A cost-utility analysis of thrombopoietin receptor agonists for treating pediatric immune thrombocytopenia purpura after failure of first-line therapies.

BACKGROUND: Thrombopoietin receptor agonists (TPO-RAs) have emerged as a recommended treatment for children with persistent and/or chronic immune thrombocytopenic purpura (ITP). The purpose of this study was to evaluate the cost-effectiveness of TPO-RAs relative to treatment without TPO-RAs (non-TPO-RAs/usual care) for ITP in children who do not respond to first-line therapy and in whom splenectomy is not recommended in Ontario, Canada, from a hospital payer perspective. PROCEDURE: A 2-year Markov model with an embedded decision tree was used. Data on medications used, dose, response rate, bleeding, and emergency treatment events were collected from the Hospital for Sick Children in Toronto. The health outcomes were described in quality-adjusted life-years (QALYs). Health-state utilities were derived from the peer-reviewed literature. Scenario analyses, deterministic, and probabilistic sensitivity analyses were conducted. Economic costs were measured in 2021 Canadian dollars ($1.00 = US$0.80) RESULTS: TPO-RAs are estimated to result in an increased cost of $27,118 and a QALY gain of 0.21 compared to non-TPO-RAs over a 2-year horizon, resulting in an incremental cost-effectiveness ratio (ICER) of $129,133. In a 5-year scenario analysis, the ICER fell to $76,403. In the probabilistic sensitivity analysis, TPO-RAs exhibit a 40.0% probability of being cost-effective at a conventional ($100,000) willingness-to-pay threshold per QALY gained. CONCLUSIONS: Further assessment of the long-term efficacy of TPO-RAs is warranted to obtain more precise long-term estimates. As the costs of TPO-RAs decline with the introduction of generic formulations, TPO-RAs may be increasingly cost-effective.

A Cost Analysis of Pulse Oximetry as a Determinant in the Decision to Admit Infants With Mild to Moderate Bronchiolitis.

OBJECTIVES: A previous randomized controlled trial showed that artificially elevating the pulse oximetry display resulted in fewer hospitalizations with no worse outcomes. This suggests that management decisions based mainly on pulse oximetry may unnecessarily increase health care costs. This study assessed the incremental cost of altered relative to true oximetry in infants with mild to moderate bronchiolitis. METHODS: A cost analysis was undertaken from the health care system and societal perspectives using patient-level data from the randomized controlled trial, with a 5-day time horizon after emergency department visit. Infants aged 4 weeks to 12 months with mild to moderate bronchiolitis were randomized to pulse oximetry measurements with true or altered saturation values displayed by artificially increasing saturation 3% points above true values. Direct and indirect health care costs were measured. Sensitivity analyses were performed to assess parameter uncertainty. RESULTS: From the health care system perspective, the average cost per patient was Can $1155 for altered oximetry and $1967 for true oximetry, with a net savings of $812. From a societal perspective, the average cost per patient was $1559 for altered oximetry and $2473 for true oximetry, with a net savings of $914. Probabilistic analyses demonstrated that altered oximetry remained the less costly study group, with an average savings of $810 (95% confidence interval, $748-$872) from the health care system perspective and $910 (95% confidence interval, $848-$973) from the societal system perspective. CONCLUSIONS: Reliance on oximetry as a major determinant in the decision to hospitalize infants with mild to moderate bronchiolitis is associated with significantly greater costs.

Cost-Utility Analysis of Electroconvulsive Therapy and Repetitive Transcranial Magnetic Stimulation for Treatment-Resistant Depression in Ontario.

OBJECTIVES: To evaluate the cost-effectiveness of repetitive transcranial magnetic stimulation (rTMS) and electroconvulsive therapy (ECT), and combining both treatments in a stepped care pathway for patients with treatment-resistant depression (TRD) in Ontario. METHODS: A cost-utility analysis evaluated the lifetime costs and benefits to society of rTMS and ECT as first-line treatments for TRD using a Markov model, which simulates the costs and health benefits of patients over their lifetime. Health states included acute treatment, maintenance treatment, remission, and severe depression. Treatment efficacy and health utility data were extracted and synthesized from randomized controlled trials and meta-analyses evaluating these techniques. Direct costing data were obtained from national and provincial costing databases. Indirect costs were derived from government records. Scenario, threshold, and probabilistic sensitivity analyses were performed to test robustness of the results. RESULTS: rTMS dominated ECT, as it was less costly and produced better health outcomes, measured in quality-adjusted life years (QALYs), in the base case scenario. rTMS patients gained an average of 0.96 additional QALYs (equivalent to approximately 1 year in perfect health) over their lifetime with costs that were $46,094 less than ECT. rTMS remained dominant in the majority of scenario and threshold analyses. However, results from scenarios in which the model's maximum lifetime allowance of rTMS treatment courses was substantially limited, the dominance of rTMS over ECT was attenuated. The scenario that showed the highest QALY gain (1.19) and the greatest cost-savings ($46,614) was when rTMS nonresponders switched to ECT. CONCLUSION: From a societal perspective utilizing a lifetime horizon, rTMS is a cost-effective first-line treatment option for TRD relative to ECT, as it is less expensive and produces better health outcomes. The reduced side effect profile and greater patient acceptability of rTMS that allow it to be administered more times than ECT in a patient's lifetime may contribute to its cost-effectiveness.

