Healthcare Payer Perspective Research Articles

Cost-effectiveness analysis of denosumab versus alendronate for improving bone mineral density in renal transplant recipients: a comparative study

BackgroundDenosumab and alendronate had a positive impact on bone mineral density (BMD) preservation after kidney transplantation. However, the cost-effectiveness of these agents in the context of kidney transplantation remains largely unexplored. We have conducted a cost-effectiveness analysis to compare the cost and the clinical outcomes of adding subcutaneous (SC) 60 mg denosumab every 6 months vs. oral weekly 70 mg Alendronate, to the standard care therapy (vitamin D and calcium) in renal transplant recipients (RTRs). The impact of both drugs on BMD t-score improvement and fracture prevention was investigated. A decision-analysis model from a health care payer perspective was applied.ResultsThe cost-effectiveness analysis has shown that alendronate add-on to the standard therapy was the most cost-effective regimen, in terms of BMD improvement and fracture prevention with an incremental cost-effectiveness ratio (ICER) of $154/patient/year. The one-way sensitivity analyses have delineated the change in cost-effectiveness when alendronate retail unit price was increased by 25% and 50%, or when denosumab retail unit price was decreased by 25% and 50%. The model was sensitive to the uncertainties (95% confidence interval) in the probabilities of fracture prevention and the probabilities of attaining the desired outcome.ConclusionThe model suggests that oral once weekly alendronate add-on regimen to standard therapy seems to be substantially more cost effective than twice yearly SC denosumab in terms of BMD improvement and fracture prevention in RTRs. Longer time horizon models with longer follow-up periods for fracture risks and adverse events are warranted to validate these data.

Cost-utility of nelarabine for the first-line treatment of newly diagnosed pediatric T-cell acute lymphoblastic leukemia in Canada.

The Children's Oncology Group (COG)-AALL0434 trial investigated the addition of nelarabine to the augmented Berlin-Frankfurt-Münster (aBFM) protocol in patients (1.0-30.99years) with newly diagnosed T-cell acute lymphoblastic leukemia (T-ALL). Despite demonstrating superior outcomes, nelarabine is not currently funded by many health systems, in part due to a lack of cost-effectiveness data. We estimated the cost-utility of nelarabine for this indication from a Canadian public healthcare payer perspective. We developed a microsimulation model that followed hypothetical patients with newly diagnosed T-ALL from post-induction therapy to death. Three health states were modeled: relapse-free, post-relapse, and death. Efficacy was estimated using AALL0434 and retrospective data from Ontario, Canada. Costs were obtained from Canadian sources. Utility estimates and long-term mortality risks were sourced from literature. Total healthcare costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER) were reported. Probabilistic and scenario analyses were conducted. Incorporating nelarabine in the aBFM protocol increased costs by $51,670 Canadian dollars per patient, but resulted in 1.97 more QALYs and an ICER of $26,184/QALY. Most of the identified cost and benefit were accrued within the AALL0434 trial period (first 11years post diagnosis) and while patients were in the relapse-free health state. Across multiple scenarios, the ICER was stable under an assumed $50,000/QALY threshold. Incorporating nelarabine into aBFM was cost-effective across different scenarios and assumptions. These results support its funding by public and private payers.

Cost-effectiveness of prophylactic ramosetron in the prevention of postoperative nausea and vomiting.

This study was conducted to assess the cost-effectiveness of prophylactic use of ramosetron compared to no antiemetic medications for the prevention of postoperative nausea and vomiting (PONV) from the healthcare payer and societal perspectives in South Korea. A decision analytic model was constructed to assess the cost-effectiveness of prophylactic ramosetron use versus no antiemetic therapy at 24-hour and 48-hour periods post-surgery over a 5-day duration. The model was populated using costs and utility parameters from published studies as well as from surveys of an expert panel of physicians using structured questionnaires. The cost parameters included the costs of drugs, treatment, patient time, productivity loss, and transportation. Effectiveness was measured using quality adjusted life years (QALYs). The study outcome was the incremental cost-effectiveness ratio (ICER). The parameter uncertainties were addressed using deterministic and probabilistic scenario analyses. The base-case analysis showed that, on average, patients treated with prophylactic ramosetron had lower costs from both the healthcare payer (US$16.88 vs US$17.33) and societal (US$16.89 vs US$18.72) perspectives and higher QALYs (0.0121 vs 0.0114) over the 5-day study duration compared to patients without any antiemetic medications. Deterministic and probabilistic sensitivity analyses were conducted to examine the robustness of results for the parameters included in the model. The acceptability curve probability showed that treating patients with ramosetron compared to no antiemetic medications was more than 99% cost-effective at a willingness-to pay threshold of US$5,000/QALY from both payer and societal perspectives. The results demonstrated that prophylactic use of ramosetron compared to no antiemetic therapy is highly cost-effective to prevent PONV for patients undergoing surgery from both healthcare payer and societal perspectives. The cost effectiveness is the result of the decrease in the incidence of PONV and the direct treatment costs of severe PONV with improved patient quality of life.

