Public Payer Research Articles

Canadian public payer best practices for providing timely patient access to cancer therapies.

Canadian public payer best practices for providing timely patient access to cancer therapies.

Telehealth for Equitable Obstetric Care: Addressing Gaps for Patients, Providers, and Payers

In June 2022, President Biden and Vice President Harris released their blueprint for addressing the maternal health crisis, stating their “vision for the future is that the United States will be considered the best country in the world to have a baby.” Currently, it is one of the worst among industrialized countries despite the US spending nearly double the average amount on healthcare per capita. The US is amidst a maternal mortality crisis, particularly for Black and American Indian/Alaska Native pregnant people, with more than 80% of the deaths preventable. Telehealth in obstetrics has the potential to reach pregnant people who are not currently being served by the medical system and to improve rates of severe maternal morbidity and mortality; however, more research is needed to understand and monitor its equity, costs, and optimal usage. Extant research shows that telehealth can produce a small positive effect for certain obstetric health outcomes, but these research findings are rarely stratified by pregnant patients’ demographics. To prevent the perpetuation of existing health inequities, gaps in obstetric telehealth research will need to be addressed. Key knowledge gaps for researchers and policy makers include outcomes, access, satisfaction by patients and providers, potential time savings for patients, and health system cost savings. Im​plementation of equitable obstetric coverage of telehealth services requires clarity from private and public payers for inter-state provisions of care, liability and risk, and service and payment parity.Note: While we use pregnant and postpartum people where possible here to recognize that not all pregnant people identify as women, we occasionally use maternal to reflect terminology used in federal, state, and local data.

EPID-32. A COST ANALYSIS OF BRAIN AND NERVOUS SYSTEM CANCER CARE: AN EXAMINATION OF HEALTHCARE EXPENDITURE TRENDS IN THE UNITED STATES FROM 1996 TO 2016

Abstract INTRODUCTION Management of brain and central nervous system malignancies is associated with substantial healthcare spending, yet estimates of per-patient cost and longitudinal temporal trends remain elusive. In this study, we investigate trends in United States (US) healthcare spending on these diseases between 1996-2016. METHODS We utilize inflation-adjusted national healthcare spending data and age-adjusted prevalence estimates from the Institute for Health Metrics and Evaluation to estimate per-patient US healthcare spending stratified by age and care setting. We employ joinpoint regression to identify temporal trends in healthcare spending, expressed as annual percent change (APC). Drivers of change data from 1996-2013 were evaluated to investigate shifts associated with increased spending. RESULTS In 2016, total healthcare spending attributable to brain and nervous system cancers was $6.85 billion (95% CI, $5.98-7.57 billion), corresponding to $2,245 (95% CI $1,962-$2,480) per patient, with the largest share coming from inpatient care settings ($4.60 billion [95% CI 3.87-5.24 billion]). Private payers shouldered the majority of costs (65.6%) compared to public payers (28.1%) and out-of-pocket expenses (6.4%). The average APC in spending between 1996-2016 was 3.69% [95% CI 3.00%-4.39%], driven primarily by an increase in the price and intensity of inpatient care (11.8% [95% CI 7.7%-16.1%). Further, three statistically significant periods of spending change were identified: 1996-2005 (2.42% [95% CI 2.04-2.80%], p < 0.001 ), 2005-2008 (4.43% [95% CI 1.08%-7.89%], p < 0.0001), 2008-2014 (6.32% [95% CI 5.41%-7.25%], p < 0.001), with spending being relatively stable from 2014-2016. DISCUSSION US expenditures for brain and nervous system malignancies are substantial and have significantly increased between 1996-2016, largely driven by increased inpatient spending. These findings may inform policy efforts to reduce the disease burden while balancing healthcare spending.

Cost-effectiveness analysis of bevacizumab biosimilars for metastatic colorectal cancer: A comparative study using real-world data.