Phase-specific healthcare costs of cervical cancer: estimates from a population-based study.

BACKGROUND: There is a lack of evidence on the economic burden of managing cervical cancer in the public payer Canadian setting. OBJECTIVE: We used individual patient-level data to obtain a comprehensive estimate of the cost of managing cervical cancer in the province of Ontario, identifying main cost drivers and predictors of increased costs. STUDY DESIGN: The cost-of-illness technique was used to estimate the incremental costs associated with cervical cancer in 4 phases: prediagnosis, initial care, continuing care, and terminal care. All patients with cervical cancer diagnosed between 2005 and 2009 in the province of Ontario were propensity-score matched to 5 noncancer controls on birth year, income quintile, rurality, comorbidities, and patterns of healthcare utilization pattern during the 2 years before cancer diagnosis. Both cases and the noncancer comparison group were followed to death or March 31, 2013. Costs for all healthcare services paid for by the Ontario Ministry of Health and Long-term Care during the follow-up period were estimated by the use of linked administrative data. Incremental costs for managing cervical cancer were calculated through generalized estimating equations. Predictors of greater health costs were explored using multivariate quantile regression models. RESULTS: All costs were presented in 2012 Canadian dollars ($1.00CDN = $1.00USD). The total incremental costs for managing cervical cancer were $362 in the pre-diagnosis phase, $15,722 in the initial phase, $3924 per year in the continuing phase, and $52,539 in the terminal phase. Inpatient care accounted for 34%, 28%, and 52% of total healthcare cost in the initial, continuing, and terminal phase, respectively. Physician services ranked first in the continuing phase (30%) and second in the initial (26%) and terminal (13%) phases. Advanced age, advanced cancer stage at diagnosis, and comorbidities were significant predictors of greater costs in most care phases. CONCLUSION: Aggregate costs of care for cervical cancer are substantial and vary by cancer stage, phase of care, patient age at diagnosis, and comorbidities before diagnosis. These estimates can serve as baseline data in economic analyses that aim to evaluate interventions for managing cervical cancer.

Traumatic Cervical Spinal Cord Injury and Income and Employment Status.

Importance: Spinal cord injury (SCI) causes drastic changes to an individual's physical health that may be associated with the ability to work. Objective: To estimate the association of SCI with individual earnings and employment status using national administrative health databases linked to income tax data. Design, Setting, and Participants: This was a retrospective, national, population-based cohort study of adults who were hospitalized with cervical SCI in Canada between January 2005 and December 2017. All acute care hospitalizations for SCI of adults ages 18 to 64 years were included. A comparison group was constructed by sampling from individuals in the injured cohort. Fiscal information from their preinjury years was used for comparison. The injured cohort was matched with the comparison group based on age, sex, marital status, province of residence, self-employment status, earnings, and employment status in the year prior to injury. Data were analyzed from August 2022 to January 2023. Main outcomes and Measures: The first outcome was the change in individual annual earnings up to 5 years after injury. The change in mean yearly earnings was assessed using a linear mixed-effects differences-in-differences regression. Income values are reported in 2022 Canadian dollars (CAD $1.00 = US $0.73). The second outcome was the change in employment status up to 5 years after injury. A multivariable probit regression model was used to compare proportions of individuals employed among those who had experienced SCI and the paired comparison group of participants. Results: A total of 1630 patients with SCI (mean [SD] age, 47 [13] years; 1304 male [80.0%]) were matched to patients in a preinjury comparison group (resampled from the same 1630 patients in the SCI group). The mean (SD) of preinjury wage earnings was CAD $46 000 ($48 252). The annual decline in individual earnings was CAD $20 275 (95% CI, -$24 455 to -$16 095) in the first year after injury and CAD $20 348 (95% CI, -$24 710 to -$15 985) in the fifth year after injury. At 5 years after injury, 52% of individuals who had an injury were working compared with 79% individuals in the preinjury comparison group. SCI survivors had a decrease in employment of 17.1 percentage points (95% CI, 14.5 to 19.7 percentage points) in the first year after injury and 17.8 percentage points (14.5 to 21.1 percentage points) in the fifth year after injury. Conclusions and Relevance: In this study, SCI was associated with a decline in earnings and employment up to 5 years after injury for adults aged 18 to 64 years in Canada.

