ClinicoEconomics and Outcomes Research最新文献

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.

Purpose: Incorporation of adverse drug events (ADEs) is suboptimal in economic evaluation, and thus the information provided by it may be inaccurate. Better guidance on incorporating ADEs into economic evaluation prompts for exploring whether the results are sensitive to ADEs.

Methods: This scoping review explored 242 cost-effectiveness models for pharmacological interventions for type 1 (T1DM) and 2 diabetes (T2DM), diabetic retinopathy (DR), and diabetic macular edema (DME), in relation to the type of ADEs included in the models (if any), whether the results were sensitive to the ADEs, and what could explain their potential impact.

Results: Of the analyses partly or completely including ADEs, 62% examined their impact on the results, with half of them (50%) reporting ADE-related sensitivity. The models included common to very common ADEs, and some rare but severe ones. The main reasons for excluding ADEs were low incidence (13%) and no reporting in clinical trials (13%). Many analyses reported no reason for the exclusion (53%). The analyses for T1DM and DR or DME included more severe ADEs and reported a higher ADE-related sensitivity compared to the analyses of T2DM (76,2%, 77.8%, and 46.4%, respectively). Higher incidence of ADEs (60,0%) and time trade off method (72,2%) were associated with higher ADE-related sensitivity (72,2%).

Conclusion: Incidence, condition, and the measure of utility were associated with the results being sensitive to ADEs. ADEs are an important outcome for the results of economic evaluation and better guidance on their inclusion and exclusion is needed.

Purpose: Serum or urine protein electrophoresis (SPEP or UPEP) and immunofixation electrophoresis (SIFE or UIFE) are routinely used to detect M-proteins in MM patients. However, SPEP and SIFE are not sensitive enough to measure M-protein levels that are low but still clinically significant. This study aimed to evaluate the potential cost savings associated with using the EXENT GAM Assay, a serum-based quantitative-immunoprecipitation mass spectrometry (QIP-MS) diagnostic test instead of SIFE to guide the timing of minimal residual disease (MRD) testing for patients with multiple myeloma (MM).

Patients and methods: A simple 2-year budget impact model was created in Excel using data from MM clinical trials and fee schedules. Patients are tested with either QIP-MS or SIFE at predetermined timepoints. If patients test negative, they will receive MRD testing. The result of the MRD test will determine if the preceding serum-based test was a true negative result (MRD test is negative) or a false negative result (MRD test is positive). Patients receiving autologous stem cell transplant (henceforth referred to as transplant) and those not receiving transplant are both eligible for one MRD test per year. MRD testing for transplant-eligible patients occurs prior to transplant and one year following transplant.

Results: Across a hypothetical population of 5154 mm patients receiving 1^st-line treatment in France, using QIP-MS instead of SIFE prior to MRD testing leads to 1973 fewer false negative results and 744 more false positive results (due, in part, to the detection of residual IgG). Net savings per QIP-MS test would be €260 or total savings of €2,481,832.

Conclusion: This study suggests that the use of QIP-MS prior to MRD testing may be cost-saving for testing French patients with MM.

Purpose: To assess the budget impact and cost-benefit of incorporating sodium-glucose cotransporter-2 inhibitors (SGLT-2i) into the benefit package for patients with heart failure (HF) under the universal health coverage (UHC) in Thailand.

Patients and methods: A budget impact analysis and cost-benefit model were developed using a five-year time horizon from the payer perspective. Dapagliflozin 10 mg daily or Empagliflozin 10 mg daily was considered as an additional treatment to standard of care (SoC) for patients with HF, under the UHC. Two analytical frameworks were applied: (1) only medicine cost and (2) medicine cost and cost of hospitalization for HF (HHF) and urinary tract infection (UTI) admission as the adverse event of SGLT-2i. The net budget impacts (NBI) were calculated along with the HHF cost reduction and benefit-cost ratio.

Results: The NBI in the first year in only medicine cost for dapagliflozin was 12,535 million Thai baht (THB) and that for empagliflozin was 13,265 million THB. The NBIs, when considering HHF and UTI admission costs, were 7661 and 7407 million THB in the first year. The prices of dapagliflozin and empagliflozin should be reduced by 57.13% and 52.07% to reach a budget impact of 500 million THB. The benefit-cost ratio was 0.396 for dapagliflozin and 0.456 for empagliflozin.

Conclusion: Incorporating SGLT-2i into the UHC would significantly impact the healthcare budget. Policymakers should consider this valuable evidence.

Purpose: Advancements in pathogen identification by diagnostic testing may improve patient outcomes. This study evaluated healthcare utilization and costs following diagnostic testing for acute oropharyngeal and respiratory tract infections (RTIs).

Patients and methods: Healthcare utilization and costs were evaluated in patients with acute oropharyngeal infections (n=1,172,693), and RTIs (n=4,005,228) who received a syndromic panel-based PCR test with next-day results (HealthTrackRx, Denton, TX), or no test in the IQVIA PharMetrics^® Plus adjudicated claims database.

Results: Statistically significant differences were observed between patients who received the PCR test compared to those who received no test. The PCR test cohort had lower total healthcare costs (mean = $5,601±$29,170, median = $807) versus the no test cohort (mean = $7,460±$40,817, median = $1,163) (p = 0.0014) over 6 months, and fewer outpatient visits, other medical service visits, emergency room visits, and inpatient stays (p<0.0001). Similarly, those who received the PCR test for oropharyngeal infection trended towards lower total healthcare costs (mean = $4,393±$13,524, median=$844) than those who received no test (mean = $5,503±$34,141, median = $956) (p=0.0525) and had fewer outpatient and other medical services (p<0.0001).

Conclusion: Next-day molecular testing for respiratory and oropharyngeal infection lowers healthcare utilization and costs, suggesting improved patient care through reduced need for healthcare resources.

Purpose: Many patients with atopic dermatitis (AD), a highly pruritic, relapsing, inflammatory skin disease, experience inadequate disease control. Ruxolitinib cream was approved in the US in September 2021 for the treatment of mild-to-moderate AD. This analysis describes treatment patterns before and after initiation of ruxolitinib cream among patients with AD.

Patients and methods: This retrospective, observational study used medical and pharmacy claims data from the Healthcare Integrated Research Database (HIRD^®) and included adults and adolescents (aged ≥12 years) with an AD diagnosis, a first claim for ruxolitinib cream (index date) between October 2021 and July 2022, and continuous enrollment in a commercial or managed Medicare plan for 6 months before and after the index date. Analyses were also conducted in a subset of patients with a history of more advanced AD therapy (ie, systemic therapies, phototherapy, or ultrahigh-potent topical corticosteroids). Data from 6 months before ruxolitinib cream initiation (baseline period) and after initiation (follow-up period) were summarized using descriptive statistics.

Results: Of 1,581 patients in the overall AD cohort, 749 had a history of more advanced AD therapy. During the follow-up period, 43.8% of patients did not receive any other AD treatment. Compared with baseline, fewer patients received topical corticosteroids (52.3% vs 30.4%), topical calcineurin inhibitors (13.9% vs 6.6%), and topical phosphodiesterase-4 inhibitors (4.4% vs 2.3%) during the follow-up period; slightly greater reductions were observed among the subset with a history of more advanced AD therapies. Oral corticosteroid use decreased from 20.9% to 15.5% overall and from 44.1% to 20.7% in the subset with more advanced baseline therapy. Among patients receiving biologics at baseline, 17.4% did not receive these treatments during the follow-up period.

Conclusion: These 6-month follow-up data suggest that initiating ruxolitinib cream for AD may reduce the overall need for other topical treatments, oral corticosteroids, and biologics.