9 Background: MVASI (Amgen) and Zirabev (Pfizer) are two of the earliest bevacizumab biosimilars approved for the first-line treatment of metastatic colorectal cancer (mCRC). While the introduction of biosimilars present an opportunity to alleviate the financial toxicity owing to the escalating costs of novel biologics, biosimilars should be comparatively assessed against the reference biologic in a real-world setting to confirm that they are indeed cost-saving or cost-effective after implementation. In this study, we aimed to confirm and quantify the real-world cost-savings of MVASI and Zirabev relative to originator bevacizumab (Avastin) for patients with mCRC. Methods: We conducted a population-based, retrospective cohort study in Ontario, Canada, where originator and biosimilar bevacizumab are universally publicly funded. Capturing the entire population of Ontario, we assessed all mCRC patients who received originator bevacizumab between January 1, 2008, and August 11, 2019, or biosimilar bevacizumab between August 12, 2019, and March 31, 2021. Biosimilar cases and originator bevacizumab controls were matched 1:4 using propensity score methods to adjust for differences at baseline. We calculated 1-year total patient-level costs (in Canadian dollars) and effects (in life years and quality-adjusted life years (QALY)) from the public health payer’s perspective. The primary outcomes for estimating cost-effectiveness were incremental net monetary benefit (INMB) and incremental net health benefit (INHB), calculated at willingness-to-pay (WTP) thresholds ranging from $50,000-200,000 per life year gained. Sensitivity analyses included a subgroup analysis by biosimilar type (MVASI/Zirabev) and an analysis using a 2-year time horizon. Results: The final propensity score matched cohort included 747 biosimilar cases and 2,945 controls. Bevacizumab biosimilars were associated with an incremental cost of -$6,379 (95% confidence interval (CI): -9,417, -3,537) (i.e., cost-saving) and an incremental effect of 0.0 (95% CI: -0.02, 0.02) life years gained and -0.01 (95% CI: -0.03, 0) QALY gained. INMB and INHB estimates indicated that biosimilar bevacizumab is cost-effective at all WTP thresholds assessed, with results remaining consistent across our biosimilar type subgroups and 2-year sensitivity analyses. Conclusions: Bevacizumab biosimilars demonstrated real-world cost-savings while providing similar survival benefit as originator bevacizumab, confirming the initial expectations of their implementation.

Pembrolizumab vs chemotherapy in the first-line setting of PD-L1≥50% metastatic non-small cell lung cancer: A real-world cost-effectiveness analysis.

21 Background: Despite the voiced concerns around the initial economic decision models for first-line pembrolizumab in PD-L1≥50% metastatic non-small cell lung cancer (mNSCLC), there has been a paucity of evidence on the anticipated cost-effectiveness of pembrolizumab in the real world. To resolve the uncertainties associated with the initial assessment, we evaluated the real-world cost-effectiveness of first line pembrolizumab versus platinum-based chemotherapy for mNSCLC patients with PD-L1≥50%. Methods: We retrospectively identified a population-based cohort of mNSCLC patients who received first-line pembrolizumab or platinum-based chemotherapy between April 1, 2013, and March 31, 2021, in Ontario, Canada. Employing a public payer’s perspective, all costs (in Canadian dollars) and effects were estimated over a 4-year horizon, adjusted for censoring and discounted at 1.5% yearly. The primary outcomes were incremental cost-effectiveness ratios (ICERs) for life-years gained (LYG) and quality-adjusted life year (QALY). To examine the sensitivity of the ICER to drug acquisition costs and discounting, we conducted a price reduction analysis and a scenario analysis of different discount rates. Results: Propensity-score matching resulted in a total of 1,142 pairs of matched mNSCLC patients. Pembrolizumab extended survival with an incremental effect of 0.37 LYG and 0.35 QALY, but at an incremental cost of $56,681; the resulting ICERs were $154,941/LYG and $163,039/QALY. Though the ICERs were not sensitive to discounting rate, 31% and 55% reductions to the price of pembrolizumab brought the ICER below $100,000/QALY and $50,000/QALY, respectively. Conclusions: In the real world, first-line pembrolizumab is not considered to be cost-effective for mNSCLC patients with PD-L1≥50%. Improvements in cost-effectiveness, however, may be achievable through price renegotiations for pembrolizumab.

