Publicly-funded Health Care System Research Articles

Interdisciplinary perspectives on accessing specialty evidence-based treatment for Medicaid-insured adolescents with eating disorders

BackgroundFamily-based treatment (FBT), the leading intervention for adolescents with anorexia nervosa (AN), is severely understudied in outpatient care settings that serve publicly-insured populations. Many individuals with public insurance are lower-income, racially and ethnically diverse, and experience barriers to accessing evidence-based interventions for eating disorders (EDs).MethodsSemi-structured interviews were conducted with ten interdisciplinary providers who provide specialty care to youth with EDs in an inpatient and/or outpatient medical setting. Interview questions were focused on the interdisciplinary providers’ experiences of caring for individuals with EDs, with a focus on differences in care for those with private insurance compared to public insurance. The interviews took place two years after training in FBT was delivered to mental health providers in San Francisco County, which created opportunities to explore provider perspectives on collaborating with newly-trained mental health providers in the community implementing FBT with publicly-insured youth.ResultsContent analysis converged on three themes: the critical importance of supporting mental health treatment within the context of medical care, complex challenges when helping patients and their families navigate publicly-funded health care systems, and the overall positive impact of the FBT rollout in San Francisco County. Participants emphasized greater confidence in patient outcomes when collaborating with FBT providers and noted discord when working with providers not trained in EDs or FBT. Referral systems, weight-based stigma, and a lack of appropriate services were highlighted as significant barriers to care. To facilitate treatment engagement in publicly-insured populations, participants stressed the importance of clinicians providing psychoeducation and providing services with a high degree of cultural competence. Participants expressed that patients’ ability to access FBT and providers’ ability to collaborate on cases markedly improved following the county training, increasing their sense of efficacy in delivering adequate patient care.ConclusionsThe discussed themes highlight the importance of access to FBT for individuals in underserved communities, which can significantly reduce both provider and patient burden. Despite various barriers to utilizing FBT in publicly-funded settings, clinicians stressed that cultural adaptations increase the implementation of and facilitate family engagement in FBT, which is consistent with previous literature examining evidence-based intervention implementation science.

Unrecognised actionability for breast cancer risk variants identified in a national-level review of Australian familial cancer centres

Breast cancer remains a significant global health challenge. In Australia, the adoption of publicly-funded multigene panel testing for eligible cancer patients has increased accessibility to personalised care, yet has also highlighted the increasing prevalence of variants of uncertain significance (VUS), complicating clinical decision-making. This project aimed to explore the spectrum and actionability of breast cancer VUS in Australian familial cancer centers (FCCs). Leveraging data from 11 FCCs participating in the Inherited Cancer Connect database, we retrieved VUS results from 1472 patients. Through ClinVar crosschecks and application of gene-specific ACMG/AMP guidelines, we showed the potential for reclassification of 4% of unique VUS as pathogenic or likely pathogenic, and 80% as benign or likely benign. Surveys conducted with FCCs and diagnostic laboratories described current practices and challenges in variant reclassifications, highlighting resource constraints preventing periodic VUS review and notifications from the laboratories to the FCCs. Our study suggests there are benefits to routine VUS review and reclassification, particularly in publicly-funded healthcare systems. Future research should focus on assessing the clinical impact and cost-effectiveness of implementing routine variant review practices, alongside efforts to enhance communication between FCCs and laboratories.

Assay-guided treatment sequencing in chronic lymphocytic leukemia (CLL): a cost-effectiveness analysis

Costly targeted cancer treatments challenge publicly-funded healthcare systems seeking to align expected benefit with value for money. In 2021, The Canadian Agency for Drugs and Technologies in Health (CADTH) published a provisional funding algorithm for risk-based treatment of chronic lymphocytic leukemia (CLL). We estimate the cost-effectiveness of this algorithm against current standard of care. We constructed a probabilistic Markov model comparing next generation sequencing (NGS) assay-guided front-line treatment of acalabrutinib versus venetoclax with obinutuzumab to a comparator wherein patients initiate acalabrutinib. The primary outcome was the incremental cost-effectiveness ratio (ICER) per quality-adjusted life-year (QALY) gained. Analyses were conducted from the British Columbia healthcare system perspective, with outcomes discounted at 1.5%. Assay informed treatment for patients with CLL resulted in an incremental cost effectiveness ratio of $18,040 (95% CI $16,491–$19,501) per quality adjusted life-year (QALY) gained. The probability of the NGS guided treatment algorithm being cost effective was 80% at a willingness to pay threshold of $50,000 and a corresponding ICER of $18,040. Assay-guided treatment sequencing adds additional costs to healthcare but may be a cost-effective intervention for adult patients with CLL. Integration of real-world evidence would improve the validity and reliability of model estimated for decision-makers.

The effect of local hospital waiting times on GP referrals for suspected cancer.