Cost-Effectiveness of Teduglutide for Pediatric Patients with Short Bowel Syndrome in Japan, Including Caregiver Burden.

Short bowel syndrome (SBS) is associated with a significant mental and physical burden for patients and caregivers. Standard of care (SOC) for SBS includes parenteral support (PS) to optimize intestinal function. Teduglutide, a recombinant human glucagon-like peptide2 analogue, reduces the need for PS in patients with SBS. In this study, we assessed the cost-effectiveness of teduglutide in pediatric patients with SBS from multiple perspectives, considering the caregiver's burden. A Markov model was used to evaluate cost (Japanese yen, JPY) and effectiveness (quality-adjusted life years, QALYs) of teduglutide compared with SOC for pediatric patients with SBS in Japan. We conducted a base-case analysis and selected sensitivity and scenario analyses from three perspectives: (1) the public healthcare payer, (2) the public healthcare and long-term care payer, and (3) society. In the base-case analysis, the incremental cost-effectiveness ratio (ICER) was 9,533,412 JPY per QALY from the public healthcare payer perspective, 6,335,980 JPY per QALY from the public healthcare and long-term care payer perspective, and 3,510,371 JPY per QALY from the societal perspective. The probability that cost-effectiveness of teduglutide is favorable from a societal perspective was 59.3%. In all scenario analyses, consistent with the base-case analysis, ICERs for teduglutide compared with SOC were different depending on whether caregiver utility and productivity loss were considered. Incorporating the caregiver's burden in the cost-effectiveness analysis of teduglutide for pediatric patients with SBS provided a more comprehensive assessment of the value of teduglutide for patients, their families, and society. This approach enhances our understanding of the overall value of a treatment, especially for diseases with significant caregiver burden.

Cost Effectiveness of Sequencing Vedolizumab as First-Line Biologic in Ulcerative Colitis and Crohn's Disease in Canada: An Analysis Using Real-World Evidence from the EVOLVE Study.

Vedolizumab is a gut-selective anti-lymphocyte trafficking biologic indicated for the treatment of adult patients with moderately to severely active ulcerative colitis (UC) and Crohn's disease (CD) in Canada. The objective of this study was to evaluate the cost effectiveness of treatment sequencing for UC and CD from a public healthcare payer perspective, leveraging new real-world evidence from the literature and the EVOLVE study, a retrospective chart review. Using separate decision tree/Markov models to assess cost effectiveness for UC and CD, two sequencing approaches were estimated for adult patients (≥ 18years) diagnosed with UC or CD who were biologic-naïve: vedolizumab as first-line biologic followed by anti-tumor necrosis factor (TNF)-α versus first-line anti-TNFα followed by vedolizumab. Treatment effectiveness (response and remission), surgery rates, dose escalation and regain of response and safety inputs were estimated from EVOLVE, a retrospective chart review of real-world data, and evidence synthesis from the literature, whereas costs and utilities were estimated from health technology assessment reports, clinical trials, and the literature. Biosimilar costs were used for anti-TNFα. Both models simulated a 5-year time horizon and discounted costs and outcomes at 1.5%. Probabilistic base-case analyses (n=10,000) reported total costs (2023 Canadian dollars) and quality-adjusted life-years (QALYs). Several scenario analyses were conducted to explore robustness of results. In UC, vedolizumab as a first-line biologic followed by anti-TNFα resulted in an incremental gain of 0.09 QALYs (2.46 vs. 2.55) and saved $7179 ($134,028 vs. $126,848), making this a dominant strategy compared with first-line anti-TNFα followed by vedolizumab. In CD, use of vedolizumab as a first-line biologic resulted in an incremental gain of 0.04 QALYs (3.35 vs. 3.39) at an incremental cost of $50,631 ($89,850 vs. $140,381) versus first-line anti-TNFα followed by vedolizumab (incremental cost-effectiveness ratio of $1,265,775 per QALY). Based on this analysis, sequencing vedolizumab as a first-line biologic prior to anti-TNFα in UC and CD provided additional clinical benefit to patients. In UC, vedolizumab as a first-line biologic also saved healthcare system costs compared with anti-TNFα, whereas in CD, vedolizumab provided incremental benefit at an incremental cost, which was not considered cost effective at a threshold of $50,000/QALY.