Budget impact of introducing oral semaglutide to the public healthcare benefit package in Saudi Arabia

BackgroundThe Kingdom of Saudi Arabia (KSA) has embarked on a Health Sector Transformation Program as part of the Kingdom’s Vision 2030 initiatives with the facilitation of access to healthcare services for the millions in KSA with diabetes an essential part of the Program. Decision-making tools, such as budget impact models, are required to consider the addition of new medications like oral semaglutide that have multifaceted health benefits and address barriers related to therapeutic inertia to reduce diabetes-related complications.ObjectiveTo determine the financial impact of the introduction of oral semaglutide as a treatment option for people with type 2 diabetes mellitus (T2DM) in KSA.MethodsFrom the public payer’s perspective, the budget impact model estimates the costs before and after the introduction of oral semaglutide over a 5-year time horizon. The budget impact of introducing oral semaglutide (primary comparator) compared with three different classes of diabetes medicines: glucagon-like peptide-1 receptor agonists (GLP-1), sodium-glucose transport protein 2 inhibitors (SGLT 2i) and dipeptidyl peptidase 4 inhibitors (DDP-4i) have been calculated based on the projected market shares. The model includes the cost of care through the incorporation of health outcomes that have an impact on the national payer’s budget in Saudi Riyals (SAR).ResultsThe budget impact over the five-year time horizon indicates a medication cost increase (17,424,788 SAR), and cost offsets which include a difference in diabetes management costs (-3,625,287 SAR), CV complications costs (-810,733 SAR) and weight loss savings of 453,936 SAR. The cumulative total cost difference is 12,427,858 SAR (0.66%).ConclusionThe introduction of oral semaglutide 14 mg as a second-line treatment option after metformin is indicated as budget-neutral to slightly budget-inflating for the public pharmaceutical formulary of KSA. The price difference is offset by positive health outcomes and costs. This conclusion was confirmed through a probabilistic sensitivity analysis.

Attributable Costs of Stroke in Ontario, Canada and Their Variation by Stroke Type and Social Determinants of Health.

Estimates of attributable costs of stroke are scarce, as most prior studies do not account for the baseline health care costs in people at risk of stroke. We estimated the attributable costs of stroke in a universal health care setting and their variation across stroke types and several social determinants of health. We undertook a population-based administrative database-derived matched retrospective cohort study in Ontario, Canada. Community-dwelling adults aged ≥40 years with a stroke between 2003 and 2018 were matched (1:1) on demographics and comorbidities with controls without stroke. Using a difference-in-differences approach, we estimated the mean 1-year direct health care costs attributable to stroke from a public health care payer perspective, accounting for censoring with a weighted available sample estimator. We described health sector-specific costs and reported variation across stroke type and social determinants of health. The mean 1-year attributable costs of stroke were Canadian dollars 33 522 (95% CI, $33 231-$33 813), with higher costs for intracerebral hemorrhage ($40 244; $39 193-$41 294) than ischemic stroke ($32 547; $32 252-$32 843). Most of these costs were incurred in acute care hospitals ($15 693) and rehabilitation facilities ($7215). Compared with all patients with stroke, the mean attributable costs were higher among immigrants ($40 554; $39 316-$41 793), those aged <65 years ($35 175; $34 533-$35 818), and those residing in low-income neighborhoods ($34 687; $34 054-$35 320) and lower among rural residents ($29 047; $28 362-$29 731). Our findings of high attributable costs of stroke, especially in immigrants, younger patients, and residents of low-income neighborhoods, can be used to evaluate potential health care cost savings associated with different primary stroke prevention strategies.

Cost-effectiveness of left atrial appendage closure with Watchman for non-valvular atrial fibrillation patients in Japan

AimsLeft atrial appendage closure (LAAC) has been demonstrated to be cost-saving relative to oral anticoagulants for stroke prophylaxis in patients with non-valvular atrial fibrillation (NVAF) in the United States and Europe. This study assessed the cost-effectiveness of LAAC with the Watchman device relative to warfarin and direct oral anticoagulants (DOACs) for stroke risk reduction in NVAF from a Japanese public healthcare payer perspective.MethodsA Markov model was developed with 70-year-old patients using a lifetime time horizon. LAAC clinical inputs were from pooled, 5-year PROTECT AF and PREVAIL trials; warfarin and DOAC inputs were from published meta-analyses. Baseline stroke and bleeding risks were from the SALUTE trial on LAAC. Cost inputs were from the Japanese Medical Data Vision database. Probabilistic and one-way sensitivity analyses were performed.ResultsOver the lifetime time horizon, LAAC was less costly than warfarin (savings of JPY 1,878,335, equivalent to US $17,600) and DOACs (savings of JPY 1,198,096, equivalent to US $11,226). LAAC also provided 1.500 more incremental quality-adjusted life years (QALYs) than warfarin and 0.996 more than DOACs. In probabilistic sensitivity analysis, LAAC was cost-effective relative to warfarin and DOACs in 99.98% and 99.73% of simulations, respectively. LAAC dominated (had higher cumulative QALYs and was less costly than) warfarin and DOACs in 89.94% and 83.35% of simulations, respectively.ConclusionsOver a lifetime time horizon, LAAC is cost-saving relative to warfarin and DOACs for stroke risk reduction in NVAF patients in Japan and is associated with improved quality-of-life.