Reducing waiting times is a major policy objective in publicly-funded healthcare systems. However, reductions in waiting times can produce a demand response, which may offset increases in capacity. Early detection and diagnosis of cancer is a policy focus in many OECD countries, but prolonged waiting periods for specialist confirmation of diagnosis could impede this goal. We examine whether urgent GP referrals for suspected cancer patients are responsive to local hospital waiting times. We used annual counts of referrals from all 6,667 general practices to all 185 hospital Trusts in England between April 2012 and March 2018. Using a practice-level measure of local hospital waiting times based on breaches of the two-week maximum waiting time target, we examined the relationship between waiting times and urgent GP referrals for suspected cancer. To identify whether the relationship is driven by differences between practices or changes over time, we estimated three regression models: pooled linear regression, a between-practice estimator, and a within-practice estimator. Ten percent higher rates of patients breaching the two-week wait target in local hospitals were associated with higher volumes of referrals in the pooled linear model (4.4%; CI 2.4% to 6.4%) and the between-practice estimator (12.0%; CI 5.5% to 18.5%). The relationship was not statistically significant using the within-practice estimator (1.0%; CI -0.4% to 2.5%). The positive association between local hospital waiting times and GP demand for specialist diagnosis was caused by practices with higher levels of referrals facing longer local waiting times. Temporal changes in waiting times faced by individual practices were not related to changes in their referral volumes. GP referrals for diagnostic cancer services were not found to respond to waiting times in the short-term. In this setting, it may therefore be possible to reduce waiting times by increasing supply without consequently increasing demand.

Remote rhythm monitoring using a photoplethysmography smartphone application after cardioversion for atrial fibrillation.

Direct current cardioversion (DCCV) is a commonly utilized rhythm control technique for atrial fibrillation. Follow-up typically comprises a hospital visit for 12-lead electrocardiogram (ECG) two weeks post-DCCV. We report the feasibility, costs, and environmental benefit of remote photoplethysmography (PPG) monitoring as an alternative. We retrospectively analysed DCCV cases at our centre from May 2020 to October 2022. Patients were stratified into those with remote PPG follow-up and those with traditional 12-lead ECG follow-up. Monitoring type was decided by the specialist nurse performing the DCCV at the time of the procedure after discussing with the patient and offering them both options if appropriate. Outcomes included the proportion of patients who underwent PPG monitoring, patient compliance and experience, and cost, travel, and environmental impact. Four hundred sixteen patients underwent 461 acutely successful DCCV procedures. Two hundred forty-six underwent PPG follow-up whilst 214 underwent ECG follow-up. Patient compliance was high (PPG 89.4% vs. ECG 89.8%; P > 0.999) and the majority of PPG users (90%) found the app easy to use. Sinus rhythm was maintained in 71.1% (PPG) and 64.7% (ECG) of patients (P = 0.161). Twenty-nine (11.8%) PPG patients subsequently required an ECG either due to non-compliance, technical failure, or inconclusive PPG readings. Despite this, mean healthcare costs (£47.91 vs. £135 per patient; P < 0.001) and median cost to the patient (£0 vs. £5.97; P < 0.001) were lower with PPG. Median travel time per patient (0 vs. 44 min; P < 0.001) and CO2 emissions (0 vs. 3.59 kg; P < 0.001) were also lower with PPG. No safety issues were identified. Remote PPG monitoring is a viable method of assessing for arrhythmia recurrence post-DCCV. This approach may save patients significant travel time, reduce environmental CO2 emission, and be cost saving in a publicly-funded healthcare system.

Anatomy of the elephant in Quebec family practice

Background&#x0D; Whole person care has been a foundation of the modern practice of family medicine, and therefore one might expect that such an approach by family practitioners would evolve and mature over time. Some of these clinicians, however, currently question their ability to provide such holistic care because of external factors they perceive as negative, and over which they have little or no control.&#x0D; Objectives&#x0D; This presentation will explore perceived inhibiting factors affecting family doctors ability to provide whole person care within the publicly-funded health care system of the Canadian province of Quebec. &#x0D; Method&#x0D; The presenter, an academic and clinician scientist with forty-four years of experience practicing family medicine, will present personal perspectives, along with those gathered informally from a broad cadre of colleagues working in different settings. Using the idiom of “elephant in the room” to identify problems or obstacles that may not be voiced, this talk will start with consideration of elephant anatomy as being comprised of thirteen distinct anatomical parts that contribute to a functional whole. An analogy will be developed in which thirteen distinct factors are presented as likely impeding or discouraging whole person care by family physicians. &#x0D; Conclusion&#x0D; Some agendas and policies of health care planners and administrators, either alone or collectively, and intentionally or unintentionally, may decrease opportunity or ability of family physicians to provide whole person care.

