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OBJECTIVES: The number of colorectal cancer (CRC) survivors is increasing and current models of survivorship care are unsustainable. There is a drive to implement alternative models of care including shared care between general practitioners (GPs) and hospital-based providers. The primary objective of this study was to explore perspectives on facilitators and barriers to shared care. The secondary objective was to explore experiences of telehealth-delivered care. METHOD: Qualitative data were collected via semi-structured interviews with participants in the Shared Care for Colorectal Cancer Survivors (SCORE) randomised controlled trial. Interviews explored patient experiences of usual and shared survivorship care during the SCORE trial. In response to the COVID pandemic, participant experiences of telehealth appointments were also explored. Interviews were recorded and transcribed for thematic analysis. RESULTS: Twenty survivors of CRC were interviewed with an even number in the shared and usual care arms; 14 (70%) were male. Facilitators to shared care included: good relationships with GPs; convenience of GPs; good communication between providers; desire to reduce public health system pressures. Barriers included: poor communication between clinicians; inaccessibility of GPs; beliefs about GP capacity; and a preference for follow-up care with the hospital after positive treatment experiences. Participants also commonly expressed a preference for telehealth-based follow-up when there was no need for a clinical examination. CONCLUSIONS: This is one of few studies that have explored patient experiences with shared and telehealth-based survivorship care. Findings can guide the implementation of these models, particularly around care coordination, communication, preparation, and personalised pathways of care.

BACKGROUND: Striking the right balance between early cancer diagnosis and the risk of excessive testing for low-risk symptoms is of paramount importance. Patient-centred care must also consider patient preferences for testing. AIM: To investigate the diagnostic testing preferences of the Australian public for symptoms associated with oesophagogastric (OG), bowel, or lung cancer. DESIGN AND SETTING: One of three discrete-choice experiments (DCEs) related to either OG, bowel, or lung cancer were administered to a nationally representative sample of Australians aged ≥40 years. METHOD: Each DCE comprised three scenarios with symptom positive predictive values (PPVs) for undiagnosed cancer ranging from 1% to 3%. The numerical risk was concealed from participants. DCE attributes encompassed the testing strategy, GP familiarity, test and result waiting times, travel duration, and test cost. Preferences were estimated using conditional and mixed logit models. RESULTS: A total of 3013 individuals participated in one of three DCEs: OG (n = 1004), bowel (n = 1006), and lung (n = 1003). Preferences were chiefly driven by waiting time and test cost, followed by the test type. There was a preference for more invasive tests. When confronted with symptoms carrying an extremely low risk (symptom PPV of ≤1%), participants were more inclined to abstain from testing. CONCLUSION: Access-related factors, particularly waiting times and testing costs, emerged as the most pivotal elements influencing preferences, underscoring the substantial impact of these systemic factors on patient choices regarding investigations.

OBJECTIVE: To summarize the key methodological challenges identified by health technology assessment (HTA) agencies assessing gene therapy (GT) and consideration of broad elements of value. METHOD: Economic evaluations (EEs) of voretigene neparvovec (VN) in RPE65-mediated inherited retinal disease (IRD) published in English were selected. HTA evaluations from Australia, Canada, Ireland, Scotland, England, and the United States were reviewed. An existing methodological framework was used to identify the challenges and considerations. RESULTS: Eight unique EEs were identified of which six were evaluated by HTA agencies. Incremental cost-effectiveness ratios ranged from $68,951 to $643,813 per quality-adjusted life-years (QALY) gained (healthcare perspective) and dominant to $480,130 per QALY gained (societal perspective). The key challenges were the lack of validated surrogate outcome, utility values and indirect costs from IRD patients, and limited evidence of the long-term treatment effect. Two HTA agencies reviewed a range of novel broader elements of value and whether they were associated with VN while other agencies discussed some elements of broader value. Caregiver disutility was included in some, but not all, evaluations. CONCLUSION: The methodological challenges were consistent with innovative interventions for rare diseases and managed using standard methods. Broader value was important to decision-makers but inconsistently applied across agencies. Possible reasons are limitations in the evidence available of the broader benefits that VN offers and how to incorporate these within an EE. A need exists for greater guidance and consistency across jurisdictions regarding the consideration of broader value that considers latest best practice.

