ClinicoEconomics and Outcomes Research最新文献

Purpose: To compare the cost-effectiveness of mosunetuzumab with tisagenlecleucel for treating patients with relapsed or refractory follicular lymphoma (R/R FL 3L+) from the perspective of the Italian National Health Service (NHS).

Patients and methods: The analysis employs a weekly cycle partitioned survival model (PSM) with a lifetime horizon. The PSM model tracks patient outcomes based on time-to-event data, including progression-free survival (PFS) and post-progression survival (PPS). A matching-adjusted indirect treatment comparison (MAIC) approach was used to account for differences in trial population characteristics on the relative efficacy of mosunetuzumab to tisagenlecleucel. PFS and overall survival (OS) were extrapolated beyond the trial period by applying the hazard ratios from the MAIC to mosunetuzumab's parametric survival curves. Utility values and patient data are retrieved from the GO29781 trial. Economic inputs, from the perspective of the Italian NHS, include direct medical costs such as drugs, administration, monitoring, adverse event (AE) management, therapy following FL progression. Discontinuation and terminal care costs were also considered. Probabilistic sensitivity (PSA) and scenario analyses were conducted.

Results: Mosunetuzumab was found to be dominant compared to tisagenlecleucel, resulting in an increase of 0.98 life years (LYs) and 0.70 quality-adjusted life years (QALYs), while also being associated with lower overall costs. The sensitivity analysis consistently favored mosunetuzumab, with 94% of simulations demonstrating its cost-effectiveness based on the Italian WTP threshold of €40,000/QALY. Even in a scenario where tisagenlecleucel maintained a PFS advantage with assumed equivalence in OS, mosunetuzumab still showed a favorable cost-saving profile due to its lower incremental costs.

Conclusion: In the Italian setting, mosunetuzumab is a cost-effective treatment option compared to tisagenlecleucel for adult patients with R/R 3L+ FL, presenting favourable outcomes from the perspective of the NHS. Future research and data collection efforts are crucial to validate these findings and reduce uncertainties regarding long-term clinical and economic implications.

Purpose: Two analyses, a cost-minimization and a budget impact, were conducted to estimate the economic and financial impact of subcutaneous (SC) vs intravenous (IV) natalizumab in terms of administration times and costs in the Italian setting from the perspective of multiple sclerosis (MS) center, patient, and society.

Patients and methods: Cost minimization analysis (CMA) adopted a Markov model with three different states, and it is based on the results of REFINE study and its post-hoc analysis, which evaluated and demonstrated the non-inferiority of natalizumab SC vs IV formulation. The economic inputs came mainly from EASIER study, that estimated the administration time, resource consumption, and costs of natalizumab SC vs IV. A lifetime horizon was considered. Budget impact analysis (BIA) was conducted with a cost calculator approach and compared a base scenario (without SC natalizumab) with an alternative scenario (with SC natalizumab). The inputs were shared with the CMA and a 3-year time horizon was considered. A progressive increase in the number of patients treated with natalizumab SC was estimated from the 1st to the 2nd to the 3rd year after reimbursement in Italy.

Results: CMA estimated that savings due to the use of SC instead of IV natalizumab would be €2,824, €1,137, and €9,170 per patient from the perspectives of MS center, patient, and society, respectively, thus depicting a weak dominance (lower costs and non-inferiority efficacy). BIA estimated that the savings were approximately 3.2 million euros from the perspective of MS centers and around 10.3 million euros from the perspective of society in the first 3 years following reimbursement.

Conclusion: Administering natalizumab subcutaneously rather than intravenously to treatment-eligible patients would result in administration time and cost savings thus determining a favorable impact for the MS center, the patient and the society.

Purpose: Standard of care for patients with sickle cell disease (SCD) includes red blood cell transfusions (RBCTs). Data on clinical and economic outcomes of patients with SCD receiving frequent RBCTs are limited.

Materials and methods: This longitudinal, retrospective, claims-based analysis used the Merative™ MarketScan^® Commercial, Medicare, and Multi-State Medicaid databases. Patients with SCD (identified using ICD-9/10 codes) receiving frequent RBCTs (≥6 RBCTs during any 12-month period) between January 1, 2015, and March 1, 2019, were included. The index date was the date of the sixth RBCT. Eligible patients were required to have ≥12 months of continuous enrollment pre- and post-index. Patients were followed from index to end of enrollment, death, or end of the study period (February 29, 2020), whichever came first. Clinical complications, all-cause healthcare resource utilization (HCRU), and healthcare costs were descriptively summarized during follow-up.