Modelling the potential clinical and economic impact of universal immunisation with nirsevimab versus standard of practice for protecting all neonates and infants in their first respiratory syncytial virus season in Spain

BackgroundRespiratory syncytial virus (RSV) is associated with substantial morbidity among infants. This study modelled the potential public health and economic impact of nirsevimab, a long-acting monoclonal antibody, as an immunoprophylactic strategy for all infants in Spain in their first RSV season.MethodsA static decision-analytic model of the Spanish birth cohort during its first RSV season was developed to estimate the impact of nirsevimab on RSV-related health events and costs versus the standard of practice (SoP). Spain-specific costs and epidemiological data were used as model inputs. Modelled outcomes included RSV-related outpatient visits, emerging room (ER) visits, hospitalisations – including pediatric intensive care unit (PICU) admission, mechanical ventilation, and inpatient mortality.ResultsUnder the current SoP, RSV caused 151,741 primary care visits, 38,798 ER visits, 12,889 hospitalisations, 1,412 PICU admissions, and 16 deaths over a single season, representing a cost of €71.8 million from a healthcare payer perspective. Universal immunisation of all infants with nirsevimab was expected to prevent 97,157 primary care visits (64.0% reduction), 24,789 ER visits (63.9%), 8,185 hospitalisations (63.5%), 869 PICU admissions (61.5%), and 9 inpatient deaths (52.6%), saving €47.8 million (62.4%) in healthcare costs.ConclusionsThese results suggest that immunisation with nirsevimab of all infants experiencing their first RSV season in Spain is likely to prevent thousands of RSV-related health events and save considerable costs versus the current SoP.

US cost-utility model of lenacapavir plus optimized background regimen (OBR) vs fostemsavir plus OBR and ibalizumab plus OBR for people with HIV with multidrug resistance.

Heavily treatment-experienced (HTE) people with HIV (PWH) have limited treatment options owing to multidrug resistance (MDR). Lenacapavir (LEN) is indicated, in combination with other antiretrovirals, for the treatment of adults with MDR HIV-1 experiencing failure of their current antiretroviral regimen because of resistance, intolerance, or safety considerations. To evaluate the cost-utility of LEN in combination with an optimized background regimen (OBR) vs alternative recently approved treatments for HTE PWH, fostemsavir (FTR)+OBR and ibalizumab (IBA)+OBR, for the treatment of PWH with MDR, from a mixed US health care payer perspective. A Markov state-transition model with a lifetime time horizon was developed. Transition probabilities between viral load categories were based on individual participant data from the CAPELLA trial for LEN+OBR and on relative efficacy parameters obtained from indirect treatment comparisons for comparators. Health state utilities were sourced from the literature. Costs included drug acquisition costs, drug administration costs, disease management costs, adverse event costs, AIDS-related event costs, and treatment switching costs and were sourced from red book costs, Medicare and Medicaid fees, and the literature. Costs and outcomes were discounted at 3% annually. The model was used to estimate total and incremental costs, life-years (LYs), quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios. A deterministic and a probabilistic sensitivity analysis, as well as scenario analyses, were performed to address elements of uncertainty in the model and to explore the robustness of the results. Over a lifetime time horizon, LEN+OBR was associated with the highest absolute QALYs (9.41) and the greatest number of LYs (12.09) compared with FTR+OBR (QALYs: 8.75; LYs: 11.26) and IBA+OBR (QALYs: 8.36; LYs: 10.78). LEN+OBR was also associated with the lowest total lifetime costs of the 3 interventions (LEN+OBR: $1,441,122 [US dollars]; FTR+OBR: $1,504,986; IBA+OBR: $1,524,396) and therefore was dominant over both comparators in the base case. LEN+OBR remained dominant vs FTR+OBR and IBA+OBR across the range of scenarios tested and LEN+OBR had a 99% probability of being cost-effective compared with FTR+OBR and IBA+OBR in the probabilistic sensitivity analysis at a willingness-to-pay threshold of $50,000/QALY. This economic evaluation demonstrated that LEN+OBR provides meaningful increases in QALYs and LYs, and is dominant over a lifetime time horizon, compared with FTR+OBR and IBA+OBR for the treatment of PWH with MDR in the United States.

Varying the intensity of cystoscopic surveillance for high-risk non-muscle-invasive bladder cancer.