84 Background: Colorectal cancer (CRC) survivorship is rising. Few studies have considered shared follow up (FU) care (SC) between oncologists and primary care providers (PCPs). SCORE is the first large RCT of SC for CRC survivors. Aim: to compare SC with usual care (UC) for CRC survivors. Primary objective: assess the effect of SC vs UC on the EORTC QLQ-C30 Global Health Status/Quality of Life (GHQ-QoL) scale to 12 months (mo). Secondary objectives: assess the effect of SC vs UC on QoL; pt perceptions of care; costs; clinical care processes (CEA tests, recurrences); and feasibility (recruitment, completion rate). Methods: Pts had stage I-III CRC, completed all treatments within 3 mo, spoke English, had a PCP, and no prior cancer. After pt consent, PCP could opt out. Pts were randomised 1 to 1 to SC or UC. SC replaced 2 routine oncologist visits with PCP visits and included a survivorship care plan, concerns checklist and management guidelines for the PCP. PCPs were asked to request CEA tests at 3 and 9 mo visits. Pts completed measures at baseline (BL) and at 6 and 12 mo FU. An estimate of the difference (diff) between groups on GHQ-QoL to 12 mo was obtained from a mixed model for repeated measures (MMRM). The test of non-inferiority was performed by evaluating the lower limit of the two-sided 95% confidence interval (CI) for the estimated diff (SC–UC) against a pre-specified non-inferiority margin (NIM) of -10 points. All randomised pts with available data were included in the primary analysis. Per-protocol population (PPP) comprised all randomised pts with ≥ 1 post-BL questionnaire (6 +/or 12 mo) and, for SC, ≥ 1 of the PCP visits. Results: 150 pts were randomised to SC (N=74) or UC (N=76); 11 PCPs declined. Median age 63 years, 39% women, 24% had radiotherapy. Primary: colon (59%), rectum (32%), overlapping (9%). 138/150 (92%) had BL and ≥1 post-BL GHQ-QoL score. 65/74 (88%) of SC pts attended 3- and/or 9-mo PCP visit. The mean (SD) GH

BACKGROUND: SCORE is the first randomised controlled trial (RCT) to examine shared oncologist and general practitioner (GP) follow-up for survivors of colorectal cancer (CRC). SCORE aimed to show that shared care (SC) was non-inferior to usual care (UC) on the EORTC QLQ-C30 Global Health Status/Quality of Life (GHQ-QoL) scale to 12 months. METHODS: The study recruited patients from five public hospitals in Melbourne, Australia between February 2017 and May 2021. Patients post curative intent treatment for stage I-III CRC underwent 1:1 randomisation to SC and UC. SC replaced two oncologist visits with GP visits and included a survivorship care plan and primary care management guidelines. Assessments were at baseline, 6 and 12 months. Difference between groups on GHQ-QoL to 12 months was estimated from a mixed model for repeated measures (MMRM), with a non-inferiority margin (NIM) of -10 points. Secondary endpoints included quality of life (QoL); patient perceptions of care; costs and clinical care processes (CEA tests, recurrences). Registration ACTRN12617000004369p. FINDINGS: 150 consenting patients were randomised to SC (N = 74) or UC (N = 76); 11 GPs declined. The mean (SD) GHQ-QoL scores at 12 months were 72 (20.2) for SC versus 73 (17.2) for UC. The MMRM mean estimate of GHQ-QoL across the 6 month and 12 month follow-up was 69 for SC and 73 for UC, mean difference -4.0 (95% CI: -9.0 to 0.9). The lower limit of the 95% CI did not cross the NIM. There was no clear evidence of differences on other QoL, unmet needs or satisfaction scales. At 12 months, the majority preferred SC (40/63; 63%) in the SC group, with equal preference for SC (22/62; 35%) and specialist care (22/62; 35%) in UC group. CEA completion was higher in SC. Recurrences similar between arms. Patients in SC on average incurred USD314 less in health costs versus UC patients. INTERPRETATION: SC seems to be an appropriate and cost-effective model of follow-up for CRC survivors. FUNDING: Victorian Cance

BACKGROUND: Multiparametric magnetic resonance imaging (mpMRI) has an established role for the diagnosis of clinically significant prostate cancer (sPCa). The PRIMARY trial demonstrated that [68Ga]Ga-PSMA-11 positron emission tomography/computed tomography (PET/CT) was associated with a significant improvement in sensitivity and negative predictive value for sPCa detection. OBJECTIVE: To demonstrate that addition of prostate-specific membrane antigen (PSMA) radioligand PET/CT will enable some men to avoid transperineal prostate biopsy without missing sPCa, and will facilitate biopsy targeting of PSMA-avid sites. DESIGN, SETTING, AND PARTICIPANTS: This multicentre, two-arm, phase 3, randomised controlled trial will recruit 660 participants scheduled to undergo biopsy. Eligible participants will have clinical suspicion of sPCa with a Prostate Imaging-Reporting and Data System (PI-RADS) score of 2 and red flags, or a PI-RADS score of 3 on mpMRI (PI-RADS v2). Participants will be randomised at a 1:1 ratio in permuted blocks stratified by centre. The trial is registered on ClinicalTrials.gov as NCT05154162. INTERVENTION: In the experimental arm, participants will undergo pelvic PSMA PET/CT. Local and central reviewers will interpret scans independently using the PRIMARY score. Participants with a positive result will undergo targeted transperineal prostate biopsies, whereas those with a negative result will undergo prostate-specific antigen monitoring alone. In the control arm, all participants undergo template transperineal prostate biopsies. Participants will be followed for subsequent clinical care for up to 2 yr after randomisation. OUTCOME MEASUREMENTS AND STATISTICAL ANALYSIS: sPCa is defined as Gleason score 3 + 4 (≥10%) = 7 disease (grade group 2) or higher on transperineal prostate biopsy. Avoidance of transperineal prostate biopsy will be measured at 6 mo from randomisation. The primary endpoints will be analysed on an intention-to-treat basis. CONCLUSIONS: Pat