Cost-Effectiveness Analysis and Budget Impact Analysis of Enzalutamide for the Treatment of Metastatic Hormone-Sensitive Prostate Cancer

We conducted cost-effectiveness analysis and budget impact analysis for androgen deprivation therapy (ADT) plus enzalutamide (ENZ) on patients with metastatic hormone-sensitive prostate cancer (mHSPC) from the publicly-funded healthcare system perspective. Using a partitioned survival model, lifetime costs, quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs) of ADT＋ENZ were estimated against ADT alone, ADT plus abiraterone (ADT＋ABI), and ADT plus apalutamide (ADT＋APA). Total healthcare cost differences with and without ENZ in mHSPC therapy were estimated for the period from 2022 to 2026. Based on cost-effectiveness analysis, the ICER of ADT＋ENZ versus ADT alone was estimated as ￥7.18 million/QALY gained. ADT＋ABI and ADT＋APA were dominated options (extended dominance). Budget impact analysis showed that incorporation of ENZ had a net budget impact of ￥57.19 billion, an 8.4% increase, over these 5 years. This amounted to a budgetary impact of ￥16,000 per patient per month at year 5. However, the number of patients with disease progressed to metastatic castration-resistant prostate cancer (mCRPC) would be reduced from 79,000 (without ENZ) to 65,000 (with ENZ), resulting in a 17% cost reduction within the mCRPC phase. In conclusion, ADT＋ENZ would be a cost-effective option, at the willingness to pay threshold of ￥7.5 million/QALY gained. Introduction of ENZ in the mHSPC treatment would result in a marginal increase in the total budget. However, ENZ is also expected to provide clinical benefits in reducing the number of patients with disease that would otherwise progress to mCRPC during these 5 years, resulting in cost savings in this phase.

Assessing the Impact of Marginalization on Survival for Patients Undergoing Autologous Stem Cell Transplant in Ontario, Canada

Background Autologous hematopoietic stem cell transplants (ASCT) are potentially life-saving interventions used to treat hematologic disorders. However, it is unclear if socioeconomic disparities impact the patient benefit from ASCT. Recent evidence suggests lower rates of ASCT for patients living in rural settings and for ethnic minority groups. Few population-based studies have assessed comprehensive indices of marginalization as predictors of outcomes after ASCT. In a publicly funded healthcare setting, it is crucial to understand if various aspects of marginalization interact to ultimately impact survival for patients undergoing ASCT. Methods We performed a retrospective population-based study using administrative healthcare databases from Ontario, Canada. Patients were included if they had undergone auto-SCT for lymphoma or myeloma between 2010 and 2022. The Ontario Marginalization Index (ON-Marg), created using Canadian census data, explores four key aspects of marginalization: residential instability (referring to housing instability), material deprivation (referring to individual and community abilities to access basic material needs), dependency (referring to lack of income from employment), and ethnic concentration (referring to individuals who are recent immigrants or belonging to a visible minority group). The primary outcome was 2-year overall survival (OS) from time of transplant to death or end of the study period stratified by ON-Marg quintiles. Multivariable Cox regression analyses were used to identify baseline characteristics associated with OS. Results A total of 1886 patients underwent ASCT for lymphoma and 2842 patients underwent ASCT for myeloma. The median age of patients undergoing ASCT for lymphoma was 56 years (IQR 44-63) and 64% of patients were male. 22% of patients had high aggregated diagnosis group (ADG) comorbidity scores and 15% had a prior history of cancer. 2-year OS for patients in the fifth quintile of marginalization of the ethnic concentration index was 80.4% (95% CI 75.9-84.1%), compared to 72.3% (95% CI 67.5-76.5%) in the first quintile ( Figure 1A). This survival advantage (HR 0.71, 95% CI 0.52-0.99) persisted after adjusting for age, comorbidity burden, and distance to hospital ( Table 1). Patients who lived 150-200 km from the transplant center had a lower risk of death compared to patients who lived within 50km of the hospital (HR 0.53, 95% CI 0.30-0.94). 2-year OS was not significantly different for patients in quintile 5 of marginalization of the dependency index compared to quintile 1 (HR 1.25, 95% CI 0.93-1.67), with similarly no significant difference across residential instability or material deprivation. The median age of patients undergoing ASCT for myeloma was 61 years (IQ 55-66), and 25% of patients had high ADG comorbidity scores. OS was not significantly different across all domains of marginalization, with a 2-year OS of 88.8% (95% CI 85.8-91.2%) in quintile 1 of ethnic deprivation and 87.5% (95% CI 84.7-89.8%) in quintile 5 ( Figure 1B). Higher comorbidity scores were associated with an increased risk of death compared to lower comorbidity scores (HR 2.17, 95% CI 1.58-2.98), as shown in Table 1. Living 150-200 km from the transplant center was associated with worse OS (HR 1.74, 95% CI 1.24-2.45). Conclusion This is one of the first studies to examine the effect of marginalization on outcomes after ASCT in a publicly-funded healthcare system. For patients undergoing ASCT for lymphoma, patients in quintile 5 of ethnic concentration had improved 2-year OS compared to patients in quintile 1. One possible explanation is the healthy immigrant effect, whereby immigrant patients are healthier than their Canadian-born counterparts. Cultural factors may also have a protective role. Patients undergoing ASCT for lymphoma who lived farther from the transplant center had a lower risk of death; these patients may be more highly selected based on fitness and disease biology to be referred for transplant compared to patients who live within close proximity. For patients undergoing ASCT for myeloma, 2-year OS was not affected by ethnic concentration quintile. The decision for referral for ASCT for myeloma is not as subjective, with ASCT commonly used in the first-line setting for eligible patients regardless of distance, which likely accounts for this difference. Further work is needed to ensure that all eligible patients receive ASCT.

