Sophy Ting-Fang Shih

Background: Point-of-care hepatitis C virus (HCV) antibody and RNA testing can simplify diagnosis and improve treatment initiation compared to the standard-of-care HCV diagnostic algorithm, but its population-level health and economic effects when implemented at scale remain uncertain. The Australian Hepatitis C Point-of-Care Testing Program (National Program) is a real-world national program implementing across community and prison settings. We evaluated the epidemiological impact and cost-effectiveness of its current and future implementation. Methods: We used a dynamic deterministic model of HCV transmission and testing, with parameters informed by empirical implementation, epidemiological, and cost data from the National Program. Current implementation and future sustainment and scale-up scenarios was compared with a no-program counterfactual using standard-of-care HCV diagnostics across community and prison settings. Cost-effectiveness was assessed from the healthcare providers’ perspective over 2022-2041 to capture long-term costs and quality-adjusted life-years (QALYs) beyond 2022-2030 implementation period. Costs and QALYs were discounted at 5% per annum. Findings: Under sustained implementation to 2030, HCV RNA prevalence was projected to decline from 9·5% in 2021 to 2·4% in 2030, versus 3·3% without the Program. Compared with no program, sustained implementation was projected to avert 4,350 infections (13·7%) and facilitate 1,242 treatments (2·3%) over 2022-2030. Over 2022-2041, A$20·5 million in incremental diagnostic costs were offset by reductions in HCV treatment (A$19·9 million) and disease management (A$10·8 million), generating net savings of A$10·2 million and A$1·34 in return per A$1 invested. All scenarios remained cost-effective across sensitivity analyses; while sustained implementation scenario was no longer dominant when treatment price fell to 10% of the PBS-listed price. Interpretation: Sustained national point-of-care HCV testing across community and prison settings was projected to reduce HCV burden and generate long-term health-care savings, mainly through infections averted by treatment as prevention. These findings demonstrate that investment in targeted point-of-care testing infrastructure can deliver health and economic benefits.

Background: The Surveillance and Treatment of Prisoners With Hepatitis C (SToP-C) study demonstrated that scaling up of direct-acting antiviral (DAA) treatment reduced hepatitis C virus (HCV) transmission. We evaluated the cost-effectiveness of scaling up HCV treatment in statewide prison services incorporating long-term outcomes across custodial and community settings. Methods: A dynamic model of incarceration and HCV transmission among people who inject drugs (PWID) in New South Wales, Australia, was extended to include former PWID and those with long-term HCV progression. Using Australian costing data, we estimated the cost-effectiveness of scaling up HCV treatment in prisons by 44% (as achieved by the SToP-C study) for 10 years (2021–2030) before reducing to baseline levels, compared to a status quo scenario. The mean incremental cost-effectiveness ratio (ICER) was estimated by comparing the differences in costs and quality-adjusted life-years (QALYs) between the scale-up and status quo scenarios over 40 years (2021–2060) discounted at 5% per annum. Univariate and probabilistic sensitivity analyses were performed. Results: Scaling up HCV treatment in the statewide prison service is projected to be cost-effective with a mean ICER of A$12 968/QALY gained. The base-case scenario gains 275 QALYs over 40 years at a net incremental cost of A$3.6 million. Excluding DAA pharmaceutical costs, the mean ICER is reduced to A$6 054/QALY. At the willingness-to-pay threshold of A$50 000/QALY, 100% of simulations are cost-effective at various discount rates, time horizons, and changes of treatment levels in prison and community. Conclusions: Scaling up HCV testing and treatment in prisons is highly cost-effective and should be considered a priority in the national elimination strategy. Clinical Trials Registration: NCT02064049.