IMPORTANCE: New dosing options for immune checkpoint inhibitors have recently been approved by the US Food and Drug Administration (FDA), including fixed dosing with extended intervals. Although the dose intensity appears the same, there is expected to be some waste with extended-interval dosing, as some drug remains in the bloodstream once a decision to stop treatment is made. The economic impact of extended-interval fixed dosing is unknown compared with standard-interval fixed dosing. OBJECTIVE: To analyze the potential health care costs of using extended-interval fixed dosing instead of standard-interval fixed dosing. DESIGN, SETTING, AND PARTICIPANTS: This economic evaluation used a pharmacoeconomic model to simulate 2 cohorts of patients with platinum-resistant metastatic urothelial cancer receiving pembrolizumab as second-line therapy at different dosing intervals using 2020 pricing data. Data were analyzed from 2020 to 2022. EXPOSURES: The simulated patients received FDA-approved regimens of either 200 mg every 3 weeks or 400 mg every 6 weeks. MAIN OUTCOMES AND MEASURES: The progression-free survival curve from the KEYNOTE-045 trial was used to estimate treatment duration. Drug, imaging, and administration costs were included in analyses. Sensitivity analyses were performed to assess how different imaging frequencies would affect the model results. The potential overall costs of using the 2 different dosing strategies were assessed. The base case was set in the US, while sensitivity analyses were set in several other countries. RESULTS: In the base case analysis, dosing every 6 weeks instead of every 3 weeks resulted in an estimated 8.9% increase in pembrolizumab costs for the health care payer. Accounting for a decrease in infusion costs would result in an estimated net additional cost of $7483 per patient in the US (7.9% cost increase). In the US, this would amount to an increase of approximately $28 million annually for health care payers. Similar percenta

PURPOSE: To assess the literature on men's preferences and perceptions regarding prostate cancer radiation therapy. METHODS: A scoping review was undertaken as per JBI guidelines. Searches were conducted in PubMed, CINAHL, Scopus and Science Direct with search terms including "prostate cancer," "radiotherapy," "radiation therapy," "radiation oncology," "patient preferences," "patient perceptions" and "patient experience." The resultant studies were mapped and grouped according to the emergent themes and pathway stages. RESULTS: A total of 779 titles and abstracts were screened by two independent reviewers. Fifty-two full-text studies were reviewed, with 27 eligible for inclusion. There were 4 pre-treatment, 13 during treatment and 10 post-treatment studies covering broad themes of information needs (n = 3), preferences and decisions (n = 6), general experiences (n = 8), side effects (n = 6), and support (n = 4). There were a mix of methodologies, including 11 qualitative, 14 quantitative (including four preference studies), one mixed methods and one narrative review. CONCLUSION: There were only four preference studies, with the remaining 23 reporting on perceptions. Overall, there is a paucity of literature regarding patient preferences and perceptions of prostate cancer radiation therapy, particularly when considering how many clinical and technical studies are published in the area. This highlights opportunities for future research.

INTRODUCTION: Glioblastoma is the most common aggressive primary central nervous system cancer in adults characterised by uniformly poor survival. Despite maximal safe resection and postoperative radiotherapy with concurrent and adjuvant temozolomide-based chemotherapy, tumours inevitably recur. Imaging with O-(2-[18F]-fluoroethyl)-L-tyrosine (FET) positron emission tomography (PET) has the potential to impact adjuvant radiotherapy (RT) planning, distinguish between treatment-induced pseudoprogression versus tumour progression as well as prognostication. METHODS AND ANALYSIS: The FET-PET in Glioblastoma (FIG) study is a prospective, multicentre, non-randomised, phase II study across 10 Australian sites and will enrol up to 210 adults aged ≥18 years with newly diagnosed glioblastoma. FET-PET will be performed at up to three time points: (1) following initial surgery and prior to commencement of chemoradiation (FET-PET1); (2) 4 weeks following concurrent chemoradiation (FET-PET2); and (3) within 14 days of suspected clinical and/or radiological progression on MRI (performed at the time of clinical suspicion of tumour recurrence) (FET-PET3). The co-primary outcomes are: (1) to investigate how FET-PET versus standard MRI impacts RT volume delineation and (2) to determine the accuracy and management impact of FET-PET in distinguishing pseudoprogression from true tumour progression. The secondary outcomes are: (1) to investigate the relationships between FET-PET parameters (including dynamic uptake, tumour to background ratio, metabolic tumour volume) and progression-free survival and overall survival; (2) to assess the change in blood and tissue biomarkers determined by serum assay when comparing FET-PET data acquired prior to chemoradiation with other prognostic markers, looking at the relationships of FET-PET versus MRI-determined site/s of progressive disease post chemotherapy treatment with MRI and FET-PET imaging; and (3) to estimate the health economic impact of in

PURPOSE: There is significant interest in the use of polygenic risk score (PRS) tests to improve cancer risk assessment and stratified prevention. Our current understanding of preferences regarding different aspects of this novel testing approach is limited. This study examined which attributes of a PRS test most influence the likelihood of testing. METHODS: A discrete choice experiment was developed to elicit preferences for different aspects of a PRS test by surveying an online sample of the Australian population. Preferences were assessed using mixed logistic regression, latent class analysis, and marginal willingness to pay. RESULTS: The 1002 surveyed respondents were more likely to choose a PRS test that was more accurate, tested for multiple cancer types, and enabled cancer risk reduction through lifestyle modification, screening, or medication. There was also a preference for testing through a primary care physician rather than online or through a genetic specialist. A test that did not impact life insurance eligibility or premiums was preferred over the one that did. CONCLUSION: This study found that the Australian population prefer a PRS test that is highly accurate, tests for multiple cancers, has noninvasive risk reduction measures, and is performed through primary care.

