal cancer on any outcome, includ...

Objective Approximately one third of children with JIA receive biologic therapy, but evidence on biologic therapy withdrawal is lacking. This study aims to increase our understanding of whether and when pediatric rheumatologists postpone a decision to withdraw biologic therapy in children with clinically inactive non-systemic JIA. Methods A survey...

Background: Despite global support, there remain gaps in the integration of early palliative care into cancer care. The methods of implementation whereby evidence of benefits of palliative care is translated into practice deserve attention. Aim: To identify implementation frameworks utilised in integrated palliative care in hospital-based oncolo...

Background: Cancer-derived material circulating in the bloodstream and other bodily fluids, referred to as liquid biopsies (LBs), has become an appealing adjunct or alternative to tissue biopsies, showing vital promise in several clinical applications. Purpose: A systematic literature review was conducted to (1) summarize the current health econ...

Objectives: Care for JIA patients has been transformed in the biologics era; however, biologics carry important (although rare) risks and are costly. Flares after biological withdrawal are seen frequently, yet there is little clinical guidance to identify which patients in clinical remission can safely have their biologic discontinued (by stopping...

NTRK gene fusions are rare somatic mutations found across cancer types with promising targeted therapies emerging. Healthcare systems face significant challenges in integrating these treatments, with uncertainty in prevalence and optimal testing methods to identify eligible patients. We performed a systematic review of NTRK fusion prevalence to inf...

Objective The aims of this study was to appraise the health economic evidence for adjuvant chemotherapy (AC) strategies in stage II and III colon cancer (CC) and identify gaps in the available evidence that might inform further research. Method A systematic review of published economic evaluations was undertaken. Four databases were searched and f...

Research focusing on timely diagnosis and treatment of colorectal cancer is necessary to improve outcomes for people with cancer. Previous attempts to consolidate research on time to diagnosis and treatment have noted varied methodological approaches and quality, limiting the comparability of findings. This systematic review was conducted to compre...

Background Chest low-dose computed tomography (LDCT) is a promising technology for population-based screening because it is non-invasive, relatively inexpensive, associated with low radiation and highly sensitive to lung cancer. To improve the cost-effectiveness of lung cancer screening, simultaneous screening for other diseases could be considered...

Introduction: Health economic outcomes of real-world treatment sequencing of androgen receptor-targeted agents (ARTA) and docetaxel (DOC) remain unclear. Material and methods: Data from the electronic Castration-resistant Prostate cancer Australian Database (ePAD) were analysed including median overall survival (mOS) and median time to treatment...

Background Diabetes mellitus, cardiovascular diseases, chronic kidney disease, and thyroid diseases are chronic diseases that require regular monitoring through blood tests. This paper first investigates the experiences of chronic care patients with venipuncture and their expectations of an at-home blood-sampling device, and then assesses the impac...

Background: Demonstrating safety and efficacy of new medical treatments requires clinical trials. But clinical trials are costly and may not provide value proportionate to their costs. In health systems with limited resources, it is important to identify the trials with the highest value. Tools exist to assess elements of a clinical trial such as s...

PURPOSE The combination of whole-genome and transcriptome sequencing (WGTS) is expected to transform diagnosis and treatment for patients with cancer. WGTS is a comprehensive precision diagnostic test that is starting to replace the standard of care for oncology molecular testing in health care systems around the world; however, the implementation...

Immune checkpoint inhibitors have been approved in the USA for tumours exhibiting mismatch repair deficiency (dMMR), microsatellite instability (MSI), or high tumour mutational burden (TMB), with regulatory and reimbursement applications in multiple other countries underway. As the estimated budget impacts of future reimbursements depend on the siz...

Biomarker testing is a laboratory test in oncology that is used in the selection of targeted cancer treatments and helping to avoid ineffective treatments. There exist several types of biomarker tests that can be used to detect the presence of particular mutations or variation in gene expression. Whole Genome Sequencing (WGS) is a biomarker test fo...

Serum tumor markers acquired through a blood draw are known to reflect tumor activity. Their non-invasive nature allows for more frequent testing compared to traditional imaging methods used for response evaluations. Our study aims to compare nine prediction methods to accurately, and with a low false positive rate, predict progressive disease desp...

Background Complex genomic profiling (CGP) has transformed cancer treatment decision making, yet there is a lack of robust and quantifiable evidence for how utilisation of CGP improves patient outcomes.Objective This study evaluated cohort level clinical effectiveness of CGP to improve overall survival (OS) in real-world advanced cancer patients us...

Introduction Drug reimbursement decisions are often made based on a price set by the manufacturer. In some cases, this price leads to public and scientific debates about whether its level can be justified in relation to its costs, including those related to research and development (R&D) and manufacturing. Such considerations could enter the decisi...