Results: A total of 919 patients with SCD receiving frequent RBCTs met the eligibility criteria for inclusion. Patients experienced a mean of 4.0 vaso-occlusive crises (VOCs) per patient per year (PPPY) and received a mean of 8.3 RBCTs PPPY during follow-up. The most common clinical complications were iron overload (77%), infections (66%), and cerebrovascular disease (48%). Patients had a mean of 2.3 inpatient admissions, 83.5 outpatient visits, and 37.4 outpatient prescriptions PPPY during follow-up. Mean total annual healthcare costs were $106,123 PPPY, including mean inpatient, outpatient medical, and outpatient pharmacy costs of $48,463, $28,307, and $29,353, respectively. Compared to those with <2 baseline VOCs, patients with ≥2 baseline VOCs had more HCRU and higher annual healthcare costs.

Conclusion: Despite utilizing available care with frequent RBCTs, patients with SCD experienced a variety of disease and transfusion-related complications, including frequent VOCs and iron overload, which led to substantial HCRU and costs. These findings highlight the need for novel therapies for this patient group.

Purpose: To estimate the budget impact of adding elranatamab to the US formulary to treat adults with RRMM who have received ≥4 prior lines of therapy including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 monoclonal antibody, and to assess the total cost of care and cost per month of progression-free survival (PFS) between elranatamab and available treatments.

Methods: An economic model was developed to assess the budget impact of elranatamab in a one-million-member US commercial and Medicare health plan. Epidemiology data was obtained from the SEER database and a large US real-world study. Key clinical inputs included treatment duration, PFS, overall survival, and adverse events (AEs). Costs associated with drug acquisition monitoring, medical resource use (specifically hospitalization and physician visits), and AEs were incorporated. Model inputs were sourced from clinical trial data, US government databases, and published literature. Total budget impact and per member per month (PMPM) were assessed. One-way sensitivity analyses (OWSA) were conducted to assess model input uncertainty. Total cost of care and cost per month of PFS were also assessed.

Results: An estimated 14 (commercial) and 60 (Medicare) RRMM patients per year would be eligible for treatment. Adding elranatamab resulted in a total budget impact of $553,607 ($0.05 PMPM) in commercial and $2,351,515 ($0.20 PMPM) in Medicare over three years. OWSA indicated results were most sensitive for elranatamab drug costs and relative dose intensity. Total cost of care per month of median PFS over one year was $19,642 with elranatamab, talquetamab ($33,391), teclistamab ($37,791), selinexor plus dexamethasone ($48,784), physician's choice of treatment ($65,886), idecabtagene vicleucel ($78,361), and ciltacabtagene autoleucel ($17,640).

Conclusion: Elranatamab for RRMM is projected to result in a minimal to small budget impact over 3 years and good economic value with lower cost of care per month of PFS compared with other available RRMM treatments except for ciltacabtagene autoleucel.

Purpose: According to the World Health Organization European Regional Obesity Report, Turkey has the highest rate of overweight and obesity in Europe. This study used a weight loss pharmacoeconomic model to assess the influence of obesity on public health by examining its effects on private health institutions and its financial costs.

Patients and methods: A micro-costing approach was used to estimate the direct healthcare costs of 10 obesity-related comorbidities from the perspective of private healthcare providers in Turkey. A survey was conducted on a representative sample of physicians in Turkey to determine resource utilization rates for comorbidities in expenditures. The unit costs of each cost item were analyzed for type A, B, and C private hospitals. Costs in the different categories were obtained by multiplying the unit costs by the health resource utilization rate.

Results: When the obesity-related complications were stratified according to weight loss rate, 5%, 10%, and 20%, a higher cost reduction was observed in the 40-49, 50-59, and 60-69 age groups. It should be noted that this decrease in healthcare expenditure was detected in the older age groups (40 to 69) and not in individuals between 20 and 39 years. Another analysis of the weight loss rate revealed that the decrease was highest in Type 2 Diabetes Mellitus costs. A health expenditure that costs 1 unit in a C-segment institution increases 1.44-fold in B-segment and 3-fold in A-segment hospitals. The effects of weight loss on reducing the cost of obesity-related complications indicated that the highest cost reduction was on T2DM, dyslipidemia, and CKD, respectively. Obesity-related complications constituted 28.87% of total costs in Segment A hospitals, 29.13% in Segment B hospitals, and 28.54% in Segment C hospitals.

Conclusion: The current pharmacoeconomic model indicated that complications were the major cost drivers in obesity. Weight loss dramatically reduced healthcare expenditures in obese patients, and T2DM was the leading cause in all age groups.