CONTEXT: Carriers of succinate dehydrogenase type B (SDHB) pathogenic variants (PVs) are at risk of pheochromocytoma and paraganglioma (PPGL) from a young age. It is widely recommended carriers enter a surveillance program to detect tumors, but there are limited studies addressing outcomes of surveillance protocols for SDHB PV carriers. OBJECTIVE: The purpose of this study was to describe surveillance-detected (s-d) tumors in SDHB PV carriers enrolled in a surveillance program and to compare their outcomes to probands. METHODS: This was a multicenter study of SDHB PV carriers with at least 1 surveillance episode (clinical, biochemical, imaging) in Australian genetics clinics. Data were collected by both retrospective and ongoing prospective follow-up. Median duration of follow-up was 6.0 years. RESULTS: 181 SDHB PV carriers (33 probands and 148 nonprobands) were assessed. Tumors were detected in 20% of nonprobands undergoing surveillance (age range 9-76 years). Estimated 10-year metastasis-free survival was 66% for probands and 84% for nonprobands with s-d tumors (P = .027). S-d tumors were smaller than those in probands (median 27 mm vs 45 mm respectively, P = .001). Tumor size ≥40 mm was associated with progression to metastatic disease (OR 16.9, 95% CI 2.3-187.9, P = .001). Patients with s-d tumors had lower mortality compared to probands: 10-year overall survival was 79% for probands and 100% for nonprobands (P = .029). CONCLUSION: SDHB carriers with s-d tumors had smaller tumors, reduced risk of metastatic disease, and lower mortality than probands. Our results suggest that SDHB PV carriers should undertake surveillance to improve clinical outcomes.

OBJECTIVES: To synthesize existing evidence on the effects of multimodal prehabilitation interventions in men affected by prostate cancer on physical, clinical, and patient-reported outcome measures. DATA SOURCES: A systematic review was conducted according to the PRISMA 2020 Statement Guidelines. Electronic databases (ie, Medline, Embase, CINAHL and Cochrane CENTRAL, and clinicaltrials.gov) were searched using key search terms. Articles were assessed according to prespecified eligibility criteria. Data extraction and quality appraisal was conducted. The findings were integrated in a narrative synthesis. CONCLUSION: Of the 5863 publications screened, 118 articles were assessed in full text and 17 studies met the prescreening eligibility criteria. There were a range of study designs that included randomized controlled clinical trials (n = 11), quasi experimental (n = 4), cohort (n = 1), and case series (n = 1), covering a total of 1739 participants. The prehabilitation interventions included physical activity, peer support, pelvic floor muscle training, diet, nurse-led prehabilitation, psychological, and prehabilitation administration of phosphodiesterase-5 inhibitors. IMPLICATIONS FOR NURSING PRACTICE: Significant heterogeneity existed in the prehabilitation intervention programs for men affected by prostate cancer in terms of the composition, duration, method of administration, and the outcomes measured to quantify their impact. This systematic review has identified that multimodal prehabilitation interventions are an emerging area for practice and research among men affected by prostate cancer. Importantly, there has been a lack of focus on the inclusion of partners as critical companions during this distressing phase of the cancer care continuum. For the moment, all members of the multidisciplinary team caring for people affected by prostate cancer are encouraged to use the findings in this review to inform holistic models of care.

AIM: To understand the factors that most influence decision-making in the treatment of infantile spinal muscular atrophy (SMA). METHOD: A discrete choice experiment was conducted among parents of people with SMA (parents), healthcare professionals (HCPs), and members of the Australian general population (GenPop). Respondents were asked to accept/reject treatment for an infant newly diagnosed with SMA in eight hypothetical scenarios, characterized by different combinations of the attributes of the treatment offered. The results were analyzed using probability analysis. RESULTS: Completed responses were provided from 1113 individuals (1024 GenPop, 21 parents, 68 HCPs). Respondents were more likely to accept treatments that improved functioning and mobility. Treatments with higher costs, invasive delivery, and risks of adverse events were accepted less often. Cost most affected treatment choices by HCPs and GenPop, while change in mobility and mode of administration were most influential for parents. INTERPRETATION: These results highlight the importance of understanding value for money and clinical impact in affecting treatment choice, which are crucial for effective planning of healthcare and the successful implementation of treatment programmes for SMA. What this paper adds Spinal muscular atrophy (SMA) treatments with a higher chance of improving functioning and mobility are preferred by the general population, parents, and healthcare professionals. Treatments with higher costs, invasive delivery, and risk of adverse events are less preferred. Willingness to pay for SMA treatments increases with impact on functioning.