Objective This study investigates the relationship of child, caregiver, and caring context measurements with the care-related quality of life (CRQoL) and health-related quality of life (HRQoL) of caregivers of children with juvenile idiopathic arthritis (JIA). Methods We performed a cross-sectional analysis of baseline data on caregivers of childr...

Objective: The aims of this study was to appraise the health economic evidence for adjuvant chemotherapy (AC) strategies in stage II and III colorectal cancer (CRC) and to identify gaps in the available evidence that might inform further research. Method : A systematic review of published economic evaluations was undertaken. Four databases were sea...

Advances in machine learning (ML) and artificial intelligence offer tremendous potential benefits to patients. Predictive analytics using ML are already widely used in healthcare operations and care delivery, but how can ML be used for health economics and outcomes research (HEOR)? To answer this question, ISPOR established an emerging good practic...

Background: Early reports suggested that COVID-19 patients with cancer were at higher risk of COVID-19-related death. We conducted a systematic review with risk of bias assessment and synthesis of the early evidence on the risk of COVID-19-related death for COVID-19 patients with and without cancer. Methods and findings: We searched Medline/Emba...

Purpose To examine how socio-demographic, comorbidities and information needs influence quality of life (QoL) outcomes of survivors of breast, colorectal, or prostate cancer, non-Hodgkin lymphoma or melanoma. Methods Cross-sectional postal survey with eligible participants identified through a population-based cancer registry. QoL outcomes were as...

Background The early COVID-19 literature suggested that people with cancer may be more likely to be infected with SARS-CoV-2 or develop COVID-19 than people without cancer, due to increased health services contact and/or immunocompromise. While some studies were criticised due to small patient numbers and methodological limitations, they created or...

Biomarker testing is a laboratory test in oncology that is used in the selection of targeted cancer treatments and helping to avoid ineffective treatments. There exist several types of biomarker tests that can be used to detect the presence of particular mutations or variation in gene expression. Whole Genome Sequencing (WGS) is a biomarker test fo...

Objective To investigate medication prescription patterns among children with juvenile idiopathic arthritis (JIA), including duration, sequence and reasons for medication discontinuation. Methods This study is a single-center, retrospective analysis of prospective data from electronic medical records of JIA patients receiving systemic therapy aged...

Objectives Estimating the maximum acceptable cost (MAC) per screened individual for low-dose computed tomography (LDCT) lung cancer (LC) screening, and determining the effect of additionally screening for chronic obstructive pulmonary disease (COPD), cardiovascular disease (CVD), or both on the MAC. Methods A model-based early health technology as...

Objective Pharmacological treatment is a cornerstone of care for children with juvenile idiopathic arthritis (JIA). The objective of this study is to evaluate prescription patterns of conventional and biologic disease modifying anti-rheumatic drugs (c-DMARDs and b-DMARDs) for patients with JIA. Methods We conducted a retrospective cohort study of...

Background This study shows how dynamic simulation modeling can be applied in the context of the nationwide implementation of Whole Genome Sequencing (WGS) for non-small cell lung cancer (NSCLC) to inform organizational decisions regarding the use of complex and disruptive health technologies and how these decisions affect their potential value. M...

Background Prostate cancer is the most common internal malignancy in Australian men, and although most patients have good survival outcomes, treatment toxicities can impair function, leading to diminished quality of life for prostate cancer survivors. Socioeconomic disadvantage and geographical remoteness have been shown to be related to worse onco...

Background: Few studies have examined pediatric rheumatologists' approaches to treatment decision making for biologic therapy for patients with juvenile idiopathic arthritis (JIA). This study presents the qualitative research undertaken to support the development of a Best-Worst Scaling (BWS) survey for tapering in JIA. The study objectives were t...

Objectives: Molecular biomarker tests can inform the clinical management of genomic heterogenous hematological malignancies, yet their availability in routine care largely depends on the supporting health economic evidence. This study aims to systematically review the economic evidence for recent molecular biomarker tests in hematological malignan...

Over half of patients with lung cancer are diagnosed at a stage when curative treatment is not possible, suggesting an earlier diagnosis could improve outcomes. This comprehensive overview summarises the evidence on 1) times to diagnosis and treatment, 2) their impact on patient outcomes, 3) risk factors and 4) interventions to reduce time interval...

Background The emergence of genetic and genomic sequencing approaches for pediatric patients has raised questions about the genomic health literacy levels, attitudes toward receiving genomic information, and use of this information to inform treatment decisions by pediatric patients and their parents. However, the methods to educate pediatric patie...

Australia spends more than $20 billion annually on medicines, delivering significant health benefits for the population. However, inappropriate prescribing and medicine use also result in harm to individuals and populations, and waste of precious health resources. Medication data linked with other routine collections enable evidence generation in p...