Clinical Benefits, Harms, and Cost-Effectiveness of Indefinite Anticoagulant Therapy for First Unprovoked Venous Thromboembolism: A Decision Analytic Modelling Study

Background: Anticoagulation for 3-6 months is the minimum required duration of treatment for patients with venous thromboembolism (VTE). Whether to extend treatment beyond the initial 3-6 months of anticoagulation in patients with a first unprovoked VTE has been debated. The American Society of Hematology guidelines suggest continuing anticoagulation indefinitely over discontinuing anticoagulation after completing 3-6 months of initial treatment for a first unprovoked VTE, except in patients who have a high risk for bleeding (Ortel T, Blood Adv 2018). However, the lifetime clinical benefits, harms, and cost-effectiveness of indefinite anticoagulation have not been formally assessed. While a randomized controlled trial (RCT) would be the optimal study design to provide evidence for or against continuing anticoagulation indefinitely in patients with a first unprovoked VTE, it is unlikely to be conducted due to the lifelong (i.e. until death) follow-up and extremely large sample size that would be required.Decision-analytic modelling can provide evidence to inform guidelines under circumstances in which RCTs are unfeasible. Objective: The aim of this modelling study was to compare the clinical benefits, harms, and cost-effectiveness of continuing versus discontinuing anticoagulation indefinitely after completing initial treatment for a first unprovoked VTE. Methods: We created a probabilistic Markov cohort simulation model [Figure 1] to simulate costs (adjusted to 2022 Canadian dollars) and outcomes (recurrent VTE, major bleeding, and quality-adjusted life-years [QALYs]) for two hypothetical cohorts of patients aged 55 years with a first unprovoked VTE that had completed 3-6 months of initial anticoagulant therapy - one cohort assigned to continue anticoagulation indefinitely with direct oral anticoagulants, and another assigned to discontinue anticoagulation indefinitely. The model adopted a cycle length of 1 month and a lifetime horizon. The economic analysis adopted a third-party payer perspective relating to the Canadian publicly-funded healthcare system. The long-term risks and case-fatality rates of recurrent VTE and major bleeding, with and without extended anticoagulation, were sourced from our recently published systematic reviews (Khan F, BMJ 2019;Khan F, Ann Intern Med 2021). Additional data related to transition probabilities, costs, and utility values were obtained from highest quality published sources identified through a targeted literature search. Future costs and QALYs were discounted at an annual rate of 1.5% as per Canadian guidelines. The model structure, input parameters, and assumptions were validated by clinical experts to ensure that they coincided with current clinical practice. Results: In a hypothetical cohort of 1000 patients, continuing anticoagulation indefinitely, compared to discontinuing anticoagulation, prevented 253 recurrent VTE events (number needed to treat = 4) and induced an additional 69 major bleeding events (number needed to harm = 15). Indefinite anticoagulation resulted in higher health system costs ($70,019.35 vs. $61,438.10 per person) and no improvement in QALYs (15.26 vs. 15.31 per person; incremental difference of -0.05 QALYs or -18.25 quality adjusted life-days) [Table 1]. Model results were most sensitive to the annual risk of major bleeding and case-fatality rate of major bleeding during extended anticoagulation however, in all one-way sensitivity analyses, discontinuing anticoagulation remained economically dominant (i.e., lower costs and similar QALYs) over continuing anticoagulation indefinitely, at a willingness-to-pay (WTP) threshold of Can$50,000 per QALY. In probabilistic analyses, the probability of indefinite anticoagulation being the cost-effective treatment strategy was 0% at a WTP threshold of Can$50,000 or Can$100,000 per QALY. Conclusions: Indefinite anticoagulation for a first unprovoked VTE is unlikely to either result in a net clinical benefit or be cost-effective. With no net gain in QALYs (albeit a small net loss), there is no rationale for continuing anticoagulation indefinitely in all (i.e., unselected) patients with a first unprovoked VTE. However, the clinical and cost-effectiveness of indefinite anticoagulation in subgroups of patients at high risk of recurrent VTE and low risk of major bleeding requires further investigation, and will be an important next step. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