BACKGROUND: The complexity of prostate cancer care can impact on patient understanding and participation in shared decision-making. This study used a survey-based approach to investigate patients' recall of their prostate cancer treatment, and more broadly, to understand the perceptions of patients and the general population of prostate cancer treatment. METHOD: The survey was completed by 236 patients with prostate cancer (PCa cohort) and 240 participants from the general population of Australia (GenPop cohort). Free-text comments from both cohorts were analysed using content analysis. The PCa cohort reported which treatments and image-guidance related procedures they had received. These patient-reports were compared to medical records and analysed using proportion agreement, kappa statistics and regression analysis. RESULTS: 135 (57%) PCa and 99 (41%) GenPop respondents provided at least one comment. Five major themes were identified by both cohorts: sharing experiences of treatment; preferences insights and reflections; mindsets; general commentary on the survey; and factors missing from the survey. There was overall good treatment recall amongst the PCa cohort, with proportions of correct recall ranging from 97.3% for chemotherapy to 66.8% for hormone therapy. There was a tendency for younger patients (<70 years old) to recall their hormone treatment more correctly. CONCLUSION: Participant comments suggest the complexity of prostate cancer diagnosis and treatment, and the varying perceptions and experiences of participants with prostate cancer. Patients' recall overall was good for both treatment and image-guidance related procedures/approaches, however the poorer recall of hormone therapy requires further investigation.

AIMS: The aim of TROG 14.04 was to assess the feasibility of deep inspiration breath hold (DIBH) and its impact on radiation dose to the heart in patients with left-sided breast cancer undergoing radiotherapy. Secondary end points pertained to patient anxiety and cost of delivering a DIBH programme. MATERIALS AND METHODS: The study comprised two groups - left-sided breast cancer patients engaging DIBH and right-sided breast cancer patients using free breathing through radiotherapy. The primary end point was the feasibility of DIBH, defined as left-sided breast cancer patients' ability to breath hold for 15 s, decrease in heart dose in DIBH compared with the free breathing treatment plan and reproducibility of radiotherapy delivery using mid-lung distance (MLD) assessed on electronic portal imaging as the surrogate. The time required for treatment delivery, patient-reported outcomes and resource requirement were compared between the groups. RESULTS: Between February and November 2018, 32 left-sided and 30 right-sided breast cancer patients from six radiotherapy centres were enrolled. Two left-sided breast cancer patients did not undergo DIBH (one treated in free breathing as per investigator choice, one withdrawn). The mean heart dose was reduced from 2.8 Gy (free breathing) to 1.5 Gy (DIBH). Set-up reproducibility in the first week of treatment assessed by MLD was 1.88 ± 1.04 mm (average ± 1 standard deviation) for DIBH and 1.59 ± 0.93 mm for free breathing patients. Using a reproducibility cut-off for MLD of 2 mm (1 standard deviation) as per study protocol, DIBH was feasible for 67% of DIBH patients. Radiotherapy delivery using DIBH took about 2 min longer than for free breathing. Anxiety was not significantly different in DIBH patients and decreased over the course of treatment in both groups. CONCLUSION: Although DIBH was shown to require about 2 min longer per treatment slot, it has the potential to reduce heart dose in left-sided breast cancer patients by near

INTRODUCTION: There are several options for real-time prostate monitoring during radiation therapy including fiducial markers (FMs) and transperineal ultrasound (TPUS). However, the patient experience for these procedures is very different. This study aimed to determine patient preferences around various aspects of prostate image-guidance, focusing on FMs and TPUS. METHODS: A discrete choice experiment (DCE) was conducted, describing the image-guidance approach by: pain, cost, accuracy, side effects, additional appointments, and additional time. Participants were males with prostate cancer (PCa) and from the general Australian population. A DCE survey required participants to make hypothetical choices in each of 8 choice sets. Multinomial logit modelling and Latent Class Analysis (LCA) were used to analyse the responses. Marginal willingness to pay (mWTP) was calculated. RESULTS: 476 respondents completed the survey (236 PCa patients and 240 general population). The most important attributes for both cohorts were pain, cost and accuracy (p < 0.01). PCa patients were willing to pay more to avoid the worst pain than the general population, and willing to pay more for increased accuracy. LCA revealed 3 groups: 2 were focused more on the process-related attributes of pain and cost, and the third was focused on the clinical efficacy attributes of accuracy and side effects. CONCLUSION: Both cohorts preferred less cost and pain and improved accuracy, with men with PCa valuing accuracy more than the general population. In addition to the clinical and technical evidence, radiation oncology centres should consider the preferences of patients when considering choice of image-guidance techniques.

OBJECTIVE: This research assesses the impact of an outcome-based payment arrangement (OBA) linking complete remission (CR) to survival as a means of maintaining cost-effectiveness for a chimeric antigen receptor T cell (CAR-T) therapy in young patients with acute lymphoblastic leukemia (ALL). METHODS: A partitioned survival model (PSM) was used to model the cost-effectiveness of tisagenlecleucel versus blinatumomab in ALL from the Australian healthcare system perspective. A decision tree modeled different OBAs by funneling patients into a series of PSMs based on response. Outcomes were informed by individual patient data, while costs followed Australian treatment practices. Costs and quality-adjusted life years (QALYs) were combined to calculate a single incremental cost-effectiveness ratio (ICER), reported in US dollars (2022) at a discount rate of 5% on costs and outcomes. RESULTS: For the base case, incremental costs and benefit were $379,595 and 4.27 QALYs, giving an ICER of $88,979. The ICER was most sensitive to discount rate ($57,660-$75,081), "cure point" ($62,718-$116,206) and extrapolation method ($76,018-$94,049). OBAs had a modest effect on the ICER when response rates varied. A responder-only payment was the most effective arrangement for maintaining the ICER ($88,249-$89,434), although this option was associated with the greatest financial uncertainty. A split payment arrangement (payment on infusion followed by payment on response) reduced variability in the ICER ($82,650-$99,154) compared with a single, upfront payment ($77,599-$107,273). CONCLUSION: OBAs had a modest impact on reducing cost-effectiveness uncertainty. The value of OBAs should be weighed against the additional resources needed to administer such arrangements, and importantly overall cost to government.