Spinal muscular atrophy (SMA) and severe combined immunodeficiency (SCID) are rare, inherited genetic disorders with severe mortality and morbidity. The benefits of early diagnosis and initiation of treatment are now increasingly recognized, with the most benefits in patients treated prior to symptom onset. The aim of the economic evaluation was to investigate the costs and outcomes associated with the introduction of universal newborn screening (NBS) for SCID and SMA, by generating measures of cost-effectiveness and budget impact. A stepwise approach to the cost-effectiveness analyses by decision analytical models nested with Markov simulations for SMA and SCID were conducted from the government perspective. Over a 60-year time horizon, screening every newborn in the population and treating diagnosed SCID by early hematopoietic stem cell transplantation and SMA by gene therapy, would result in 95 QALYs gained per 100,000 newborns, and result in cost savings of USD 8.6 million. Sensitivity analysis indicates 97% of simulated results are considered cost-effective against commonly used willingness-to-pay thresholds. The introduction of combined NBS for SCID and SMA is good value for money from the long-term clinical and economic perspectives, representing a cost saving to governments in the long-term, as well as improving and saving lives.

Evidence on the cost-effectiveness of newborn screening (NBS) for severe combined immunodeficiency (SCID) in the Australian policy context is lacking. In this study, a pilot population-based screening program in Australia was used to model the cost-effectiveness of NBS for SCID from the government perspective. Markov cohort simulations were nested within a decision analytic model to compare the costs and quality-adjusted life-years (QALYs) over a time horizon of 5 and 60 years for two strategies: (1) NBS for SCID and treat with early hematopoietic stem cell transplantation (HSCT); (2) no NBS for SCID and treat with late HSCT. Incremental costs were compared to incremental QALYs to calculate the incremental cost-effectiveness ratios (ICER). Sensitivity analyses were performed to assess the model uncertainty and identify key parameters impacting on the ICER. In the long-term over 60 years, universal NBS for SCID would gain 10 QALYs at a cost of US $0.3 million, resulting in an ICER of US$33,600/QALY. Probabilistic sensitivity analysis showed that more than half of the simulated ICERs were considered cost-effective against the common willingness-to-pay threshold of A$50,000/QALY (US$35,000/QALY). In the Australian context, screening for SCID should be introduced into the current NBS program from both clinical and economic perspectives.

Background: Timely diagnosis and treatment of hepatitis C virus (HCV) is critical to achieve elimination goals. This study evaluated the cost-effectiveness of point-of-care testing strategies for HCV compared to laboratory-based testing in standard of care. Methods: Cost-effectiveness analyses were undertaken from the perspective of Australian Governments as funders by modelling point-of-care testing strategies compared to standard of care in needle and syringe programs, drug treatment clinics, and prisons. Point-of-care testing strategies included immediate point-of-care HCV RNA testing and combined point-of-care HCV antibody and reflex RNA testing for HCV antibody positive people (with and without consideration of previous treatment). Sensitivity analyses were performed to investigate the cost per treatment initiation with different testing strategies at different HCV antibody prevalence levels. Findings: The average costs per HCV treatment initiation by point-of-care testing were up to 35% lower (A$890–A$1,406) compared to standard of care (A$1,248–A$1,632) depending on settings. The average costs per treatment initiation by point-of-care testing were A$1,080–A$1,406 for RNA, A$960–A$1,310 for combined antibody/RNA without treatment history consideration, and A$890– A$1,189 for combined antibody/RNA with treatment history consideration. When HCV antibody prevalence was <74%, combined point-of-care HCV antibody and point-of-care RNA testing (with and without consideration of treatment history) were the most cost-effective strategies. Modest increases in treatment uptake by 8%-31% were required for immediate point-of-care HCV RNA testing to achieve equivalent cost per treatment initiation compared to standard of care. Interpretation: Point-of-care testing is more cost-effective than standard of care for populations at risk of HCV. Testing strategies combining point-of-care HCV antibody and RNA testing are likely to be cost-effective in most settings. Funding: National Health and Medical Research Council Declaration of Interest: RTG has received funding for his research from WHO and has provided non-funded project advice to Gilead and ViiV. YS is a co-investigator on investigatorinitiated research grants from AbbVie and Gilead Sciences. ARL has received investigatorinitiated research grants from AbbVie, Gilead Sciences, and Sequiris. GJD is a consultant/advisor and has received research grants from Abbvie, Abbot Diagnostics, Gilead Sciences, Bristol Myers Squibb, Cepheid, GlaxoSmithKline, Merck, Janssen and Roche. JG is a consultant/advisor and has received research grants from AbbVie, Biolytical, Camurus, Cepheid, Gilead Sciences, Hologic, Indivor, and Merck/MSD and has received honoraria from AbbVie, Cepheid, Gilead Sciences, and Merck. No input into this work was provided by any of the above listed organisations or institutions. All other authors have no conflicts of interest to declare.