The PRIDx framework to engage payers in reducing diagnostic errors in healthcare.

No framework currently exists to guide how payers and providers can collaboratively develop and implement incentives to improve diagnostic safety. We conducted a literature review and interviews with subject matter experts to develop a multi-component 'Payer Relationships for Improving Diagnoses (PRIDx)' framework, that could be used to engage payers in diagnostic safety efforts. The PRIDx framework, 1) conceptualizes diagnostic safety links to care provision, 2) illustrates ways to promote payer and provider engagement in the design and adoption of accountability mechanisms, and 3) explicates the use of data analytics. Certain approaches suggested by PRIDx were refined by subject matter expert interviewee perspectives. The PRIDx framework can catalyze public and private payers to take specific actions to improve diagnostic safety. Implementation of the PRIDx framework requires new types of partnerships, including external support from public and private payer organizations, and requires creation of strong provider incentives without undermining providers' sense of professionalism and autonomy. PRIDx could help facilitate collaborative payer-provider approaches to improve diagnostic safety and generate research concepts, policy ideas, and potential innovations for engaging payers in diagnostic safety improvement activities.

The economic impact of obesity in Kuwait: a micro-costing study evaluating the burden of obesity-related comorbidities

Objective44% of Kuwait’s population live with obesity and the health consequences place a significant burden on the public health system. This study provides an assessment of the cost burden of obesity-related comorbidities (ORC).MethodsA retrospective micro-costing analysis was conducted to quantify the direct cost associated with ORCs. ORCs and their cost categories were informed by a systematic literature review and validated by a local steering committee comprising three experts. Seventy public sector clinicians and eight hospital procurement staff were surveyed to provide healthcare resource utilization estimates and medical resource cost data, respectively. The annual cost of each ORC and the cost drivers were also validated by the steering committee.ResultsIndividuals in Kuwait with any single ORC incurred direct healthcare costs ranging 1,748–4,205 KWD annually. Asthma, chronic kidney disease and type 2 diabetes were the costliest ORCs, incurring an annual cost that exceeds 3,500 KWD per patient. Hypertension, angina and atrial fibrillation were the least costly ORCs. In general, costs were driven by drug costs and resources allocated to address treatment-related adverse events.LimitationsIn the absence of an official patient registry in Kuwait, our study provides a conservative estimate of direct costs derived from a nationwide survey. Additionally, the cost estimates in this study assumes that a patient with obesity will only experience one ORC. In reality, multi-morbid states may incur additional costs that are not currently captured.ConclusionsOur study confirms that ORCs generate a significant financial burden to the public payer. The study provides an economic case for policymakers to recognize the exigency for obesity prevention and control in accordance with the ORC prevalence, and the need for sustainable investments towards body-mass index management to prevent individuals from developing multiple comorbidities.

Health and economic outcomes of 20-valent pneumococcal conjugate vaccine compared to 15-valent pneumococcal conjugate vaccine strategies for adults in Greece.