Background: The timing and necessity of repeated blood cultures (BCs) in children with cancer and febrile neutropenia (FN) are unknown. We evaluated the diagnostic yield of BCs collected pre- and post-empiric FN antibiotics. Method(s): Data collected prospectively from the Australian Predicting Infectious ComplicatioNs in Children with Cancer (PICNICC) study were used. Diagnostic yield was calculated as the number of FN episodes with a true bloodstream infection (BSI) detected divided by the number of FN episodes that had a BC taken. Result(s): A BSI was identified in 13% of 858 FN episodes. The diagnostic yield of pre-antibiotic BCs was higher than of post-antibiotic cultures (12.3% vs 4.4%, P <. 001). Two-thirds of the post-antibiotic BSIs were associated with a new episode of fever or clinical instability, and only 2 new BSIs were identified after 48 hours of empiric antibiotics and persistent fever. A contaminated BC was identified more frequently in post-antibiotic cultures. Conclusion(s): In the absence of new fever or clinical instability, BCs beyond 48 hours of persistent fever have limited yield. Opportunity exists to optimize BC collection in this population and reduce the burden of unnecessary tests on patients, healthcare workers, and hospitals.Copyright © 2020 The Author(s) 2020. Published by Oxford University Press on behalf of The Journal of the Pediatric Infectious Diseases Society. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.

BACKGROUND: The emerging role of genomically guided precision medicine in pediatric cancer care presents significant clinical, practical, and ethical challenges. We investigated the factors that influence decision-making in genomic medicine from the perspective of different stakeholders in the context of difficult-to-treat childhood cancer. METHODS: Health care providers (HCPs), parents of childhood cancer survivors, and general community members completed an online discrete choice experiment survey. Respondents considered whether to recommend (HCPs) or choose (parents/community) a genomically guided approach to pediatric cancer treatment. Respondents completed eight choice questions varying by survival benefit, prognosis, likelihood of finding a target, quality of life (QoL), HCP/parent preference, need for biopsy, cost, and who pays. Data were analyzed using a probability regression model, with findings expressed as relative importance, stated importance, and marginal willingness to pay (mWTP). RESULTS: One hundred twenty-six HCPs, 130 parents, and 531 community members participated. The probability of recommending/choosing genomically guided treatment increased significantly with better prognosis, survival benefit, improvements in QoL, and decision-making partner support. It decreased with increasing costs and if parents paid for treatment. HCPs were more responsive to all factors but were most influenced by survival outcomes, and parents and community members by QoL. In contrast to these forced choice preference results, HCPs stated they were most influenced by QoL and community members by survival. CONCLUSION: Our findings support the primacy of QoL in genomic decision-making, with some differences across stakeholders in the other factors influencing decision-making. These findings emphasize the need for high-quality information giving and communication to support genomic medicine choices.

OBJECTIVES: Febrile neutropenia (FN) causes treatment disruption and unplanned hospitalization in children with cancer. Serum biomarkers are infrequently used to stratify these patients into high or low risk for serious infection. This study investigated plasma abundance of cytokines in children with FN and their ability to predict bacteraemia. METHODS: Thirty-three plasma cytokines, C-reactive protein (CRP) and procalcitonin (PCT) were measured using ELISA assays in samples taken at FN presentation (n = 79) and within 8-24 h (Day 2; n = 31). Optimal thresholds for prediction of bacteraemia were identified and the predictive ability of biomarkers in addition to routinely available clinical variables was assessed. RESULTS: The median age of included FN episodes was 6.0 years and eight (10%) had a bacteraemia. On presentation, elevated PCT, IL-10 and Mip1-beta were significantly associated with bacteraemia, while CRP, IL-6 and IL-8 were not. The combination of PCT (≥0.425 ng/ml) and IL-10 (≥4.37 pg/ml) had a sensitivity of 100% (95% CI 68.8-100%) and specificity of 89% (95% CI 80.0-95.0%) for prediction of bacteraemia, correctly identifying all eight bacteraemia episodes and classifying 16 FN episodes as high-risk. There was limited additive benefit of incorporating clinical variables to this model. On Day 2, there was an 11-fold increase in PCT in episodes with a bacteraemia which was significantly higher than that observed in the non-bacteraemia episodes. CONCLUSION: Elevated PCT and IL-10 accurately identified all bacteraemia episodes in our FN cohort and may enhance the early risk stratification process in this population. Prospective validation and implementation is required to determine the impact on health service utilisation.