P11.41.B The Cost of Inpatient Care For Adult Primary Brain Tumour Patients in England

Abstract Background Primary brain tumours are rare, but are the cause of the most life-years lost of any cancer. Given their poor prognosis, questions about their treatment and care costs are important especially in publicly-funded healthcare systems. There is currently very little robust data on the cost of care for brain tumour patients. Here we present up-to-date estimations of the direct inpatient care costs of all adult primary brain tumour patients in England, with a specific breakdown of costs attributed to cranial glioblastomas and cranial meningiomas, the commonest primary brain tumours. Material and Methods GlioCova uses a linked English national cancer data on over 50,000 adult primary brain tumour patients diagnosed between 2013-2018, with data on secondary healthcare activities three months before diagnosis - the last three months of 2012, and follow-up data after diagnosis - up to the last admission of 2019. We examined inpatient care with a breakdown of costs attributed to different treatment types: neurosurgery, chemotherapy and radiotherapy. We used the NHS HRG4+ Reference Costs Grouper 2017/2018 to assign costs matched from the National Schedule of Reference Costs 2017/2018. We converted all values to 2021 using the Health CPI Index. Results There were a total of 51,775 adult primary brain tumour patients diagnosed in England in the 6 year period between 2013 and 2018. 48,608 of these were admitted to hospital between the last three months of 2012 and the end of 2019, of which we were able to assign costs to 47,521 patients (98%). Total inpatient costs for the whole brain tumour cohort were over £973 million during the time period (34% attributed to inpatient care for neurosurgery, chemotherapy, and radiotherapy). 14,691 (31%) of patients were diagnosed with a cranial glioblastoma, and 9,501 (20%) of patients were diagnosed with a cranial meningioma. Total inpatient costs were £349 million and £163 million for glioblastoma and meningioma respectively, with 34% again devoted to direct treatment costs. Conclusion The estimated direct inpatient care costs for all 51,775 primary brain tumour patients diagnosed in England between 2013 and 2018 were nearly £1 billion (£166 million/year cohort), 2/3 of which was not for direct treatment. This does not include outpatient chemotherapy and radiotherapy costs, other outpatient appointments, patient out-of-pocket costs, primary care, social care, or end-of-life care costs. Future work will examine variation in care and costs and extend the analysis to include outpatients. We hope these data will help make an economic argument for improving care for brain tumour patients. More information on GlioCova: https://blogs.imperial.ac.uk/gliocova/about-gliocova/.

Socioeconomic inequality in access to timely and appropriate care in emergency departments

In publicly-funded healthcare systems, waiting times for care should be based on need rather than ability to pay. Studies have shown that individuals with lower socioeconomic status face longer waits for planned inpatient care, but there is little evidence on inequalities in waiting times for emergency care. We study waiting times in emergency departments (EDs) following arrival by ambulance, where health consequences of extended waits may be severe. Using data from all major EDs in England during the 2016/17 financial year, we find patients from more deprived areas face longer waits during some parts of the ED care pathway. Inequalities in waits are small, but more deprived individuals also receive less complex ED care, are less likely to be admitted for inpatient care, and are more likely to re-attend ED or die shortly after attendance. Patient-physician interactions and unconscious bias towards more deprived patients may be important sources of inequalities.

Surgical wait times and socioeconomic status in a public healthcare system: a retrospective analysis

BackgroundOne aim of publicly-funded health care systems is to provide equitable access to care irrespective of ability to pay. At the same time, differences in socioeconomic status (SES) are associated with health outcomes and access to care, including waiting times for surgery. In public systems where both high- and low-SES patients use the same resources, low-SES patients may be adversely impacted in surgical waiting times. The purpose of this study was to determine whether a publicly-funded health system can provide equitable access to surgical care across socioeconomic status.MethodsPatient-level records were obtained from a comprehensive provincially-administered surgical wait time database, encompassing years 2006–2015 and 98% of Ontario hospitals. Patient SES was determined by linking postal code with the Material and Social Deprivation Index. Surgical waiting times (time in days between decision to treat and surgery) accounted for patient-initiated delays in treatment, and regression analysis considered age, SES, rurality, sex, priority level for surgical urgency (assigned by surgeons), surgical subspecialty, number of visits, and procedure year.ResultsFor the 4,253,305 surgical episodes, the mean wait time was 62.3 (SD 75.4) days. Repeated measures least squares regression analysis showed the least deprived SES quintile waited 3 days longer than the most deprived quintile. Wait times dropped in the initial study period but then increased. The proportion of procedures exceeding wait time access targets remained low at 11–13%.ConclusionsThe least deprived SES quintile waited the longest, although the absolute difference was small. This study demonstrates that publicly-funded healthcare systems can provide equitable access to surgical care across SES.

