ClinicoEconomics and Outcomes Research最新文献

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.

Purpose: Disease-modifying therapies (DMTs) are vital for managing multiple sclerosis (MS), but research using administrative data often excludes patient preferences and factors clinicians consider in treatment decisions. Patient experience data are crucial to understand and improve MS treatment initiation, adherence, and outcomes.

Methods: A cross-sectional survey of US adults with MS or clinically isolated syndrome was conducted online from December 2022 to January 2023 by the MS Coalition. A mixed methods analysis was conducted: logistic regression for quantitative data and thematic analysis of qualitative data.

Results: Among 1,323 participants (median age 55; 78% female), 80% expressed concerns about loss of independence, 65% about financial impacts, 64% about emotional impacts, 57% about relationships, and 42% about careers. Emotional tolls included identity loss, stress from navigating healthcare, and financial strain on families. Concerns varied by age, sex, and disability status. Nearly all participants (97%) reported DMT experience, with 73% having used two or more DMTs. Key factors in initiating DMT included slowing disease progression (92%), preventing relapses (89%), and following medical advice (89%). Financial barriers, such as high out-of-pocket costs, led to treatment delays or discontinuation in 19%. Barriers varied by demographic factors and included stress from medication costs, insurance denials, and fear of losing health coverage. Financial assistance was crucial for many. Half of participants had stopped a DMT due to doctor recommendations, side effects, or insurance issues.

Conclusion: The survey highlights the emotional and financial burdens of living with MS, including concerns about independence and relationships. The findings underscore the need for comprehensive care and provide actionable recommendations for managed care, research, and healthcare providers.

Purpose: Intravenous (IV) decitabine is a therapeutic option for patients with newly diagnosed acute myeloid leukemia (AML) ineligible for induction chemotherapy. Recently, the oral formulation of decitabine-cedazuridine demonstrated comparable efficacy and safety to IV decitabine, and pharmacokinetic equivalence. This study estimates the direct non-drug healthcare costs of IV decitabine administration in Italy, including central venous catheter (CVC) and infection management, and assesses the economic impact of oral decitabine introduction.

Methods: A micro-costing analysis from the Italian National Health Service (NHS) perspective was developed in four steps: 1) identification of the phases of IV and oral decitabine administration process, including CVC and infection management; 2) estimation of resource consumption, frequencies and proportion of patients for each phase; 3) collection of unit costs; 4) development of a cost analysis model. Inputs were retrieved from literature, public sources, IQVIA proprietary databases and a panel composed of clinicians, nurses and hospital pharmacists working in oncology departments. Two scenarios were explored: the first applying the economic impact to the population of interest over three years, the second including the cost of blood transfusions.

Results: The analysis estimated a total non-drug administration cost per patient of € 3574.6 and € 781.4 for a treatment course with IV and oral decitabine, respectively, leading to a cost impact of oral drug introduction of - € 2793.2 (-78.1%). The first scenario estimated a total saving for the Italian NHS of € 1.09 million over three years, the second scenario estimated a potential additional impact of - € 3418.6/patient due to transfusions.

Conclusion: The administration of oral versus IV decitabine is expected to generate cost savings for the Italian NHS in terms of drug administration, CVC and infection management, in patients with AML ineligible for induction chemotherapy.

Purpose: Chronic skin ulcers in diabetic foot patients are a significant health concern. Diabetic foot ulcers (DFUs) significantly threaten the health and longevity of individuals with diabetes, leading to severe complications like infection and amputation and contributing to high morbidity and mortality rates. Given the severe implications, practical strategies to prevent and manage DFUs are crucial to reducing amputation rates. Platelet-rich plasma (PRP) has emerged as a popular treatment option due to its properties that mimic the body's natural healing process. The objective of the study was to evaluate the cost-effectiveness of PRPR vs standard of care in US context.

Methods: Decision analytical model was used to synthesize clinical and economic parameters. In detail a CEA analysis was employed using a Markov decision-making model to evaluate patients with chronic DFUs lasting over three weeks and at high risk for orthopedic complications. The study assessed the effectiveness of different treatments, measured in quality-adjusted life years (QALYs), and reported costs in 2023 dollars using a micro-costing approach alongside a clinical trial.