Higher valency pneumococcal conjugate vaccines (PCVs) are expected to improve protection against pneumococcal disease through coverage of additional serotypes. The aim of the present study was to evaluate the cost-effectiveness of 20-valent pneumococcal conjugate vaccine (PCV20) compared to 15-valent pneumococcal conjugate vaccine (PCV15) alone or followed by 23-valent polysaccharide vaccine (PPV23) for adults in Greece. A published Markov model was adapted to simulate lifetime risk of clinical and economic outcomes from the public payer's perspective. The model population was stratified based on age and risk profile (i.e., low, moderate, or high-risk of developing pneumococcal disease). Epidemiologic parameters, serotype coverage and vaccines' effectiveness were based on published literature, while direct medical costs (prices €, 2022) were obtained from official sources. Main model outcomes were projected number of invasive pneumococcal disease (IPD) and all-cause non-bacteremic pneumonia (NBP) cases and attributable deaths, costs and quality-adjusted life-years (QALY) for each vaccination strategy. Sensitivity analyses were performed to ascertain the robustness of model results. Over the modeled time horizon, vaccination with PCV20 compared to PCV15 alone or PCV15 followed by PPV23 prevents an additional 747 and 646 cases of IPD, 10,334 and 10,342 cases of NBP and 468 and 455 deaths respectively, resulting in incremental gain of 1,594 and 1,536 QALYs and cost savings of €11,183 and €48,858, respectively. PSA revealed that the probability of PCV20 being cost-effective at the predetermined threshold of €34,000 per QALY gained was 100% compared to either PCV15 alone or the combination of PCV15 followed by PPV23. PCV20 is estimated to improve public health by averting additional pneumococcal disease cases and deaths relative to PCV15 alone or followed by PPV23, and therefore translates to cost-savings for the public payer. Overall results showed that vaccination with PCV20 was estimated to be a dominant vaccination strategy (improved health outcomes with reduced costs) over PCV15 alone or followed by PPV23 for prevention of pneumococcal disease in adults in Greece.

Health economic analysis of the integrated cognitive assessment tool to aid dementia diagnosis in the United Kingdom.

The aim of this study was to develop a comprehensive economic evaluation of the integrated cognitive assessment (ICA) tool compared with standard cognitive tests when used for dementia screening in primary care and for initial patient triage in memory clinics. ICA was compared with standard of care comprising a mixture of cognitive assessment tools over a lifetime horizon and employing the UK health and social care perspective. The model combined a decision tree to capture the initial outcomes of the cognitive testing with a Markov structure that estimated long-term outcomes of people with dementia. Quality of life outcomes were quantified using quality-adjusted life years (QALYs), and the economic benefits were assessed using net monetary benefit (NMB). Both costs and QALYs were discounted at 3.5% per annum and cost-effectiveness was assessed using a threshold of £20,000 per QALY gained. ICA dominated standard cognitive assessment tools in both the primary care and memory clinic settings. Introduction of the ICA tool was estimated to result in a lifetime cost saving of approximately £123 and £226 per person in primary care and memory clinics, respectively. QALY gains associated with early diagnosis were modest (0.0016 in primary care and 0.0027 in memory clinic). The net monetary benefit (NMB) of ICA introduction was estimated at £154 in primary care and £281 in the memory clinic settings. Introduction of ICA as a tool to screen primary care patients for dementia and perform initial triage in memory clinics could be cost saving to the UK public health and social care payer.

Cost-effectiveness of treatment intervention in prediabetic patients in Bulgaria

Introduction: The study aims to evaluate the cost-effectiveness (CE) of an early intervention in prediabetes (metformin) in order to prevent or slow down the onset of diabetes in those at high risk compared with the current “do nothing” approach. Materials and methods: An Excel-based model was developed. The results of the CE and cost-utility analyses are presented as an ICER (incremental cost-effectiveness ratio) and ICUR (incremental cost-utility ratio), respectively. Markov model of the cost or potential savings from the perspective of the National Health Insurance Fund in Bulgaria was performed. Results: The ICER of the metformin intervention in prediabetes patients compared with “do nothing” routine shows that metformin treatment produced more health benefits (number of prevented diabetes cases) on a lower cost for the public payer. The ICER calculated is -7,122.32 BGN per number of prevented diabetes cases and it confirms cost-savings are possible when metformin is applied compared with the “do nothing” approach. The ICUR per quality-adjusted life years (QALYs) gained also shows the metformin preventive intervention as a dominant and cost-saving alternative. The Markov model simulation confirms the intervention with metformin is less costly in a long-term and leads to higher QALYs. Conclusion: The investment in a preventive intervention with metformin offers an excellent value for money. The ICER of the metformin intervention in prediabetes patients compared with “do nothing” routine shows that metformin preventive intervention produced more health benefits on a lower cost for the public payer in Bulgaria.