To compare the clinical, economic, and health utility outcomes associated with alternative cystoscopic surveillance regimens for high-risk non-muscle-invasive bladder cancer (HRNMIBC). We performed real-world clinical data-driven microsimulations of a hypothetical cohort of 100 000 patients diagnosed with HRNMIBC at age 70 years. The cohort was simulated to undergo alternative surveillance regimens recommended by five guidelines, and two hypothetical regimens-surveillance intensity escalation and de-escalation-which had a surveillance intensity moderately higher and lower, respectively, than the guideline-recommended regimens. We evaluated the 10-year cumulative incidence of muscle-invasive bladder cancer (MIBC), cancer-specific survival (CSS), overall survival (OS), and cost-effectiveness from a United States healthcare payer perspective. The guideline-recommended surveillance regimens led to an estimated 10-year cumulative incidence of MIBC ranging from 11.0% to 11.6%, CSS 95.0% to 95.2%, and OS 69.7% to 69.8%. Surveillance intensity escalation resulted in a 10-year cumulative incidence of MIBC of 10.5% (95% confidence interval [CI] 10.3-10.7%), CSS of 95.4% (95% CI 95.2-95.5%), and OS of 69.9% (95% CI 69.6-70.1%), vs 11.9% (95% CI 11.7-12.1%), 94.9% (95% CI 94.8-95.1%), and 69.6% (95% CI 69.3-69.9%), respectively, from surveillance intensity de-escalation. By increasing surveillance intensity, the number-needed-to-treat to prevent one additional MIBC progression over 10 years was ≥80, and ≥257 to avoid one additional cancer-related mortality. Compared to surveillance intensity de-escalation, higher-intensity regimens incurred an incremental cost of ≥$336 000 per incremental quality-adjusted life year gained, which well exceeded conventional willingness-to-pay thresholds, ≥$686 000 per additional MIBC progression prevented, and ≥$2.2 million per additional cancer-related mortality avoided. In microsimulations testing a wide range of cystoscopic surveillance intensity for patients newly diagnosed with HRNMIBC, moderate surveillance de-escalation appears associated with an insignificant change in 10-year OS and furthermore is cost-effective vs higher-intensity surveillance regimens. These results suggest that moderate surveillance de-escalation can reduce costs of care without compromising life expectancy for many patients.

Comparative Cost-Effectiveness of Gemcitabine and Cisplatin in Combination with S-1, Durvalumab, or Pembrolizumab as First-Line Triple Treatment for Advanced Biliary Tract Cancer.

The clinical effectiveness of triple chemotherapy consisting of gemcitabine, cisplatin plus either S-1 (GCS), durvalumab (DGC), or pembrolizumab (PGC) as first-line treatment for advanced biliary tract cancer (BTC) has been reported. However, their comparative cost-effectiveness is unclear. We conducted a model-based cost-effectiveness analysis from the perspective of Japanese healthcare payer. A 10-year partitioned survival model was constructed by comparing the time-dependent hazards of the KHBO1401-MITSUBA, TOPAZ-1, and KEYNOTE-966 trials. The cost and utility came from previously published reports. Quality-adjusted life years (QALY) were used to measure the effects on health. Costs for direct medical care were taken into account. There was a one-way analysis and a probability sensitivity analysis. A willingness-to-pay threshold of 7.5 million yen (57,034 USD) per QALY was defined. The incremental costs per QALY for GCS, DGC, and PGC in the base case study were 3,779,374 JPY (28,740 USD), 86,058,056 JPY (65,4434 USD), and 28,982,059 JPY (220,396 USD), respectively. No parameter had an influence beyond the threshold in a one-way sensitivity analysis. A probabilistic sensitivity analysis revealed that the probability of GCS, DGC, and PGC being cost-effective at the threshold was 85.6%, 0%, and 0%, respectively. Given the current circumstances, it is probable that triple therapy utilizing GCS will emerge as a plausible and efficient primary chemotherapy strategy for patients with advanced BTC in the Japanese healthcare system, as opposed to DGC and PGC.

Cost-effectiveness of blinatumomab for the treatment of B‑precursor acute lymphoblastic leukemia pediatric patients with high‑risk first‑relapse in Mexico

BackgroundBlinatumomab is a CD3/CD19-directed bispecific T-cell engager molecule that engages T cells to lyse CD19-expressing B cells. Based on a multicenter, open-label, phase 3, randomized clinical trial (Clinical Trials ID: NCT02393859), we aimed to evaluate the cost-effectiveness (CE) of blinatumomab compared to standard consolidation chemotherapy (SC) for the treatment of pediatric patients with high-risk first-relapsed Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (B-ALL) from a Mexico healthcare payer perspective. MethodsA decision-analytic model, a partitioned survival model, was used to estimate the life-years (LYs) and costs over a lifetime horizon. We assumed that patients who remained alive beyond a 5-year period were cured. To account for the lingering impacts of cancer treatment, an excess mortality rate was incorporated into the model. Event-free survival (EFS) and overall survival (OS) were estimated by fitting mixture-cure and standard parametric survival distributions to the time-to-event data from the phase 3 trial. The model accounted for treatment costs, adverse event costs, follow-up costs, subsequent allogeneic hematopoietic stem cell transplantation (alloHSCT) costs, and subsequent treatment costs. ResultsBlinatumomab was associated with a lifetime gained of 5.11 years at an incremental cost of $621,111 MXN, relative to SC. The ICER for blinatumomab vs Standard of care was estimated to be $121,526 MXN/LY gained in the base case. Cost-effectiveness was sensitive to varying the time horizon. Blinatumomab had a probability of 99 % of being cost-effective, relative to SC, at the willingness to pay threshold defined in Mexico. LimitationsHealth-related quality of life values were not included in the analysis and therefore we did not estimate the quality-adjusted life-years gained. ConclusionsBlinatumomab was associated with greater benefit in terms of OS and EFS relative to SC. Probabilistic, deterministic, and scenario analyses indicate that blinatumomab represents the best value for money. Therefore, blinatumomab administered as part of consolidation therapy in B-ALL pediatric patients with high-risk first relapse is a cost-effective option.