The Soteria model: implementing an alternative to acute psychiatric hospitalization in Israel

ABSTRACT Background Since 2016, Soteria homes have been operating in Israel. In this report we describe the implementation of the model. Methods Data for 486 residents staying in one of three Soteria homes from 2016 through 2020 are presented. The model, and necessary modifications, are discussed. Results The majority of the residents in the Soteria homes suffered from psychotic (41.3%) or bipolar disorder (20.9%) and were of a mean age of 34.5 (SD = 12.83). While operating according to the principles of Soteria, adaptations had to be made. The homes used professional staff as well as companions. Accepting a wide range of residents exposed the home to situations of violent behaviour which required adjustment in admission policy. Work with insurers limited the possible length of stay. Financial constraints expanded the home capacity to 10 residents, while limiting work shifts to 12 hours. Cultural considerations led to the establishment of single-gender homes. Discussion Soteria homes can be a viable component of publicly-funded mental health care systems. The implementation of the Soteria model can provide important lessons for the future development of a professional and humane mental health care service – not as an alternative, but as an integral part of the system.

Cost-effectiveness analysis of first-line treatment with crizotinib in ROS1-rearranged advanced non-small cell lung cancer (NSCLC) in Canada

IntroductionWhile no direct comparative data exist for crizotinib in ROS1+ non-small cell lung cancer (NSCLC), studies have suggested clinical benefit with this targeted agent. The objective of this study was to assess the cost-effectiveness of crizotinib compared to standard platinum-doublet chemotherapy for first-line treatment of ROS1+ advanced NSCLC.MethodsA Markov model was developed with a 10-year time horizon from the perspective of the Canadian publicly-funded health care system. Health states included progression-free survival (PFS), up to two further lines of therapy post-progression, palliation and death. Given a lack of comparative data and small study samples, crizotinib or chemotherapy studies with advanced ROS1+ NSCLC patients were identified and time-to-event data from digitized Kaplan-Meier curves were collected to pool PFS data. Costs of drugs, treatment administration, monitoring, adverse events and palliative care were included in 2018 Canadian dollars, with 1.5% discounting. An incremental cost-effectiveness ratio (ICER) was estimated probabilistically using 5000 simulations.ResultsIn the base-case probabilistic analysis, crizotinib produced additional 0.885 life-years and 0.772 quality-adjusted life-years (QALYs) at an incremental cost of $238,077, producing an ICER of $273,286/QALY gained. No simulations were found to be cost-effective at a willingness-to-pay threshold of $100,000/QALY gained. A scenario analysis assuming efficacy equivalent to the ALK+ NSCLC population showed a slightly more favorable cost-effectiveness profile for crizotinib.ConclusionsAvailable data appear to support superior activity of crizotinib compared to chemotherapy in ROS1+ advanced NSCLC. At the list price, crizotinib was not cost-effective at commonly accepted willingness-to-pay thresholds across a wide range of sensitivity analyses.

The Canadian prospective cohort study to understand progression in multiple sclerosis (CanProCo): rationale, aims, and study design

BackgroundNeurological disability progression occurs across the spectrum of people living with multiple sclerosis (MS). Although there are a handful of disease-modifying treatments approved for use in progressive phenotypes of MS, there are no treatments that substantially modify the course of clinical progression in MS. Characterizing the determinants of clinical progression can inform the development of novel therapeutic agents and treatment approaches that target progression in MS, which is one of the greatest unmet needs in clinical practice. Canada, having one of the world’s highest rates of MS and a publicly-funded health care system, represents an optimal country to achieve in-depth analysis of progression. Accordingly, the overarching aim of the Canadian Prospective Cohort Study to Understand Progression in MS (CanProCo) is to evaluate a wide spectrum of factors associated with the clinical onset and rate of disease progression in MS, and to describe how these factors relate to one another to influence progression.MethodsCanProCo is a prospective, observational cohort study with investigators specializing in epidemiology, neuroimaging, neuroimmunology, health services research and health economics. CanProCo’s study design was approved by an international review panel, comprised of content experts and key stakeholders. One thousand individuals with radiologically-isolated syndrome, relapsing-remitting MS, and primary-progressive MS within 10–15 years of disease onset will be recruited from 5 academic MS centres in Canada. Participants will undergo detailed clinical evaluation annually over 5 years (including advanced, app-based clinical data collection). In a subset of participants within 5–10 years of disease onset (n = 500), blood, cerebrospinal fluid, and research MRIs will be collected allowing an integrated, in-depth evaluation of factors contributing to progression in MS from multiple perspectives. Factors of interest range from biological measures (e.g. single-cell RNA-sequencing), MRI-based microstructural assessment, participant characteristics (self-reported, performance-based, clinician-assessed, health-system based), and micro and macro-environmental factors.DiscussionHalting the progression of MS remains a fundamental need to improve the lives of people living with MS. Achieving this requires leveraging transdisciplinary approaches to better characterize why clinical progression occurs. CanProCo is a pioneering multi-dimensional cohort study aiming to characterize these determinants to inform the development and implementation of efficacious and effective interventions.

