ClinicoEconomics and Outcomes Research最新文献

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.

Purpose: Lung cancer remains a leading cause of cancer-related mortality. Tumor Treating Fields (TTFields) therapy extended survival in patients with metastatic non-small cell lung cancer (NSCLC) on or after platinum-based therapy. This study evaluates the cost-effectiveness of TTFields therapy concomitant with immune checkpoint inhibitors (ICIs) or docetaxel.

Methods: A model-based health economic evaluation estimated lifetime costs, clinical benefits, and humanistic outcomes of TTFields therapy plus ICI or docetaxel versus ICI or docetaxel alone in metastatic NSCLC. The model used clinical data from the LUNAR study, US healthcare cost data, and quality-adjusted life year (QALY) measures.

Results: The addition of TTFields therapy to an ICI or docetaxel resulted in a mean life-year gain of 0.92 and a QALY gain of 0.66, with an incremental cost-effective ratio (ICER) of $89,808 per QALY gained. TTFields therapy plus an ICI had 1.67 additional life years and 1.21 additional QALYs compared to an ICI alone, with an ICER of $58,764 per QALY gained. For TTFields therapy plus docetaxel, the life-year gain was 0.23 and the QALY gain was 0.17, with an ICER of $306,029 per QALY gained. Sensitivity analyses confirmed the robustness of these findings.

Conclusion: The addition of TTFields therapy to an ICI or docetaxel in metastatic NSCLC demonstrates comparable cost-effectiveness to other approved treatments. ICERs fall within the accepted range for US cost-effectiveness thresholds, supporting their use in clinical practice. TTFields therapy extended mean lifetime survival, offering a clinically meaningful and economically justifiable option for patients progressing after platinum-based chemotherapy.

Purpose: This study aimed to evaluate the costs and benefits of genetic testing, specifically mutation analysis and prenatal diagnostic testing, for the confirmation of thalassemia in at-risk pregnancies in Thailand, providing crucial insights to inform public health policy decision-making.

Patients and methods: We analyzed the costs and benefits of following standard screening guidelines, which included a sequence of tests such as mean corpuscular volume (MCV)/mean corpuscular hemoglobin (MCH) with dichlorophenol indophenol precipitation (DCIP), hemoglobin (Hb) typing, genetic testing, and amniocentesis. A decision-tree model was employed for this analysis. The study compared the scenarios with and without genetic testing, adopting a societal perspective that accounted for costs during pregnancy and the lifetime of a child born with thalassemia. Both one-way and probabilistic sensitivity analyses were conducted to account for uncertainties in the parameters used.

Results: The results revealed that adhering to the standard screening program with genetic testing resulted in a cost-savings of approximately 490 USD per prevented thalassemia case. Among the diagnostic methods, the specificity of the MCV/MCH with DCIP showed a higher degree of sensitivity relative to other testing methods, significantly influencing the outcomes. From a governmental perspective, with a full uptake of genetic testing, the incremental budget required was estimated to be 3.7 million USD (131 million THB) for one year.

Conclusion: These findings are particularly valuable for policymakers, as they provide robust evidence supporting potential revisions to the reimbursement structure within Thailand's Universal Health Coverage benefit package, facilitating better management of thalassemia and improving prenatal care.

Context: Polytrauma constitutes a major public health issue that is steadily increasing. In developing countries, including the Democratic Republic of Congo, this phenomenon is exacerbated by a combination of factors, such as inadequate funding mechanisms, the high cost of healthcare services and the low socio-economic level of the populations at risk. This study aims to assess the financial cost of treating polytrauma in a tertiary hospital in the Democratic Republic of the Congo.

Patients and methods: A case series study was conducted at the Jason SENDWE provincial referral general hospital in Lubumbashi, with data collected from 1 January to 31 December 2023. The study was based on the operational definitions of Heinrich and the economic burden of care at the individual level was calculated by dividing the average direct costs by the GDP per capita, PPP of the country.

Results: The present study comprised forty patients with polytrauma, with a mean age of 29.73 ± 9.9 years, ranging from 9 to 45 years.Approximately 65% of cases were attributed to road accidents, with a male predominance of 82.5%.The most frequently observed form of vital distress was neurological, accounting for 60% of cases, and le parage chirurgical comme acte chirurgical (28.11%). The overall survival rate was 7.50%, with a mean direct cost per patient of USD 608.77 ± 370.85 (range: USD 139.78 to USD 1826.34). This equates to a financial burden of 93.79 ± 57.13% of GDP per capita, ranging from 21.54 to 281.36% of GDP per capita.The proportion of out-of-pocket payments was 97.5%. The highest proportion of expenditure (42.2%) was attributed to medications, followed by the cost of surgical procedures (23.21%), and then imaging examinations (19.8%). Conversely, the lowest expenditure was observed to be related to resuscitation (1.21%) and laboratory tests (1.83%). It was observed that only polytrauma patients admitted to intensive care and hospitalised for 43 days or more exhibited a higher mean direct cost, with statistically significant differences (at a risk of 5%).

Conclusion: Patients with polytrauma are at significant risk of incurring catastrophic health expenditures. The results provide insight into the financial implications of polytrauma, which may inform the organisation of trauma care.

Background/context: Chronic low back pain (CLBP) is a significant US healthcare burden with millions of lumbar spine procedures annually. Diagnostic tests are essential to guide treatment but provocative discography (PD), the most common diagnostic procedure, is without robust evidence of its value. A non-invasive alternative using Magnetic Resonance Spectroscopy (MRS) offers a potential solution.

Context/purpose: We assess cost-effectiveness of MRS with NOCISCAN diagnostic algorithm compared to PD for identifying lumbar discs requiring surgical intervention.

Study design/setting: We conducted cost-effectiveness analysis using modelling.

Patient sample: We used data from a clinical study of 139 CLBP patients who met criteria for and received PD of lumbar spine and presented with an ODI score ≥40; comparing PD and MRS-based diagnostics.

Outcome measures: We considered diagnostic costs, adverse events, surgical costs and outcomes based on a 15-point improvement on the Oswestry Disability Index.

Methods: Incremental cost-effectiveness ratios (ICERS) and probabilistic sensitivity analyses were determined. Some authors have consulted for Aclarion.

Results: Mean total cost per PD patient was $59,711, and $57,998 for MRS, demonstrating $1712 cost savings per MRS diagnosed patient. Diagnostic costs ($1950 for PD; $1450 for MRS), saved $500 per MRS patient. PD incurred adverse event costs ($57,323) for 1% of patients, which MRS eliminated. MRS-based diagnosis showed 78% surgical success, whereas PD achieved 68%. MRS was the dominant diagnostic strategy, with better clinical outcomes and cost savings. Probabilistic sensitivity analysis confirmed MRS dominance and was cost-effective across a wide range of willingness-to-pay thresholds and across 2 different scenarios which vary base-case outcomes and surgical rates.

Conclusion: This study demonstrates cost-effectiveness dominance of MRS with the Nociscan diagnostic algorithm over PD for identifying CLBP surgical candidates. MRS provides significant cost savings and leads to better surgical outcomes, making it a preferred choice for insurers and health systems.