PharmacoEconomics最新文献

Background: Recently, the gene therapy eladocagene exuparvovec received accelerated approval from the US Food and Drug Administration (as eladocagene exuparvovec-tneq) for treatment of aromatic L-amino acid decarboxylase deficiency (AADCd), a rare, infantile-onset disorder characterized by developmental delays.

Objectives: To conduct a US, modified societal perspective cost-utility analysis comparing eladocagene exuparvovec versus best supportive care (BSC).

Methods: Multistate survival modeling was implemented tracking disease progression from a "no motor function" health state to achievement of motor-function improvements, measured by: (1) multiples of the meaningful score difference (MSD) of the Peabody Developmental Motor Scales-Second Edition (PDMS-2) total score and (2) motor milestones. Eladocagene exuparvovec trials informed clinical inputs. Health-state utilities were from a US time-trade-off study that valued AADCd quality-of-life impacts. Outcomes were discounted (3%); costs were reported in 2024 US dollars. Scenario analyses, characterizing alternative approaches of the multistate survival model analyses and probabilistic sensitivity analysis to assess the impact of parameter uncertainty, were conducted.

Results: Discounted incremental quality-adjusted life-years (QALYs) for eladocagene exuparvovec were 20.83 (multiples of the MSD of total PDMS-2) and 18.44 (motor milestones). Incremental cost per QALY ranged from $199,007-$224,104. The scenario and sensitivity analyses results supported the validity of the base case analysis.

Conclusions: Eladocagene exuparvovec is associated with considerable QALY gains compared with BSC. Within the context of other ultra-rare and/or one-time treatments, eladocagene exuparvovec provides substantial clinical improvements at lower cost than many other rare-disease treatments. Findings from this study highlight that eladocagene exuparvovec is an important treatment option for patients with AADCd.

Background and objective: Although heterogeneous treatment effects of sodium-glucose cotransporter 2 inhibitors (SGLT2is) have been revealed, the heterogeneous economic value of SGLT2is in real-world type 2 diabetes (T2D) populations with diverse clinical characteristics remains unclear. We conducted subgroup cost-effectiveness analyses of SGLT2is versus dipeptidyl peptidase 4 inhibitors (DPP4is) among patients with T2D.

Methods: A multi-state transition model was used to estimate the incremental cost-effectiveness ratios (ICERs, in US$ per quality-adjusted life-years [QALYs] gained) and value-based pricing (VBP) among patients with T2D stratified by age, estimated glomerular filtration rate (eGFR), glycated hemoglobin (HbA1c), and body mass index (BMI) over 5 years and a Lifetime horizon from a healthcare sector perspective, with both costs and quality-adjusted Life years discounted at 3% annually. Model inputs included treatment effects derived from analysis of individual-level data in Taiwan, and health utilities and costs sourced from published Literature of Taiwanese populations. Deterministic and probabilistic sensitivity analyses across subgroups were performed. All costs were standardized to 2022.

Results: Over 5 years, the ICERs of SGLT2is versus DPP4is were as follows: age subgroups (< 65 versus ≥ 65 years: $26,520 versus $2298/QALY-gained), eGFR subgroups (60 ~ < 90 versus ≥ 90 ml/min/1.73 m²: $7700 versus $12,884/QALY-gained), HbA1c subgroups (< 8.5 versus ≥ 8.5%: $7001 versus $9488/QALY-gained), and BMI subgroups (< 30 versus ≥ 30 kg/m²: $7266 versus $9714/QALY-gained). Over a lifetime, the ICERs became lower, ranging from $2369/QALY-gained for those aged ≥ 65 years to $4239/QALY-gained for those aged < 65 years. Over 5 years, the annual VBP of SGLT2is ranged from $310 for those aged < 65 years to $1267 for those aged ≥ 65 years.

Conclusions: Our analysis suggests that adopting SGLT2is over DPP4is for T2D is highly cost-effective across patient subgroups, particularly for the elderly and patients with mild renal impairment.

Background and objective: Aztreonam-avibactam (ATM-AVI) is a novel combination antibiotic approved in Europe for the treatment of complicated intra-abdominal infection, hospital-acquired pneumonia, including ventilator-associated pneumonia; complicated urinary tract infection, including pyelonephritis and for infections due to aerobic Gram-negative organisms with limited treatment options. This analysis assessed the cost effectiveness of ATM-AVI ± metronidazole versus colistin + meropenem (COL + MER) for the treatment of patients with complicated intra-abdominal infection and hospital-acquired pneumonia/ventilator-associated pneumonia, including infections with suspected metallo-β-lactamase-producing Enterobacterales from the public payer perspective in Italy using phase III trial data.

Methods: The cost-effectiveness analysis adopted a decision tree model to simulate the clinical pathway of complicated intra-abdominal infection and hospital-acquired pneumonia/ventilator-associated pneumonia, followed by a Markov model to capture lifetime health outcomes on cured patients, with costs valued in 2024 Euros and discounted at 3%. The model captures the impact of resistant pathogens and side effects (i.e. nephrotoxicity). Model uncertainty was assessed using a probabilistic and deterministic sensitivity analysis.