Cost-effectiveness of a gene sequencing test for Alzheimer's disease in Ontario.

Alzheimer's f disease (AD) affects approximately 250,000 Ontarians, a number that is expected to double by 2040. The Ontario Neurodegenerative Disease Research Initiative has developed an in-province genetic test (ONDRISeq), which currently runs in Ontario in an experimental capacity. The aim of this study is to estimate the costs and health outcomes associated with ONDRISeq to diagnose AD relative to out-of-country (OOC) testing (status quo). A cost-utility analysis was developed for a hypothetical cohort of 65-year-olds at risk of AD in Ontario over a 25-year time horizon. Costs and health outcomes (quality-adjusted life years (QALYs)) were assessed from a healthcare payer perspective. Cost-effectiveness was assessed with a $50,000 cost-effectiveness threshold. Probabilistic sensitivity analyses were conducted to evaluate parameter uncertainty. ONDRISeq saved $54 per patient relative to OOC testing and led to a small QALY gain in the base case (0.0014 per patient). Results were most sensitive to testing costs, uptake rates, and treatment efficacy. ONDRISeq represented better value for money relative to OOC testing throughout 75% of 10,000 probabilistic iterations. Using ONDRISeq is expected to provide health system cost savings. Switching to ONDRISeq for AD genetic testing in Ontario would be dependent on the ability to accommodate the expected testing volumes.

The Impact of Dental Care Programs on Individuals and Their Families: A Scoping Review.

BACKGROUND: Despite significant global improvements in oral health, inequities persist. Targeted dental care programs are perceived as a viable approach to both improving oral health and to address inequities. However, the impacts of dental care programs on individual and family oral health outcomes remain unclear. OBJECTIVES: The purpose of this scoping review is to map the evidence on impacts of existing dental programs, specifically on individual and family level outcomes. METHODS: We systematically searched four scientific databases, MEDLINE, EMBASE, CINAHL, and Sociological Abstracts for studies published in the English language between December 1999 and November 2021. Search terms were kept broad to capture a range of programs. Four reviewers (AG, VD, AE, and KKP) independently screened the abstracts and reviewed full-text articles and extracted the data. Cohen's kappa inter-rater reliability score was 0.875, indicating excellent agreement between the reviewers. Data were summarized according to the PRISMA statement. RESULTS: The search yielded 65,887 studies, of which 76 were included in the data synthesis. All but one study assessed various individual-level outcomes (n = 75) and only five investigated family outcomes. The most common program interventions are diagnostic and preventive (n = 35, 46%) care, targeted children (n = 42, 55%), and delivered in school-based settings (n = 28, 37%). The majority of studies (n = 43, 57%) reported a significant improvement in one or more of their reported outcomes; the most assessed outcome was change in dental decay (n = 35). CONCLUSIONS: Dental care programs demonstrated effectiveness in addressing individual oral health outcomes. However, evidence to show the impact on family-related outcomes remains limited and requires attention in future research.

Public preferences for counseling regarding antidepressant use during pregnancy: a discrete choice experiment.

BACKGROUND: Counseling about medication safety during pregnancy is delivered inconsistently. The objectives were to determine public preferences and willingness to pay (WTP) for attributes of counseling regarding antidepressant use during pregnancy. Attributes reflected counseling via a telephone Teratology Information Service (TIS) or a visit to a general practitioner (GP). METHODS: A discrete choice survey was conducted with volunteers recruited from the general public. Stated preferences and WTP for teratology counseling were described by six attributes: training of information provider (IP), method of contact, knowing the IP, confidence in the IP, helpfulness of information, and cost. Interactions of preferences with participant characteristics were examined. RESULTS: Of 175 participants, 85% were women and 91% had some college or university education. All attributes had a significant effect on choice. The most important attribute was the helpfulness of information received (WTP C$59 for very helpful information). Counseling via telephone by a trained specialist was preferred, as in a TIS. It was preferred, however, to speak with a provider known to the user (WTP C$43) which is common in a GP setting. Maximum willingness to pay for very helpful information was less for respondents with less education. Respondents who stated that an antidepressant exposure would make them anxious about the pregnancy were willing to pay more for all attributes. CONCLUSIONS: The results suggest that TIS is the preferred model for counseling regarding to antidepressant use during pregnancy. The public valued information that was helpful and preferred receiving information in nontraditional formats; however, familiarity with the provider was important.