Purpose: To evaluate economic outcomes in patients with hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) metastatic breast cancer (mBC) treated with a first- or second-line cyclin-dependent kinase 4/6 inhibitor (CDK4/6i).

Methods: This retrospective analysis utilized Optum's Clinformatics DataMart (January 1, 2014-September 30, 2021). Included patients had ≥1 pharmacy claim for palbociclib, abemaciclib, or ribociclib in first or second-line and ≥6 months of continuous health plan enrollment in preindex (index: date of first CDK4/6i claim) and follow-up periods. Mean all-cause per patient per month (PPPM) medical, healthcare resource utilization (HCRU) and costs, and outpatient pharmacy prescriptions costs were compared among CDK4/6is using stabilized inverse probability of treatment weighting (sIPTW).

Results: We identified 3,182 patients taking palbociclib, 286 taking abemaciclib, and 149 taking ribociclib, with median follow-ups of 20.8, 16.6, and 19.9 months, respectively. After sIPTW, palbociclib was associated with a lower risk of inpatient (IP) admissions versus abemaciclib (35.8% vs 41.6%; odds ratio: 1.31; P=0.034). No other significant differences were seen for HCRU. PPPM outpatient costs were significantly lower with palbociclib versus abemaciclib ($754; P=0.05). PPPM IP ($2,252 vs $6,286), medical ($6,948 vs $11,717), and total ($19,370 vs $23,639) costs were also lower with palbociclib versus abemaciclib, although not significant. There were no significant differences in PPPM HCRU or costs between palbociclib and ribociclib. In patients with Medicare, PPPM total medical costs were lower with palbociclib versus abemaciclib by $1,608 (P=0.04), while other costs were not significantly different. No significant differences in costs were seen with palbociclib versus ribociclib.

Conclusion: All-cause HCRU and costs were generally not different between the CDK4/6is but favored palbociclib for medical (including IP) costs versus abemaciclib. Due to limited patient numbers, uncertainty exists about abemaciclib and ribociclib cost estimations. Further studies of HCRU and costs are needed to support a cost-minimizing strategy for mBC.

Purpose: This study aimed to assess the cost-effectiveness of medical nutritional support in a cancer care program in North Macedonia, comparing specialized oral nutritional supplements (ONS) with the standard of care (SOC) in managing disease-related malnutrition (DRM) in patients with or at risk of tumor cachexia syndrome.

Methods: A previously published decision tree model was employed to evaluate the economic impact of supportive treatment in cancer patients eligible for ONS. Monthly transition probabilities between health states, length of hospital stay for each treatment strategy, and utility parameters were derived from the literature. For base-case analysis, the cancer care program duration was set at 30 days. The analysis was conducted from the perspective of a national health insurance fund, utilizing a 13-year time horizon with monthly cycles. Only direct supportive care costs, estimated from publicly available data, were considered. Quality-adjusted life-years (QALYs) gained per patient and associated costs were calculated, with outcomes and costs discounted at 3.0% annually. One-way and probabilistic sensitivity analyses were performed to assess results robustness.

Results: In the base case analysis, ONS was the dominant treatment strategy, with total costs per patient of €2605.01 for ONS versus €3759.23 for SOC, indicating a significant cost reduction. Reduced hospitalization expenses outweighed the higher acquisition costs of ONS. Additionally, ONS provided greater health benefits, achieving 8.21 QALY vs 7.91QALY in the SOC group. The resulting Incremental Cost-Effectiveness Ratio (ICER) was negative, reinforcing ONS as the dominant strategy. Sensitivity analyses confirmed that the cost-effectiveness was primarily driven by cancer program duration, with cost-saving benefits up to 132 days.

Conclusion: Our findings demonstrate that specialized ONS is a cost-effective treatment option within a cancer care program compared with SOC. While this study focuses on North Macedonia, the results are applicable to countries with similar economic and healthcare structures, reinforcing ONS as a valuable intervention across comparable healthcare systems.

Background: The rising prevalence of obesity in the Kingdom of Saudi Arabia (KSA) poses a significant public health challenge. Estimates of the economic cost of obesity are crucial for prioritizing healthcare interventions, guiding policy choices, and justifying budget allocations aimed at reducing obesity prevalence. This study aimed to estimate the cost of obesity in the KSA in 2022.

Methods: A prevalence-based cost-of-illness approach was used to determine the cost of obesity. This analysis encompasses 29 diseases, namely obesity and twenty-eight diseases attributable to obesity. Both direct and indirect costs were considered. The annual cost of treatment for each obesity-attributable disease was obtained from the hospital records of one tertiary hospital in the KSA. Data on direct non-medical costs were obtained from the patient survey. The human capital approach was used to estimate the indirect costs of morbidity and mortality.