Cost-effectiveness of combination therapy of polatuzumab vedotin plus rituximab, cyclophosphamide, doxorubicin, and prednisolone for previously untreated diffuse large B-cell lymphoma in Japan

AimThe POLARIX trial showed that Pola + R-CHP (polatuzumab vedotin plus rituximab, cyclophosphamide, doxorubicin, and prednisolone) prolongs progression-free survival (PFS) in patients with previously untreated diffuse large B-cell lymphoma (DLBCL) compared with R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisolone), the conventional standard of care, with a similar safety profile. However, Pola + R-CHP has not been evaluated from the viewpoint of health economics in Japan. This study evaluates the cost-effectiveness of Pola + R-CHP for previously untreated DLBCL from a Japanese public healthcare payer’s perspective.MethodsA partitioned survival analysis model was constructed to estimate lifetime costs and effectiveness of Pola + R-CHP and R-CHOP in previously untreated DLBCL who had an International Prognostic Index score (IPI) score of ≥2. A parametric survival model was applied to data analyzed in the POLARIX trial to estimate the lifetime overall survival (OS) and PFS for each treatment. The parameters required for the model were based on the results of a literature search and expert opinion.ResultsThe incremental cost-effectiveness ratio (ICER) of Pola + R-CHP vs. R-CHOP was JPY2,710,238 per quality-adjusted life year (QALY), less than the ICER of JPY7.5 million per QALY that is considered to be cost-effective based on the threshold of the Japanese cost-effectiveness evaluation system. One-way sensitivity analysis showed that the parameters influencing the results of the analysis were median PFS and the total cost per regimen of salvage chemotherapy, patient weight, and patient age. Probabilistic sensitivity analysis showed that the probability of Pola + R-CHP having superior cost-effectiveness was 99.2% when the reference value was JPY7.5 million. The results of scenario analysis suggested that prolongation of PFS was an important factor in the evaluation of cost-effectiveness in previously untreated DLBCL with or without prolongation of OS.ConclusionsThis study suggests that Pola + R-CHP is a cost-effective treatment for previously untreated DLBCL in Japan under the public health insurance system.

Providing doula support to publicly insured women in central Texas: A financial cost-benefit analysis.

Disparities in birth outcomes continue to exist in the United States, particularly for low-income, publicly insured women. Doula support has been shown to be a cost-effective intervention in predominantly middle-to-upper income White populations, and across all publicly insured women at the state level. This analysis extends previous studies by providing an estimate of benefits that incorporates variations in averted outcomes by race and ethnicity in the context of one region in Texas. The objectives of this study were to determine (1) whether the financial value of benefits provided by doula support exceeds the costs of delivering it; (2) whether the cost-benefit ratio differs by race and ethnicity; and (3) how different doula reimbursement levels affect the cost-benefit results with respect to pregnant people covered by Medicaid in central Texas. We conducted a forward-looking cost-benefit analysis using secondary data carried out over a short-term time horizon taking a public payer perspective. We focused on a narrow set of health outcomes (preterm delivery and cesarean delivery) that was relatively straightforward to monetize. The current, usual care state was used as the comparison condition. Providing pregnant people covered by Texas Medicaid with access to doulas during their pregnancies was cost-beneficial (benefit-to-cost ratio: 1.15) in the base model, and 65.7% of the time in probabilistic sensitivity analyses covering a feasible range of parameters. The intervention is most cost-beneficial for Black women. Reimbursing doulas at $869 per client or more yielded costs that were greater than benefits, holding other parameters constant. Expanding Medicaid pregnancy-related coverage to include doula services would be cost-beneficial and improve health equity in Texas.

Why it is so challenging to perform economic evaluations of interventions in autism and what to do about it.