An Electronic Patient-Reported Outcomes Tool for Older Adults With Complex Chronic Conditions: Cost-Utility Analysis.

BACKGROUND: eHealth technologies for self-management can improve quality of life, but little is known about whether the benefits gained outweigh their costs. The electronic patient-reported outcome (ePRO) mobile app and portal system supports patients with multiple chronic conditions to collaborate with primary health care providers to set and monitor health-related goals. OBJECTIVE: This study aims to estimate the cost of ePRO and the cost utility of the ePRO intervention compared with usual care provided to patients with multiple chronic conditions and complex needs living in the community, from the perspective of the publicly funded health care payer in Ontario, Canada. METHODS: We developed a decision tree model to estimate the incremental cost per quality-adjusted life year (QALY) gained for the ePRO tool versus usual care over a time horizon of 15 months. Resource utilization and effectiveness of the ePRO tool were drawn from a randomized clinical trial with 6 family health teams involving 45 participants. Unit costs associated with health care utilization (adjusted to 2020 Canadian dollars) were drawn from literature and publicly available sources. A series of sensitivity analyses were conducted to assess the robustness of the findings. RESULTS: The total cost of the ePRO tool was CAD $79,467 (~US $ 63,581; CAD $1733 [~US $1386] per person). Compared with standard care, the ePRO intervention was associated with higher costs (CAD $1710 [~US $1368]) and fewer QALYs (-0.03). The findings were consistent with the clinical evidence, suggesting no statistical difference in health-related quality of life between ePRO and usual care groups. However, the tool would be considered a cost-effective option if it could improve by at least 0.03 QALYs. The probability that the ePRO is cost-effective was 17.3% at a willingness-to-pay (WTP) threshold of CAD $50,000 (~US $40,000)/QALY. CONCLUSIONS: The ePRO tool is not a cost-effective technology at the commonly used WTP value of CAD $50,000 (~US $40,000)/QALY, but long-term and the societal impacts of ePRO were not included in this analysis. Further research is needed to better understand its impact on long-term outcomes and in real-world settings. The present findings add to the growing evidence about eHealth interventions' capacity to respond to complex aging populations within finite-resourced health systems. TRIAL REGISTRATION: ClinicalTrials.gov NCT02917954; https://clinicaltrials.gov/ct2/show/NCT02917954.

A cost effectiveness analysis of thiopurine methyltransferase testing for guiding 6-mercaptopurine dosing in children with acute lymphoblastic leukemia.

BACKGROUND: An increased understanding of the genetic basis of disease creates a demand for personalized medicine and more genetic testing for diagnosis and treatment. The objective was to assess the incremental cost-effectiveness per life-month gained of thiopurine methyltransferase (TPMT) genotyping to guide doses of 6-mercaptopurine (6-MP) in children with acute lymphoblastic leukemia (ALL) compared to enzymatic testing and standard weight-based dosing. PROCEDURE: A cost-effectiveness analysis was conducted from a health care system perspective comparing costs and consequences over 3 months. Decision analysis was used to evaluate the impact of TPMT tests on preventing myelosuppression and improving survival in ALL patients receiving 6-MP. Direct medical costs included laboratory tests, medications, physician services, pharmacy and inpatient care. Probabilities were derived from published evidence. Survival was measured in life-months. The robustness of the results to variable uncertainty was tested in one-way sensitivity analyses. Probabilistic sensitivity analysis examined the impact of parameter uncertainty and generated confidence intervals around point estimates. RESULTS: Neither of the testing interventions showed a benefit in survival compared to weight-based dosing. Both test strategies were more costly compared to weight-based dosing. Incremental costs per child (95% confidence interval) were $277 ($112, $442) and $298 ($392, $421) for the genotyping and phenotyping strategies, respectively, compared to weight-based dosing. CONCLUSIONS: The present analysis suggests that screening for TPMT mutations using either genotype or enzymatic laboratory tests prior to the administration of 6-MP in pediatric ALL patients is not cost-effective.