COST ANALYSIS OF DIRECT ANTERIOR VERSUS DIRECT LATERAL APPROACH FOR OUTPATIENT TOTAL HIP ARTHROPLASTY

The increased demand for total hip arthroplasty (THA) is having a significant impact on healthcare resources, resulting in increased interest in outpatient care pathways to reduce resource consumption. This study compared costs between patients who underwent outpatient THA using a Direct Anterior (DA) approach compared to a Direct Lateral (DL) approach to understand the effect of surgical approach on resource use.We conducted a prospective randomized controlled trial for DA patients undergoing primary THA. We compared patients in the outpatient arm of the trial to a prospective cohort of outpatient DL approach THAs. We recorded all costs including: equipment, length of stay in hospital, and laboratory or other medical tests. Following discharge, participants also completed a self-reported cost diary recording resource utilization such as emergency department visits or subsequent hospitalizations, tests and procedures, consultations or follow-up, healthcare professional services, rehabilitation, use of pain medications, informal care, productivity losses and out of pocket expenditures.We report costs from both Canadian public health care payer (HCP) and a societal perspective. The HCP perspective includes any direct health costs covered by the publicly funded system. In addition to the health care system costs, the societal perspective also includes additional costs to the patient (e.g. physiotherapy, medication, or assistive devices), as well as any indirect costs such as time off paid employment for patients or caregivers.We included 127 patients in the DA group (66.6 years old) and 51 patients in the DL group (59.4 years old) (p<0.01).There were no statistically significant differences in costs between groups from both the healthcare payer (DA= 7910.19, DL= 7847.17, p=0.80) and societal perspectives (DA= 14657.21, DL= 14581.21, p=0.96)In patients undergoing a successful outpatient hip replacement, surgical approach does not have an effect on cost from in hospital or societal perspectives.

A Pilot Study on the Drug Price Revision Strategy in Japan: A Comparison Among Fiscal Years 2018, 2020, and 2022

Objective: Japan has resumed its health technology assessment to decide how to reduce high-cost drug prices. While drug price rules in Japan are comprehensive, they do not necessarily capture differences in product characteristics. This study examined the drug price revision strategy in Japan using migraine treatment with triptans as an example. Cost data from fiscal years (FY) 2018, 2020, and 2022 were utilized. Methods: A cost-utility analysis was conducted from the perspective of healthcare payers, focusing on Japanese patients aged over 18 years experiencing migraines. The study employed a base-case model with probabilities derived from a network meta-analysis. Direct costs included medical and drug costs. Effectiveness was assessed using the European Quality of Life 5-dimensions—3-level questionnaire. Deterministic and probabilistic sensitivity analyses were conducted to examine the level of uncertainty. Results: In FY2018, sumatriptan and eletriptan were cost-effective; however, the other triptans were dominated by sumatriptan. In FY2020, sumatriptan and eletriptan were cost-effective, and rizatriptan was extended-dominated; nevertheless, the other triptans were dominated by sumatriptan. In FY2022, naratriptan and eletriptan were cost-effective; however, the other triptans were dominated by naratriptan. The hierarchy of triptan strategies varied in each fiscal year. Conclusions: This study provides valuable insights into the drug price revision strategy in Japan. The variations could be problematic because in Japan, formulary management of triptans, for example, those for migraine, may face revaluation every other year. Discussions regarding this issue will be further explored in the future.

Silicone adhesive multilayer foam dressings to prevent hospital-acquired sacrum pressure ulcers: An economic evaluation based on a publicly funded pragmatic randomized controlled trial linked with real-world data

ObjectivesTo estimate the cost-effectiveness of sacrum multilayer silicone foam dressings as an adjuvant prophylactic therapy compared to standard pressure ulcer prevention in a hospital population at high risk for pressure ulcer development. MethodsAn economic evaluation is performed from a healthcare payer's perspective. This evaluation is based on a Belgian publicly funded pragmatic randomized controlled trial (RCT), linked with real-world data from administrative claims database and a Belgian cost analysis. A cost-consequences analysis with a one-year time horizon is performed. ResultsThe RCT has shown that the risk of developing a new pressure ulcer on the sacrum was statistically significantly reduced by 41 % in the treatment group (RR = 0.59, 95 % CI 0.35–0.98, p = 0.04). The absolute risk reduction of 2.0 % (95 % CI -0.1–4.1 %) coincides with a number needed to treat of 50.0 to prevent one new pressure ulcer of category II or worse. The evolution of quality of life is on average negative for patients who developed a pressure ulcer before day 3, while it is positive for patients without pressure ulcers. In a scenario with conservative assumptions, i.e. without inclusion of price discounts for the multilayer silicone foam dressings and only including costs during the hospitalization, pressure ulcer prevention with dressings on the sacrum was already cost-neutral. ConclusionsThe preventive use of silicone adhesive multilayer foam dressings on the sacrum for a population similar to the pragmatic trial population can be supported both from a clinical and economic point of view.