AIM: The Australian 'There is no place like home' project is implementing a paediatric low-risk febrile neutropenia (FN) programme across eight paediatric hospitals. We sought to identify the impact of the coronavirus disease 2019 (COVID-19) pandemic on programme implementation. METHODS: Paediatric oncology, infectious diseases and emergency medicine health-care workers and parent/carers were surveyed to explore the impact of the COVID-19 pandemic on home-based FN care. Online surveys were distributed nationally to health-care workers involved in care of children with FN and to parents or carers of children with cancer. RESULTS: Surveys were completed by 78 health-care workers and 32 parents/carers. Overall, 95% of health-care workers had confidence in the safety of home-based FN care, with 35% reporting changes at their own hospitals in response to the pandemic that made them more comfortable with this model. Compared to pre-pandemic, >50% of parent/carers were now more worried about attending the hospital with their child and >80% were interested in receiving home-based FN care. Among both groups, increased telehealth access and acceptance of home-based care, improved patient quality of life and reduced risk of nosocomial infection were identified as programme enablers, while re-direction of resources due to COVID-19 and challenges in implementing change during a crisis were potential barriers. CONCLUSION: There is strong clinician and parent/carer support for home-based management of low-risk FN across Australia. Changes made to the delivery of cancer care in response to the pandemic have generally increased acceptance for home-based treatments and opportunities exist to leverage these to refine the low-risk FN programme.

Telehealth interventions offer a practical platform to support adolescent and young adult (AYA) cancer survivors' mental health needs after treatment, yet efficacy data are lacking. We evaluated an online, group-based, videoconferencing-delivered cognitive-behavioral therapy (CBT) intervention ('Recapture Life') in a 3-arm randomized-controlled trial comparing Recapture Life with an online peer-support group, and a waitlist control, with the aim of testing its impact on quality of life, emotional distress and healthcare service use. Forty AYAs (Mage = 20.6 years) within 24-months of completing treatment participated, together with 18 support persons. No groupwise impacts were measured immediately after the six-week intervention. However, Recapture Life participants reported using more CBT skills at the six-week follow-up (OR = 5.58, 95% CI = 2.00-15.56, p = 0.001) than peer-support controls. Recapture Life participants reported higher perceived negative impact of cancer, anxiety and depression at 12-month follow-up, compared to peer-support controls. Post-hoc analyses suggested that AYAs who were further from completing cancer treatment responded better to Recapture Life than those who had completed treatment more recently. While online telehealth interventions hold promise, recruitment to this trial was challenging. As the psychological challenges of cancer survivorship are likely to evolve with time, different support models may prove more or less helpful for different sub-groups of AYA survivors at different times.

Objective The aim of this study was to evaluate the cost-effectiveness of the Australian Contraceptive ChOice pRoject (ACCORd) intervention. Methods An economic evaluation compared the costs and outcomes of the ACCORd intervention with usual care (UC). Data from the ACCORd trial were used to estimate costs and efficacy in terms of contraceptive uptake and quality of life. Rates of contraceptive failure and pregnancy were sourced from the literature. Using a Markov model, within-trial results were extrapolated over 10 years and subjected to univariate sensitivity analyses. Model outputs were expressed as the cost per quality-adjusted life years (QALY) gained and cost per unintended pregnancy resulting in birth (UPB) avoided. Results Over 10 years, compared with UC, initiating contraception through the ACCORd intervention resulted in 0.02 fewer UPB and higher total costs (A$2505 vs A$1179) per woman. The incremental cost-effectiveness of the ACCORd intervention versus UC was A$1172 per QALY gained and A$7385 per UPB averted. If the start-up cost of the ACCORd intervention was removed, the incremental cost-effectiveness ratio was A$81 per QALY gained and A$511 per UPB averted. The results were most sensitive to the probability of contraceptive failure, the probability of pregnancy-related healthcare service utilisation or the inclusion of the costs of implementing the ACCORd intervention. Conclusions From a health system perspective, if implemented appropriately in terms of uptake and reach, and assuming an implicit willingness to pay threshold of A$50 000 the ACCORd intervention is cost-effective. What is known about the topic? The uptake of long-active reversible contraceptives (LARC) in Australia is low. The ACCORd trial assessed the efficacy of providing structured training to general practitioners (GPs) on LARC counselling, together with access to rapid referral to insertion clinics. What does this paper add? This study is the first to assess the cost-effectivenes

BACKGROUND:The timing and necessity of repeated blood cultures (BCs) in children with cancer and febrile neutropenia (FN) are unknown. We evaluated the diagnostic yield of BCs collected pre- and post-empiric FN antibiotics. METHODS:Data collected prospectively from the Australian Predicting Infectious ComplicatioNs in Children with Cancer (PICNICC) study were used. Diagnostic yield was calculated as the number of FN episodes with a true bloodstream infection (BSI) detected divided by the number of FN episodes that had a BC taken. RESULTS:A BSI was identified in 13% of 858 FN episodes. The diagnostic yield of pre-antibiotic BCs was higher than of post-antibiotic cultures (12.3% vs 4.4%, P < .001). Two-thirds of the post-antibiotic BSIs were associated with a new episode of fever or clinical instability, and only 2 new BSIs were identified after 48 hours of empiric antibiotics and persistent fever. A contaminated BC was identified more frequently in post-antibiotic cultures. CONCLUSIONS:In the absence of new fever or clinical instability, BCs beyond 48 hours of persistent fever have limited yield. Opportunity exists to optimize BC collection in this population and reduce the burden of unnecessary tests on patients, healthcare workers, and hospitals.