BACKGROUND Horizon scanning (HS) aims to systematically identify novel therapies and technologies of interest to inform policy and decision-makers. Given the rapid rate of therapeutic advances, there has been increasing recognition of the need for HS globally. Prior Australian HS initiatives have not been sustained and currently there is no nationa...

BACKGROUND Horizon scanning (HS) aims to systematically identify novel therapies and technologies of interest to inform policy and decision-makers. Traditionally HS is performed by a dedicated team of experts. Despite increasing international focus and resourcing, Australian HS initiatives have not been sustained and currently there is no national...

Genomic markers define molecular subtypes and measurable residual disease (MRD) targets in B-cell acute lymphoblastic leukemia/lymphoma (B-ALL) and are essential determinants of treatment. Current diagnostic approaches typically involve serial multi-step testing utilizing conventional cytogenetics (CC)/FISH and molecular genetic (RT-qPCR, MLPA, clo...

Purpose Risk-stratified screening has potential to improve the cost effectiveness of national breast cancer screening programs. This study aimed to inform a socially acceptable and equitable implementation framework by determining what influences a woman’s decision to accept a personalized breast cancer risk assessment and what the relative impact...

Background: In some countries, such as the Netherlands and Norway, point-of-care testing (POCT) is more widely implemented in general practice compared to countries such as England and Australia. To comprehend what is necessary to realize the benefits of POCT, regarding its integration in primary care, it would be beneficial to have an overview of...

Background Linked, population-level data is valuable for mapping patterns of care and evaluating health service utilisation, particularly in difficult-to-reach populations. Upper gastrointestinal (UGI) cancers have a dismal prognosis, creating difficulties engaging patients in research. The utility of a linked dataset in this population is of high...

Objectives This study aimed to provide detailed guidance on modeling approaches for implementing competing events in discrete event simulations based on censored individual patient data (IPD). Methods The event-specific distributions (ESDs) approach sampled times from event-specific time-to-event distributions and simulated the first event to occu...

Introduction: Juvenile idiopathic arthritis (JIA) is a chronic rheumatic disease, whose multifaceted care path can lead to significant expenditure for the healthcare system. We aim to assess the real-world healthcare resource use (HCRU) and associated cost for children with JIA in a single center in Canada. Methods: A single-center consecutive c...

Background: The COVID-19 pandemic has had a vast impact on cancer service delivery around the world. Previously reported results from our international survey of oncology clinicians, conducted through March-April 2020, found that clinicians reported altering management in both the curative and palliative settings and not in proportion to the COVID...

Objectives To estimate the lifetime health and economic outcomes of selecting active surveillance (AS), radical prostatectomy (RP), or radiation therapy (RT) as initial management for low- or favorable-risk localized prostate cancer. Methods A discrete-event simulation model was developed using evidence from published randomized trials. Health out...

Background and Objective Substantial adjuvant chemotherapy (AC) overtreatment for stage II colorectal cancer results in a health and financial burden. Circulating tumour DNA (ctDNA) can improve patient selection for AC by detecting micro-metastatic disease. We estimated the health economic potential of ctDNA-guided AC for stage II colorectal cancer...

Background Current international consensus is that ‘early’ referral to palliative care services improves cancer patient and family carer outcomes. In practice, however, these referrals are not routine. An approach which directly addresses identified barriers to early integration of palliative care is required. This protocol details a trial of a sta...

e18531 Background: Prostate cancer patients generally have good survival outcomes, but some survivors have poor functional outcomes with persisting symptoms and poor quality of life (QoL). Socio-economic status (SES), access to specialist care and place of residence (including rural, remote areas) are associated with poor survival. These variables...

e18572 Background: Lung cancer is characterized by the aggressive nature and lack of early noticeable symptoms, highlighting the importance of timely care for lung cancer. This systematic review of systematic and scoping reviews provides the summary of review-level results related to the length of time intervals and the impact of these intervals on...

e18575 Background: Lung cancer is the leading cause of cancer death worldwide, and over half of lung cancer cases are diagnosed with advanced stage, indicating an urgent need to improve the timeliness of lung cancer services. In this systematic review of systematic and scoping reviews, we summarize the current evidence of risk factors and intervent...

e18851 Background: Studies evaluating the clinical benefit from Comprehensive Genomic Sequencing (CGS) in cancer patients (pts) have been limited by heterogeneous populations in cancer types and treatment history, outcomes analysis focused on pts who received treatment, and single arm designs. This study evaluated the clinical utility of CGS in a u...

Context Several liquid- and tissue-based biomarker tests (LTBTs) are available to inform the need for prostate biopsies and treatment of localised prostate cancer (PCa) through risk stratification, but translation into routine practice requires evidence of their clinical utility and economic impact. Objective To review and summarise the health eco...