Results: The total economic burden of obesity (2022 values) was estimated at US$116.85 billion from a societal perspective and US$109.67 billion from a healthcare system perspective. From a societal perspective, the total direct medical cost accounted for the largest portion of the total cost (94%). In terms of direct medical costs, the cost of treating diseases attributable to obesity was substantially greater than the cost of treating obesity itself. According to the sensitivity analysis, the total cost ranged from 3.4% of the country's Gross domestic product (GDP) when the unit cost of treatment was reduced by 74% to 9.5% of the country's GDP when the prevalence of obesity and its comorbidities was reduced by 5%.

Conclusion: Obesity imposes a substantial economic burden on the healthcare system and society in the KSA. Interventions aimed at promoting healthier lifestyles to reduce the prevalence and incidence of obesity and its comorbidities are highly warranted to alleviate the impact of obesity in the country.

Background and objectives: Ofatumumab, a fully human anti-CD20 monoclonal antibody, is a promising disease-modifying therapy (DMT) for relapsing-remitting multiple sclerosis (RRMS). This study investigates its cost-effectiveness compared to teriflunomide from the perspective of Saudi healthcare payers. This comparison is crucial for informing treatment strategies and resource allocation in Saudi Arabia, where RRMS poses a significant healthcare burden and access to newer DMTs is evolving.

Patients and methods: A Markov model was constructed to evaluate the long-term cost-effectiveness of ofatumumab compared to teriflunomide for treating RRMS in Saudi Arabia. This model simulates disease progression over 10 years, a timeframe chosen for its clinical relevance and consistency with similar studies. To reflect the Saudi patient population, the model uses a hypothetical cohort with characteristics mirroring those in the ASCLEPIOS I/II clinical trials. The model incorporates transition probabilities between disease states, primarily derived from the British Columbia MS (BCMS) database and further refined using data from the ASCLEPIOS trials. To ensure relevance to the Saudi context, local data sources were utilized, including drug costs from the Saudi Food and Drug Authority (SFDA) and health state costs from published local studies. Clinical expert input was incorporated to validate model assumptions.The primary outcome measure was the incremental cost per quality-adjusted life-year (QALY) gained. Sensitivity analyses were conducted to assess the robustness of the model findings.

Results: Compared to teriflunomide, ofatumumab yielded incremental cost-effectiveness ratios (ICERs) of $46,188 per QALY over the 10-year period. Ofatumumab demonstrated a greater impact on reducing disability progression, particularly in the early stages of the disease. At a willingness-to-pay (WTP) threshold of $99,120 per QALY, ofatumumab demonstrated a 99.14% probability of cost-effectiveness in probabilistic sensitivity analyses.

Conclusion: This cost-effectiveness analysis demonstrates that ofatumumab is a cost-effective treatment for RRMS in Saudi Arabia, with an ICER below the WTP. Policymakers should consider including ofatumumab in national formularies and prioritize its use in early-stage RRMS to maximize patient benefit and cost-effectiveness.

Background: Cangrelor is an intravenous, reversible P2Y12 inhibitor indicated for the reduction of thrombotic cardiovascular events in patients undergoing percutaneous coronary intervention (PCI) who have not received an oral P2Y12 inhibitor prior to the PCI procedure, and in whom oral therapy with P2Y12 inhibitors is not feasible or desirable (for example, in the out-of-hospital cardiac arrest [OHCA] population).

Objective: This study aimed to estimate the affordability and budget impact, in the United Kingdom, of introducing cangrelor within the licenced OHCA population.

Methods: A budget impact model was developed to estimate the impact of introducing cangrelor to hospitals over 5 years. Efficacy (thrombotic events) and safety (bleeding events) data were based on clinical trials, cost data (2021/22 GBP), literature, NHS reference costs and British National Formulary data. Comparators were glycoprotein IIb/IIIa inhibitors and aspirin in combination with heparin, reflecting current treatments used in UK centres for the target population. Cangrelor uptake was estimated as 50% in Year 1, 75% in Year 2, and 100% in Years 3-5. The OHCA population was estimated from the British Cardiovascular Intervention Society National Audit 2021/22.

Results: Over 5 years, cangrelor leads to modelled cost savings of £2,709,853 (-9.84%), varying from £322,218 in Year 1 (-5.85%) to £636,150 (-11.55%) in Year 5). This is driven by approximately 6,882 hospital days being avoided over 5 years due to fewer bleeding events.

Conclusion: Cangrelor for OHCA patients who cannot take oral P2Y12 inhibitors may lead to cost savings in the UK.