INTRODUCTION: Home-based treatment of febrile neutropenia (FN) in children with cancer with oral or intravenous antibiotics is safe and effective. There are limited data on the economic impact of this model of care. We evaluated the cost-effectiveness of implementing an FN programme, incorporating home-based intravenous antibiotics for carefully selected patients, in a tertiary paediatric hospital. METHODS: A decision analytic model was constructed to compare costs and outcomes of the home-based FN programme, with usual in-hospital treatment with intravenous antibiotics. The programme included a clinical decision rule to stratify patients by risk for severe infection and home-based eligibility criteria using disease, chemotherapy and patient-level factors. Health outcomes (quality of life) and probabilities of FN risk classification and home-based eligibility were based on prospectively collected data between 2017 and 2019. Patient-level costs were extracted from hospital administrative records. Cost-effectiveness was expressed as the incremental cost per quality-adjusted life year (QALY). FINDINGS: The mean health care cost of home-based FN treatment in low-risk patients was Australian dollars (A$) 7765 per patient compared to A$20,396 for in-hospital treatment (mean difference A$12,632 [95% CI: 12,496-12,767]). Overall, the home-based FN programme was the dominant strategy, being more effective (0.0011 QALY [95% CI: 0.0011-0.0012]) and less costly. Results of the model were most sensitive to proportion of children eligible for home-based care programme. CONCLUSION: Compared to in-hospital FN care, the home-based FN programme is cost-effective, with savings arising from cheaper cost of caring for children at home. These savings could increase as more patients eligible for home-based care are included in the programme.

Background The impact febrile neutropenia (FN) has on the health-related quality of life (HRQoL) of children with cancer and their families is poorly understood. We sought to characterize the course of child and parent HRQoL during and following FN episodes. Method Data on HRQoL were collected in the multisite Australian Predicting Infectious ComplicatioNs in Children with Cancer (PICNICC) study. Participants were enrolled between November 2016 to January 2018. The Child Health Utility (CHU9D) was used to assess HRQoL in children (N = 167 FN events) and the Assessment of Quality of Life (AQoL-8D) was used to assess HRQoL parents (N = 218 FN events) at three time points: 0–3 days, 7-days and 30-days following the onset of FN. Group-based trajectory modeling (GBTM) was used to characterize the course of HRQoL. Findings For children, three distinct groups were identified: persistently low HRQoL over the 30-day course of follow-up (chronic: N = 78/167; 47%), increasing HRQoL after the onset of FN to 30 days follow-up (recovering: N = 36/167; 22%), and persistently high HRQoL at all three timepoints (resilient: N = 53/167; 32%). Applying these definitions, parents were classified into two distinct groups: chronic (N = 107/218, 49%) and resilient (N = 111/218, 51%). The child being male, having solid cancer, the presence of financial stress, and relationship difficulties between the parent and child were significant predictors of chronic group membership for both parents and children. Children classified as high-risk FN were significantly more likely to belong to the recovery group. Being female, having blood cancers and the absence of financial or relationship difficulties were predictive of both parents and children being in the resilient group. Interpretation Approximately half the children and parents had chronically low HRQoL scores, which did not improve following resolution of the FN episode. The child's sex, cancer type, and presence of financial and relationship

IntroductionGrades 2 and 3 gliomas (G2/3 gliomas), when combined, are the second largest group of malignant brain tumours in adults. The outcomes for G2/3 gliomas at progression approach the dismal outcomes for glioblastoma (GBM), yet there is a paucity of trials for Australian patients with relapsed G2/3 gliomas compared with patients with GBM. LUMOS will be a pilot umbrella study for patients with relapsed G2/3 gliomas that aims to match patients to targeted therapies based on molecular screening with contemporaneous tumour tissue. Participants in whom no actionable or no druggable mutation is found, or in whom the matching drug is not available, will form a comparator arm and receive standard of care chemotherapy. The objective of the LUMOS trial is to assess the feasibility of this approach in a multicentre study across five sites in Australia, with a view to establishing a national molecular screening platform for patient treatment guided by the mutational analysis of contemporaneous tissue biopsiesMethods and analysisThis study will be a multicentre pilot study enrolling patients with recurrent grade 2/3 gliomas that have previously been treated with radiotherapy and chemotherapy at diagnosis or at first relapse. Contemporaneous tumour tissue at the time of first relapse, defined as tissue obtained within 6 months of relapse and without subsequent intervening therapy, will be obtained from patients. Molecular screening will be performed by targeted next-generation sequencing at the reference laboratory (PathWest, Perth, Australia). RNA and DNA will be extracted from representative formalin-fixed paraffin embedded tissue scrolls or microdissected from sections on glass slides tissue sections following a review of the histology by pathologists. Extracted nucleic acid will be quantified by Qubit Fluorometric Quantitation (Thermo Fisher Scientific). Library pr