Economic evaluation is used to determine the optimal provision of services and programs under budget constraints and to inform public and private payer funding decisions. To maximize value-for-money in the design and delivery of programs and services for persons with autism spectrum disorder (ASD), it's essential to generate high-quality economic evidence to inform budget allocation. There is a paucity however, of economic evaluations of interventions for ASD. This is due in part to challenges in conducting economic evaluations in this population and the lack of guidance on suitable approaches. These challenges are related to the inherent heterogeneity of the autistic population; establishing short- and long-term effectiveness; measurement of costs and the availability of valid instruments for collecting economic data; the appropriateness of outcomes for use in economic evaluation; and achieving statistical power. This commentary addresses a lack of awareness and needed guidance on these issues by discussing the challenges and providing recommendations for how economic evaluations in ASD could be improved to generate high-quality evidence for program funding decision-making.

Cost-effectiveness analysis of cabozantinib compared with everolimus, axitinib, and nivolumab in subsequent line advanced renal cell carcinoma in Japan

AimsThe treatment landscape of renal cell carcinoma has changed with the introduction of targeted therapies. While the clinical benefit of cabozantinib is well-established for Japanese patients who have received prior treatment, the economic benefit remains unclear. The objective of this study was to assess the cost-effectiveness of cabozantinib compared with everolimus, axitinib, and nivolumab in patients with advanced renal cell carcinoma who have failed at least one prior therapy in Japan.MethodsA cost-effectiveness model was developed using a partitioned survival approach and a public healthcare payer’s perspective. Over a lifetime horizon, clinical and economic implications were estimated according to a three-health–state structure: progression-free, post-progression, and death. Key clinical inputs and utilities were derived from the METEOR trial, and a de novo network meta-analysis and cost data were obtained from publicly available Japanese data sources. Costs, quality-adjusted life-years, and incremental cost-effectiveness ratios were estimated. Costs and health benefits were discounted annually at 2%.ResultsCabozantinib was more costly and effective compared with everolimus and axitinib, with deterministic incremental cost-effectiveness ratios of ¥5,375,559 and ¥2,223,138, respectively. Compared to nivolumab, cabozantinib was predicted to be less costly and more effective. Sensitivity and scenario analyses demonstrated that the key drivers of cost-effectiveness results were the estimation of overall survival and treatment duration, relative efficacy, drug costs, and subsequent treatment costs.LimitationsMETEOR was an international trial but did not enroll any patients from Japan. Efficacy and safety data from METEOR were used as a proxy for the Japanese population following validation by clinical experts, and alternative assumptions specific to clinical practice in Japan were evaluated in scenario analyses.ConclusionsIn Japan, cabozantinib is a cost-effective alternative to everolimus, axitinib, and nivolumab for the treatment of patients with advanced renal cell carcinoma who have received at least one prior line of therapy.

Costs of plasmocytic myeloma therapy in the drug programme at a Regional Oncology Centre in Poland.

The rapidly growing market for drugs, including oncology and haemato-oncology drugs, is generating enormous financial expenditure for healthcare systems. In Poland, access to high-cost treatments is possible mainly within drug programmes, funded by public healthcare systems. The path of proceeding adopted in Polish regulations is similar to the solutions adopted in other countries. The aim of this study was to demonstrate the actual costs incurred by the treatment entity in the process of treating patients under the drug programme at the Regional Oncology Centre in Olsztyn, north-east Poland. The oncology drug programme B.54 'Treatment of patients with refractory or malignant plasmocytic myeloma' implemented at the Regional Oncology Centre in Poland between 2018-2021, was selected for the analysis. The choice of the B.54 programme was based on the small population of patients meeting the inclusion criteria for this programme, and the large number of diagnostic procedures stipulated in the drug programme description. On average, 25 patients were treated per year. The financial analysis used the financial categories related to hospital billing information. The costs were presented based on the purchasing power parity of money in 2021, i.e. 1 USD-inter is equivalent to 1.837 PLN. The flat rate form of financing medical services does not cover the actual costs of treatment. Providing patients with necessary medical services without their full coverage by the public payer, burdens the budget of the centre and may lead to indebtedness of the treatment entity. Without an increase in the valuation of benefits under drug programmes, corresponding to the actual costs of treatment, the expected increase in access to innovative therapies will be difficult to accomplish.