Results: The study concluded that PRP gel is a cost-effective treatment for non-healing DFUs, resulting in lower care costs over one year compared to other treatments and cost savings over five years.

Conclusion: Thus, PRP treatment is a promising and practical option, improving patient outcomes and reducing healthcare costs. It is an attractive choice for healthcare providers and insurers in managing non-healing diabetic foot ulcers.

Purpose: To describe patients with hormone receptor positive and human epidermal growth factor receptor 2 negative metastatic breast cancer (HR+/HER2- mBC) in Italy for demographic and clinical variables, comorbidity profile, metastases and therapeutic pathways.

Patients and methods: From 2017 to 2021, HR+/HER2- mBC patients were extrapolated from administrative databases of healthcare entities covering a catchment area of about 3 million health-assisted women. The study included patients with a hospital discharge diagnosis for mBC; AND with specific prescriptions of therapies for HR+; AND without HER2-targeted therapy; OR with at least one prescription for CDK4/6 inhibitors. The following data were collected: age at inclusion, previous drug prescriptions, causes of hospitalization, site and number of metastases, therapeutic pathways and drug utilization during follow-up.

Results: The study was focused on 6603 women with HR+/HER2- mBC subtype, at least two prior systemic therapies for metastatic status or at least one endocrine-based therapy, at least one taxane prescription and at least one CDK4/6 inhibitor prescription and at least 12-months of data available before and after inclusion. Mean age was 59 years; the most common pre-existing conditions were hypertension (53.7%), distantly followed by chronic obstructive pulmonary disease, diabetes and cardiovascular disease. The analysis of treatment patterns during follow-up, which considered 3-month or 6-month gaps for identification of two different aspecific chemotherapies, showed that 97% (N = 236) had a subsequent line and 86% (N = 211) a further treatment during follow-up. The most common prior anticancer treatments, found in almost all patients, were endocrine therapy and CKD4/6i, with 66% patients receiving an aspecific chemotherapy.

Conclusion: This real-world analysis provides key insights into HR+/HER2- mBC in Italy, highlighting treatment patterns, rising diagnoses in younger women, and challenges in managing heavily pretreated patients. It emphasizes the need for further research on treatment sequencing, emerging therapies, and prior treatment duration to enhance clinical decision-making and patient care.

Background: Dose escalation to optimize advanced therapies is common in ulcerative colitis (UC) to avoid intra-class or inter-class drug switching and maintain clinical response and has impact on costs. Given the limited real-world data available, this study aims to understand real-world dose escalation UC advanced therapies patterns in France and United Kingdom [UK].

Methods: Retrospective study in adult patients with moderate-to-severe UC starting an advanced UC therapy (adalimumab [ADA], golimumab [GOL], infliximab [IFX], tofacitinib [TOF], ustekinumab [UST], or vedolizumab [VED]) with first prescription (and/or dispensation for France) between January 2017 and February 2022 (ie advanced UC therapy new users, by excluding patients who used any of these drugs in the previous 12 months to their index date). Proportions of patients with dose escalation/de-escalation (±20% versus Summary of Product Characteristics) after maintenance date were estimated using Kaplan-Meier (KM) survival analyses. Clinical response, healthcare resource utilization (HRU) and direct costs related to UC were also analyzed.

Results: Within 6 months after start of maintenance, rate of at least one dose escalation was 74.1%. Overall, 83.9-89% of patients had dose escalation within the 12-24 months, respectively, and 61.6% had clinical response [ranging from 56.3% (ADA) to 77.0% (IFX)]. Direct annual HRU costs related to UC ranged between 7426 (IFX) EUR and 22,265 (UST) in France, with mean 11,181 EUR in the dose-escalation group vs 8323 EUR in the de-escalation group (+11.5%). In the UK costs ranged between 5006 (ADA) EUR and 11,975 (UST).

Conclusion: Dose escalation of UC advanced therapies is a common strategy to avoid treatment-switching. Despite dose escalations and their cost to the system, a proportion of patients fail to achieve clinical response. This study highlights the need for more efficacious, durable treatments for moderate-to-severe UC patients, as the initiation of the advanced therapies did not reduce overall systemic/rectal corticosteroid burden.