Economic evaluation of the “paramedics and palliative care: bringing vital services to Canadians” program compared to the status quo

ObjectiveBased on programs implemented in 2011–2013 in three Canadian provinces to improve the support paramedics provide to people receiving palliative care, the Canadian Partnership Against Cancer and Healthcare Excellence Canada provided support and funding from 2018 to 2022 to spread this approach in Canada. The study objectives were to conduct an economic evaluation of “the Program” compared to the status quo.MethodsA probabilistic decision analytic model was used to compare the expected costs, the quality-adjusted life years (QALYs) and the return on investment associated with the Program compared to the status quo from a publicly funded healthcare payer perspective. Effectiveness data and Program costs, expressed in 2022 Canadian dollars, from each jurisdiction were supplemented with literature data. Probabilistic sensitivity analyses varying key model assumptions were conducted.ResultsAnalyses of 5416 9-1-1 calls from five jurisdictions where paramedics provided support to people with palliative care needs between April 1, 2020 and March 31, 2022 indicated that 60% of the 9-1-1 calls under the Program enabled people to avoid transport to the emergency department and receive palliative care at home. Treating people at home saved paramedics an average of 31 min (range from 15 to 67). The Program was associated with cost savings of $2773 (95% confidence interval [CI] $1539–$4352) and an additional 0.00069 QALYs (95% CI 0.00024–0.00137) per 9-1-1 palliative care call. The Program return on investment was $4.6 for every $1 invested. Threshold analyses indicated that in order to be cost saving, 33% of 9-1-1 calls should be treated at home under the Program, the Program should generate a minimum of 97 calls per year with each call costing no more than $2773.ConclusionThe Program was cost-effective in the majority of the scenarios examined. These results support the implementation of paramedic-based palliative care at home programs in Canada.

Adverse drug events in cost-effectiveness models of pharmacological interventions for diabetes, diabetic retinopathy, and diabetic macular edema: a scoping review.

The objective of this review was to examine the role of adverse drug events (ADEs) caused by pharmacological interventions in cost-effectiveness models for diabetes mellitus, diabetic retinopathy, and diabetic macular edema. Guidelines for economic evaluation recognize the importance of including ADEs in the analysis, but in practice, consideration of ADEs in cost-effectiveness models seem to be vague. Inadequate inclusion of these harmful outcomes affects the reliability of the results, and the information provided by economic evaluation could be misleading. Reviewing whether and how ADEs are incorporated in cost-effectiveness models is necessary to understand the current practices of economic evaluation. Studies included were published between 2011-2022 in English, representing cost-effectiveness analyses using modeling framework for pharmacological interventions in the treatment of diabetes mellitus, diabetic retinopathy, or diabetic macular edema. Other types of analyses and other types of conditions were excluded. The databases searched included MEDLINE (PubMed), CINAHL (EBSCOhost), Scopus, Web of Science Core Collection, and NHS Economic Evaluation Database. Gray literature was searched via the National Institute for Health and Care Excellence, European Network for Health Technology Assessment, the National Institute for Health and Care Research, and the International Network of Agencies for Health Technology Assessment. The search was conducted on January 1, 2023. Titles and abstracts were screened for inclusion by 2 independent reviewers. Full-text review was conducted by 3 independent reviewers. A data extraction form was used to extract and analyze the data. Results were presented in tabular format with a narrative summary, and discussed in the context of existing literature and guidelines. A total of 242 reports were extracted and analyzed in this scoping review. For the included analyses, type 2 diabetes was the most common disease (86%) followed by type 1 diabetes (10%), diabetic macular edema (9%), and diabetic retinopathy (0.4%). The majority of the included analyses used a health care payer perspective (88%) and had a time horizon of 30 years or more (75%). The most common model type was a simulation model (57%), followed by a Markov simulation model (18%). Of the included cost-effectiveness analyses, 26% included ADEs in the modeling, and 13% of the analyses excluded them. Most of the analyses (61%) partly considered ADEs; that is, only 1 or 2 ADEs were included. No difference in overall inclusion of ADEs between the different conditions existed, but the models for diabetic retinopathy and diabetic macular edema more often omitted the ADE-related impact on quality of life compared with the models for diabetes mellitus. Most analyses included ADEs in the models as probabilities (55%) or as a submodel (40%), and the most common source for ADE incidences were clinical trials (65%). The inclusion of ADEs in cost-effectiveness models is suboptimal. The ADE-related costs were better captured than the ADE-related impact on quality of life, which was most pronounced in the models for diabetic retinopathy and diabetic macular edema. Future research should investigate the potential impact of ADEs on the results, and identify the criteria and policies for practical inclusion of ADEs in economic evaluation.