Public satisfaction with health care system in 30 countries: The effects of individual characteristics and social contexts

In this article, the associations among individual socio-economic characteristics, the institutional set-up of health care systems, and satisfaction with the health care system are investigated. Data from the 2011 International Social Survey Program (30 countries, 34,212 respondents) is used. Multilevel analyses across countries have shown how the state financing context affects satisfaction at the individual level. Consistent with previous research, at individual level, personal experiences with medical providers, age, gender as well as income are significant predictors of satisfaction with the health care system. At the country level, real input indicators such as density of physicians and density of hospital beds are negative predictors of satisfaction with the health care system whereas the percentage of total health expenditures comprised by public sources is a positive predictor of satisfaction with the health care system. However, findings from the cross-level interactions indicate that the negative effect of lower income is more prominent in predominantly publicly-funded health care systems. Specifically, in primarily publicly-funded health care systems, the model-predicted probability of satisfaction with the health care system is higher, but the gap in the probability of satisfaction with the health care system between individuals with lower income and those with higher income is greater than that in mostly privately-financed health care systems. The findings in this study suggest that the future direction of health care system reform should be focused on balancing the distribution of resources between private and public sectors.

A Decision Model to Estimate Risk Thresholds for Guiding Duration of Anticaogulation in Unprovoked Venous Thromboembolism

▪Background: For patients with a first unprovoked venous thromboembolism (VTE), the optimal duration of anticoagulant therapy (AT) is a crucial clinical dilemma which has yet to be resolved . Choosing short-term (3-6 months) or indefinite AT, requires balancing the long-term risk of recurrent VTE when AT is discontinued, with the long-term risk of major bleeding (MB) from extended AT. Current guidelines suggest considering indefinite AT in all unprovoked VTE patients with non-high bleeding risk. This however, is a weak-evidence recommendation based on limited data, and no precise risk thresholds have been established to help guide treatment duration in this patient population. From an economic perspective, we aimed to identify thresholds of the risk of MB and recurrent VTE for which indefinite AT, as compared to stopping AT after initial short-term treatment, may be warranted.Methods: We constructed a Markov decision model of patients with a first unprovoked VTE who had completed at least 3-6 months of initial AT. The model adopted a lifetime horizon and a publicly-funded healthcare system perspective. Using a cycle length of one year, the model tracked recurrent VTE (DVT and/or PE), MB, and death from any cause [Figure 1]. For the base case, we considered patients aged 57 years, having a life expectancy of 30 years, annual risk of MB on Vitamin K Antagonists of 1.5%, annual recurrent VTE risk of 5% per year off therapy, case-fatality-rate (CFR) for MB of 11.3%, and CFR for recurrent VTE of 3.6%. We expressed our results as Incremental Net Benefit (INB) which incorporates costs, life-expectancy, quality-adjusted life-years (QALYs), and a willingness to pay (WTP) value. INB is the incremental benefit, expressed in monetary terms ($), of offering a patient treatment (indefinite AT) rather than the comparator (stopping AT). An INB > 0 favours the treatment, whereas an INB < 0 favours the comparator.Results: For the base case analysis with assuming a decision-maker's WTP threshold of $50,000/QALY, indefinite AT was not justified (i.e. INB < 0) when the annual risk of recurrent VTE off therapy was less than 7.1% [Figure 2], or when the annual risk of MB exceeded 2.4% [Figure 3]. Furthermore, in a two-way sensitivity analysis [Figure 4], as the MB risk increased, so did the recurrent VTE risk threshold for which indefinite AT would be warranted. As illustrated through three hypothetical situations outlined in Figure 4; for a patient with an annual MB risk of 3.5% [scenario A], a recurrence risk of >15% would be required for indefinite AT to be favorable (i.e. INB > 0). Similarly, using a base case annual MB risk of 1.5% [scenario B], from an economic standpoint, the decision to offer indefinite AT would not be preferred for a patient in whom the risk of recurrence is 3%, but may be supported for a patient in whom the risk of recurrence is 10%. Lastly, for a patient with an annual recurrent VTE risk of < 1% [scenario C], continuing AT indefinitely would never be justified, irrespective of the risk of MB.Conclusions/Implications: To the best of our knowledge, this is the first decision analysis of a hypothetical cohort of unprovoked VTE patients that identifies risk thresholds of MB and recurrent VTE to guide treatment duration. From an economic viewpoint, our analysis demonstrates that choosing indefinite AT in such patients may not be justified at the proposed risk thresholds. Furthermore, long-term follow up demonstrates that VTE recurs in less than 50% of such patients after stopping AT;thus, considering indefinite AT in all unprovoked VTE patients is an inefficient use of resources, and exposes a large proportion of patients to the unnecessary risks, burdens and costs of lifelong AT. Establishing risk thresholds, as achieved through our decision model, allows individualized treatment and helps guide patient-centered decision making. If validated and further optimized (as planned prior to the ASH), findings from our study could change clinical practice. [Display omitted] DisclosuresNo relevant conflicts of interest to declare.