Protocol for a Prospective, Observational Cost-effectiveness Analysis of Returning Secondary Findings of Genome Sequencing for Unexplained Suspected Genetic Conditions

PurposeAlthough costly, genome-wide sequencing (GWS) detects an extensive range of variants, enhancing our ability to diagnose and assess risk for an increasing number of diseases. In addition to detecting variants related to the indication for testing, GWS can detect secondary variants in BRCA1, BRCA2, and other genes for which early intervention may improve health. As the list of secondary findings grows, there is increased demand for surveillance and management by multiple specialists, adding pressure to constrained health care budgets. Secondary finding testing is actively debated because some consider it opportunistic screening for future health risks that may not manifest. Given the economic implications of secondary finding testing and follow-up and its unproven clinical utility, the objective is to assess the incremental cost-effectiveness of secondary finding ascertainment per case detected and per unit of improved clinical utility in families of children with unexplained suspected genetic conditions undergoing clinical GWS. MethodsThose undergoing trio genome or exome sequencing are eligible for the study. Positive secondary finding index cases will be matched to negative controls (1:2) based on age group, primary result(s) type, and clinical indication. During the 2-year study, 71 cases and 142 matched controls are expected. Health service use will be collected in patients and 1 adult family member every 6 months. The per-child and per-dyad total cost will be determined by multiplying use of each resource by a corresponding unit price and summing all cost items. Costs will be estimated from the public and societal payer perspectives. The mean cost per child and per dyad for secondary finding–positive and secondary finding–negative groups will be compared statistically. If important demographic differences are observed between groups, ordinary least-squares regression, log transformation, or other nonparametric technique will be used to compare adjusted mean costs. The ratio of the difference in mean cost to the secondary finding yield will be used to estimate incremental cost-effectiveness. In secondary analyses, effectiveness will be estimated using the number of clinical management changes due to secondary findings or the Clinician-Reported Genetic Testing Utility Index (C-GUIDE) score, a validated measure of clinical utility. Sensitivity analysis will be undertaken to assess the robustness of the findings to variation in key parameters. ImplicationsThis study generates key evidence to inform clinical practice and funding allocation related to secondary finding testing. The inclusion of family members and a new measure of clinical utility represent important advancements in economic evaluation in genomics.

Cost-effectiveness analysis of 7 treatments in metastatic hormone-sensitive prostate cancer: a public-payer perspective.

Recently, several new treatment regimens have been approved for treating metastatic hormone-sensitive prostate cancer, building on androgen deprivation therapy alone. These include docetaxel androgen deprivation therapy, abiraterone acetate-prednisone androgen deprivation therapy, apalutamide androgen deprivation therapy, enzalutamide androgen deprivation therapy, darolutamide-docetaxel androgen deprivation therapy, and abiraterone-prednisone androgen deprivation therapy with docetaxel. There are no validated predictive biomarkers for choosing a specific regimen. The goal of this study was to conduct a health economic outcome evaluation to determine the optimal treatment from the US public sector (Veterans Affairs). We developed a partitioned survival model in which metastatic hormone-sensitive prostate cancer patients transitioned between 3 health states (progression free, progressive disease to castrate resistance state, and death) at monthly intervals based on Weibull survival model estimated from published Kaplan-Meier curves using a Bayesian network meta-analysis of 7 clinical trials (7208 patients). The effectiveness outcome in our model was quality-adjusted life-years (QALYs). Cost input parameters included initial and subsequent treatment costs and costs for terminal care and for managing grade 3 or higher drug-related adverse events and were obtained from the Federal Supply Schedule and published literature. Average 10-year costs ranged from $34 349 (androgen deprivation therapy) to $658 928 (darolutamide-docetaxel androgen deprivation therapy) and mean QALYs ranged from 3.25 (androgen deprivation therapy) to 4.57 (enzalutamide androgen deprivation therapy). Treatment strategies docetaxel androgen deprivation therapy, enzalutamide androgen deprivation therapy docetaxel, apalutamide androgen deprivation therapy, and darolutamide-docetaxel androgen deprivation therapy were eliminated because of dominance (ie, they were more costly and less effective than other strategies). Of the remaining strategies, abiraterone acetate-prednisone androgen deprivation therapy was the most cost-effective strategy at a willingness-to-pay threshold of $100 000/QALY (incremental cost-effectiveness ratios = $21 247/QALY). Our simulation model found abiraterone acetate-prednisone androgen deprivation therapy to be an optimal first-line treatment for metastatic hormone-sensitive prostate cancer from a public (Veterans Affairs) payer perspective.