Health-Related Quality of Life and Economic Analysis of Olanzapine Versus Aprepitant in Preventing Chemotherapy-Induced Nausea and Vomiting in Patients Receiving Highly Emetogenic Chemotherapy in Malaysia

ObjectivesOlanzapine has been shown to be effective in preventing chemotherapy-induced nausea and vomiting (CINV) after highly emetogenic chemotherapy (HEC); however, there is limited work on the impact of CINV on health-related quality of life (HRQoL) and the comparative cost-effectiveness of CINV prophylaxis in the Malaysian context. Therefore, this study was conducted to determine the HRQoL using EQ-5D-5L and the cost-effectiveness of olanzapine compared with aprepitant for CINV prophylaxis in Malaysia using data from a local study. MethodsFifty-nine chemo-naive patients receiving either olanzapine or aprepitant were randomly recruited and completed the EQ-5D-5L before and day 5 after HEC. HRQoL utility scores were analyzed according to the Malaysian valuation set. The economic evaluation was conducted from a healthcare payer perspective with a 5-day time horizon. Quality-adjusted life days (QALD) and the rate of successfully treated patients were used to measure health effects. The incremental cost-effectiveness ratio is assessed as the mean difference between groups’ costs per mean difference in health effects. A one-way sensitivity analysis was performed to assess variations that might affect outcomes. ResultsAprepitant and olanzapine arms’ patients had comparable baseline mean HRQoL utility scores of 0.920 (SD = 0.097) and 0.930 (SD = 0.117), respectively; however, on day 5, a significant difference (P value = .006) was observed with mean score of 0.778 (SD = 0.168) for aprepitant and 0.889 (SD = 0.133) for olanzapine. The cost per successfully treated patient in the aprepitant arm was 60 times greater than in the olanzapine arm (Malaysian Ringgit [MYR] 927 vs MYR 14.83). Likewise, the cost per QALD gain in the aprepitant arm was 36 times higher than in the olanzapine arm (MYR 57.05 vs MYR 1.57). Incremental cost-effectiveness ratio of MYR −937.00 (USD −200.98) per successfully treated patient and MYR −391.84 (USD −85.43) per QALD gained for olanzapine compared with the aprepitant-based regimen. ConclusionsAn olanzapine-based regimen is a cost-effective therapeutic substitution in patients receiving HEC in Malaysia.

Comparative cost-effectiveness analysis of CDK4/6 inhibitors in the first-line treatment of HR-positive and HER2-negative advanced breast cancer: a Markov's model-based evaluation.

CDK4/6 inhibitors are the first-line treatment for HR+/HER2- advanced breast cancer. Despite their clinical benefit, they can increase healthcare expenditure. To date, there is no thorough comparison among the three approved CDK4/6 inhibitors in terms of their cost-effectiveness. To investigate and compare the cost-effectiveness of CDK4/6 inhibitors in combination with letrozole as a first-line treatment for advanced breast cancer with hormonal-receptor-positivity and HER-2-negativity versus one another and versus letrozole monotherapy. A 10-year within-cycle-corrected Markov's model was employed from the healthcare payer perspective. Costs were obtained from the National Center for Cancer Care and Research (NCCCR) in Qatar. Utilities and transition probabilities were calculated from published landmark trials of PALOMA-2, MONALEESA-2, MONARCH-3, PO25, and other relevant literature. Costs, measured in Qatari Riyal (QAR), and effectiveness, measured in quality-adjusted-life-years (QALYs), were incremented and the incremental cost-effectiveness ratio (ICER) was compared to a willingness-to-pay threshold (WTP) of 1.5 Qatari GDP (448,758 QAR). A deterministic sensitivity analysis was implemented to account for uncertainties. Ribociclib was the most effective option, generating 4.420 QALYs, followed by palbociclib (4.406 QALYs), abemaciclib (4.220 QALYs), then letrozole monotherapy (2.093 QALYs). As for cost-effectiveness, ribociclib dominated palbociclib. However, it was not cost-effective compared to abemaciclib (ICER=1,588,545 QAR/QALY). Ribociclib remained dominant over palbociclib with all uncertainties. The base-case conclusion of ribociclib versus abemaciclib remained robust over all uncertainties. From the healthcare payer perspective in Qatar, ribociclib is the most effective CDK4/6 inhibitor. It was dominant over palbociclib in terms of cost-effectiveness; however, it was not cost-effective compared to abemaciclib at current prices.

Shall We Screen Lung Cancer with Volume Computed Tomography in Austria? A Cost-Effectiveness Modelling Study.