Objective To assess cost-effectiveness of newborn screening (NBS) for spinal muscular atrophy (SMA) and early treatment with nusinersen or onasemnogene abeparvovec (gene therapy), compared with nusinersen without SMA screening. Methods Informed by an Australian state-wide SMA NBS programme, a decision analytical model nested with Markov models was constructed to evaluate costs and quality-adjusted life-years (QALYs) from a societal perspective with sensitivity analyses. Results By treating one presymptomatic SMA infant with nusinersen or gene therapy, an additional 9.93 QALYs were gained over 60 years compared with late treatment in clinically diagnosed SMA. The societal cost was $9.8 million for early nusinersen treatment, $4.4 million for early gene therapy and $4.8 million for late nusinersen treatment. Compared with late nusinersen treatment, early gene therapy would be dominant, gaining 9.93 QALYs while saving $360 000; whereas early nusinersen treatment would result in a discounted incremental cost-effectiveness ratio (ICER) of $507 000/QALY. At a population level, compared with no screening and late treatment with nusinersen, NBS and early gene therapy resulted in 0.00085 QALY gained over 60 years and saving $24 per infant screened (85 QALYs gained and $2.4 million saving per 100 000 infants screened). More than three quarters of simulated ICERs by probability sensitivity analyses showed NBS and gene therapy would be dominant or less than $50 000/QALY, compared with no screening and late nusinersen treatment. Conclusion NBS coupled with gene therapy improves the quality and length of life for infants with SMA and would be considered value-for-money from an Australian clinical and policy context.

Objective: To examine the health care costs associated with mental disorders and subthreshold mental disorders within a nationally representative sample of children and adolescents in Australia. Method: Data were derived from the Young Minds Matter Survey (N = 6,310). Mental disorders were classified using the Diagnostic Interview Schedule for Children Version IV. Participant data were linked to administrative data on health care costs. Adjusted generalized linear regression models and two-part models were used to estimate mean differences in costs between those with a mental disorder or subthreshold disorder and those without. Results: Costs associated with health care attendances and medications were higher for children and adolescents with mental disorders and subthreshold mental disorders compared to those without a mental disorder. The additional population health care costs due to mental disorders amounted to AUD$234 million annually in children and adolescents, of which approximately 16% was attributed to out-of-pocket costs. Findings showed that those with subthreshold mental disorders or comorbid mental disorders have substantial additional costs of Medicare-funded medical and pharmaceutical services. Conclusion and implication: Mental disorders in children and adolescents are associated with significant health care costs. Further research is needed to ensure that this population is receiving effective and efficient care.

The aim of this economic assessment was to evaluate the impact of a new nurse-led model of care, the Symptom and Urgent Review Clinic (SURC), for patients with cancer experiencing disease- or treatment-related symptoms. An economic assessment was undertaken to estimate costs of the SURC from the service funder perspective and to compare the cost with cost offsets stemming from the implementation of the SURC. The cost offsets focused on the changes in emergency department (ED) presentations and inpatient admissions during a comparable 6-month period before and after the SURC implementation. Costs were analyzed in 2018 Australian dollars, and return on investment was calculated by comparing the cost offsets in the ED and inpatient units with the cost of the SURC. After the implementation of the SURC, patients were less likely to present to the ED (7.2% v 8.5%; P = .01), and patients who did present to the ED were more likely to be admitted to inpatient units (78% v 71%; P = .03) for additional treatment. The post-SURC period had a net cost savings of $37,090 compared with the pre-SURC period. From the service funder perspective, the SURC achieved an investment return of $1.73 for every dollar invested in the new service. Our study establishes the economic credentials of a new care model using empirical linked hospital service data. The SURC presents a new cancer care service for policy consideration from an economic standpoint. It demonstrates an efficient approach to hospital resource allocation to deliver quality cancer care.