The role of professional elites in healthcare governance: Exploring the work of the medical director

Medical leaders occupy a prominent position in healthcare policy in many countries, both in terms of the governance of quality and safety within healthcare organisations, and in broader system-wide governance. There is evidence that having doctors on hospital boards is associated with higher quality services. What is not known is how they have this effect. Analysing data collected from observations, interviews and documents from 15 healthcare providers in England (2014–2019), we elaborate the role of medical directors in healthcare governance as ‘translation work’, ‘diplomatic work’, and ‘repair work’. Our study highlights the often enduring emotional effects of repeated structural changes to clinical services. It also contributes to theories of professional restratification, showing the work of medical directors as regional ‘political elites’, and as ‘corporate elites’ in publicly-funded healthcare systems.

Determinants of attitude and intention towards private health insurance: a comparison of insured and uninsured young adults in Australia

BackgroundSince the introduction in 1984 of Australia’s publicly-funded universal healthcare system, Medicare, healthcare financing has relied on a mix of public and private sources to meet the needs of the population (Sowa et al., Appl Health Econ Health Policy 15:31–41, 2018). However, in recent years, there has been a decline in the number of Australians choosing to purchase private health insurance (PHI), particularly within the young adult age group with the proportion of insurance customers aged 20 to 29 falling from 10.3 to 9.4% between 2012 and 2017 (Sivey, The Conversation, 2017). Young adults are critical to private health insurance funding models as their involvement offsets the drawdown by older adults (Dalzell and Borys, ABC News, 2019). While this issue is widely reported in the Australian media, few empirical studies have explored the factors that enable or constrain young adults’ enrolment in PHI.MethodsTo address the scarcity of research about the motivational factors behind young adult decision-making, this study conducted a survey of 594 Australian young adults aged between 18 and 30 years. Within this age group, the survey sought an equal split of participants who were members and non-members of PHI schemes.ConclusionThe findings identified perceived value and trust in insurers as additional motivational factors alongside traditional measures of recognition of the problem and involvement in the problem. Differences between the insured and uninsured groups were identified which help to shape a more holistic understanding of the key motivational factors and barriers in relation to Australian young adults’ enrolment in PHI.

The Integrated Tracking, Referral, and Electronic Decision Support, and Care Coordination (I-TREC) program: scalable strategies for the management of hypertension and diabetes within the government healthcare system of India

BackgroundHypertension and diabetes are among the most common and deadly chronic conditions globally. In India, most adults with these conditions remain undiagnosed, untreated, or poorly treated and uncontrolled. Innovative and scalable approaches to deliver proven-effective strategies for medical and lifestyle management of these conditions are needed.MethodsThe overall goal of this implementation science study is to evaluate the Integrated Tracking, Referral, Electronic decision support, and Care coordination (I-TREC) program. I-TREC leverages information technology (IT) to manage hypertension and diabetes in adults aged ≥30 years across the hierarchy of Indian public healthcare facilities. The I-TREC program combines multiple evidence-based interventions: an electronic case record form (eCRF) to consolidate and track patient information and referrals across the publicly-funded healthcare system; an electronic clinical decision support system (CDSS) to assist clinicians to provide tailored guideline-based care to patients; a revised workflow to ensure coordinated care within and across facilities; and enhanced training for physicians and nurses regarding non-communicable disease (NCD) medical content and lifestyle management. The program will be implemented and evaluated in a predominantly rural district of Punjab, India. The evaluation will employ a quasi-experimental design with mixed methods data collection. Evaluation indicators assess changes in the continuum of care for hypertension and diabetes and are grounded in the Reach, Effectiveness, Adoption Implementation, and Maintenance (RE-AIM) framework. Data will be triangulated from multiple sources, including community surveys, health facility assessments, stakeholder interviews, and patient-level data from the I-TREC program’s electronic database.DiscussionI-TREC consolidates previously proven strategies for improved management of hypertension and diabetes at single-levels of the healthcare system into a scalable model for coordinated care delivery across all levels of the healthcare system hierarchy. Findings have the potential to inform best practices to ultimately deliver quality public-sector hypertension and diabetes care across India.Trial registrationThe study is registered with Clinical Trials Registry of India (registration number CTRI/2020/01/022723). The study was registered prior to the launch of the intervention on 13 January 2020. The current version of protocol is version 2 dated 6 June 2018.