This study assessed the cost-effectiveness of a lung cancer screening (LCS) program using low-dose computed tomography (LDCT) in Austria. An existing decision tree with an integrated Markov model was used to analyze the cost-effectiveness of LCS versus no screening from a healthcare payer perspective over a lifetime horizon. A simulation was conducted to model annual LCS for an asymptomatic high-risk population cohort aged 50-74 with a smoking history using the Dutch-Belgian Lung Cancer Screening Study (NEderlands-Leuvens Longkanker ScreeningsONderzoek, NELSON) screening outcomes. The principal measure utilized to assess cost-effectiveness was the incremental cost-effectiveness ratio (ICER). Sensitivity and scenario analyses were employed to determine uncertainties surrounding the key model inputs. At an uptake rate of 50%, 300,277 eligible individuals would participate in the LCS program, yielding 56,122 incremental quality-adjusted life years (QALYs) and 84,049 life years gained compared to no screening, with an ICER of EUR 24,627 per QALY gained or EUR 16,444 per life-year saved. Additionally, LCS led to the detection of 25,893 additional early-stage lung cancers and averted 11,906 premature lung cancer deaths. It was estimated that LCS would incur EUR 945 million additional screening costs and EUR 386 million additional treatment costs. These estimates were robust in sensitivity analyses. Implementation of annual LCS with LDCT for a high-risk population, using the NELSON screening outcomes, is cost-effective in Austria, at a threshold of EUR 50,000 per QALY.

Cost analysis of patiromer vs sodium zirconium cyclosilicate for the treatment of hyperkalemia in Spain and the UK

Background Hyperkalemia is an electrolyte abnormality with potentially life-threatening consequences. Published data have shown that potassium-binding polymer patiromer (Veltassa) is associated with reduced rates of severe edema and hospitalization for heart failure compared with sodium zirconium cyclosilicate (SZC, Lokelma) when treating hyperkalemia. The aim of this study was to evaluate the possible costs associated with these interventions in the Spanish and UK settings. Methods A cost-analysis model was developed in Microsoft Excel to compare the costs associated with patiromer and SZC for the management of hyperkalemia. Clinical event rates were taken from a published real-world comparative study, with the base case capturing the statistically significant reduction in severe edema with patiromer vs SZC and a sensitivity analysis also including the non-statistically significant reduction in hospitalization for heart failure. Country-specific costs, expressed in 2022 Euros (EUR) and British pounds sterling (GBP), were evaluated from a healthcare payer perspective and included pharmacy costs and costs of clinical events. Results Patiromer may be associated with cost savings of EUR 107 and GBP 630 per patient-year of treatment vs SZC in Spain and the UK, respectively. The majority of cost savings were due to the possible lower daily cost of patiromer compared with SZC. Including the difference in heart failure hospitalization rates in a sensitivity analysis led to greater cost savings with patiromer over SZC, increasing to EUR 460 and GBP 902 in Spain and the UK, respectively. Extrapolation of patient-level economic outcomes to a population level found that patiromer was associated with annual cost savings of EUR 30.6 million in Spain, and GBP 801.7 million in the UK vs SZC. Conclusions Patiromer has the potential to be cost saving vs SZC for the treatment of hyperkalemia in Spain and the UK based on the results of a real-world evidence analysis.

Cost-Effectiveness of Dupilumab and Oral Janus Kinase Inhibitors for the Treatment of Moderate-to-Severe Atopic Dermatitis in Singapore.

Atopic dermatitis (AD) affects both adults and children, impacting their quality of life and productivity; however, traditional systemic treatments such as cyclosporine have limitations. Emerging novel systemic interventions, including monoclonal antibodies and Janus kinase (JAK) inhibitors, have been shown to improve patient outcomes. This study evaluates the cost-effectiveness of novel systemic interventions for moderate-to-severe AD in adults compared with the best supportive care (BSC) in Singapore. The economic evaluation used a hybrid model consisting of a decision tree and Markov model. Treatment responses at 16 weeks were based on a network meta-analysis that was developed specifically for this study. Long-term response, discontinuation rates, episodes of flares and treatment-emergent adverse events were obtained from key dupilumab, abrocitinib, baricitinib and upadacitinib trials. The study had a 5-year time horizon and considered the healthcare payer's perspective. Sensitivity and scenario analyses were performed as well. Baricitinib 4mg and 2mg have the lowest incremental cost-effectiveness ratios, at Singapore dollars (S$)60,730/quality-adjusted life-year (QALY) and S$66,842/QALY, respectively. Upadacitinib 30mg offers the highest incremental QALY gain, while baricitinib 2mg offers the least. The cost of the intervention drugs accounted for the highest proportion of the overall expenses (68-93%) for those in the maintenance state. Other influential factors within the model included (1) the incremental utility derived from intervention response; (2) the probability of achieving Eczema Area and Severity Index 75 (EASI-75) with BSC; and (3) the relative risk of achieving EASI-75 with the interventions. In a scenario where the cost of all drugs is matched to the lowest-priced drug, the top three cost-effectiveness interventions are dupilumab, upadacitinib 30mg and abrocitinib 200mg, respectively. The interventions are not found to be cost-effective at their existing prices when compared with BSC. Ideally, a composite score of treatment success and quality-of-life scores ought to be included, but such data were unavailable. Future research should consider conditional discontinuation data and long-term outcomes when such data become accessible.