Results: The ATM-AVI treatment sequence (ATM-AVI ± metronidazole followed by cefiderocol after treatment failure) had improved clinical outcomes and higher cure rates, shorter hospital stays and higher quality-adjusted life-year gains compared with the COL + MER sequence (COL + MER followed by cefiderocol after treatment failure). The incremental cost-effectiveness ratio in the ATM-AVI sequence was dominant for complicated intra-abdominal infection and was €1552 per quality-adjusted life-year for hospital-acquired pneumonia/ventilator-associated pneumonia, well below the willingness-to-pay threshold of €30,000 in Italy.

Conclusions: Our analysis suggests that ATM-AVI is expected to be a cost-effective use of Italian healthcare resources for treating suspected metallo-β-lactamase-producing Enterobacterales, including complicated intra-abdominal infection and hospital-acquired pneumonia/ventilator-associated pneumonia.

Background: Immune checkpoint inhibitors (ICIs) are clinically beneficial but associated with high costs that represent a growing challenge for healthcare budgets and may affect affordability, especially in resource-limited settings. Moreover, the healthcare sector is a significant source of greenhouse gas emissions, and medication-related waste-such as that from vial-based therapies-has been identified as a contributing factor. Alternative dosing strategies could reduce the environmental and financial impact of ICI therapy while maintaining clinical safety and efficacy.

Methods: Population pharmacokinetic simulations were performed using virtual cohorts representative of the original cancer populations treated with ICIs. The analysis was conducted from a Western European hospital perspective, using Dutch public data to estimate costs (based on volume-dependent pricing) and carbon emissions from drug production, travel, and medical waste.

Results: Under the US Food and Drug Administration exposure-matching criteria, optimized dosing regimens reduced drug costs by up to €23,311 (- 28%) and carbon emissions by up to 255 kgCO₂e (- 30%) per patient, depending on the drug and dosing strategy. Using a broader therapeutic window approach, cost savings reached up to €40,135 (- 69%) and carbon reductions up to 501 kgCO₂e (- 63%) per patient. Incorporating vial sharing further increased potential cost savings to €5,721 per patient (- 31%). All estimates reflect European pricing and emissions factors, modeled over an 8-month treatment period.

Conclusions: These findings suggest that optimizing dosing strategies can yield meaningful economic and environmental benefits in ICI therapy while maintaining drug exposure within levels defined by US Food and Drug Administration criteria or broader therapeutic windows. A user-friendly application developed in this study allows users to generate virtual populations and evaluate tailored dosing strategies, facilitating practical implementation in diverse healthcare settings.

Objectives: The EQ-5D-5L is a multi-attribute utility instrument recommended by many health technology assessment agencies. This study aimed to develop an EQ-5D-5L value set for Singapore.

Methods: A 'lite' version of the EuroQol Research Foundation's EQ-5D-5L valuation protocol, which was designed to value a total of 91 health states using a composite time trade-off (cTTO) method, was followed. Five hundred members of the general public in Singapore were quota-sampled and invited to a personal interview face-to-face or via Zoom. All participants completed 20 cTTO tasks administered using the EuroQol Valuation Technology (EQ-VT) program. Cross-validation analysis was performed to identify the best-performing model for estimating the values of all the 3,125 EQ-5D-5L health states.

Results: A 20-parameter main-effects model with two two-way interaction terms outperformed other models in the cross-validation analysis. The value set estimated using this model ranges from - 0.851 (for state 55555) to 1.000 (for state 11111), with pain/discomfort and anxiety/depression dimensions associated with the greatest disutility.

Conclusions: We developed an EQ-5D-5L value set based on the health preferences of Singaporeans. We recommend EQ-5D-5L users in Singapore to use this value set and encourage a more systematic and dedicated methodological effort to understand interaction effects and potential non-linearities in the valuation of multi-attribute health descriptive systems.

Objectives: The clinical pathway for patients with moderate-to-severe Crohn's disease (CD) typically includes sequential pharmacologic treatment as well as surgery, but positioning of different therapies within these sequences remains challenging. Cost-utility analysis rarely captures these sequences and does not incorporate registry data on long-term effectiveness. In this study, we aim to overcome these limitations.

Methods: We developed an individual state transition model with four health states (active disease, remission, and remission due to surgery and death), five sequential treatment lines, and surgery. Efficacy data from network meta-analyses (NMA) for biologic naive and biologic exposed patients were combined with Dutch registry data to forecast long-term benefit, calculate costs, and estimate utilities. Analyses had a Dutch societal perspective with a lifetime time horizon. Costs were reported in 2023 euros and discounted with 3%. Effects were reported in quality-adjusted life years (QALYs) and discounted with 1.5%. The cost-per-QALY threshold was €20,000. Deterministic analyses for the base case, three scenarios (including recently published trials or price declines for ustekinumab), and one-way sensitivity analysis were run with 30,000 patients. The probabilistic sensitivity analysis was conducted by sampling 1000 patients in 1000 model runs.

Results: When opting for step-up sequences, the most cost-effective sequence (out of 156 sequences) starts with either azathiopurine/6-mp or methotrexate and is followed by combination therapy (infliximab + azathioprine) when patients discontinue their first line owing to disease activity or discontinuation. The most cost-effective top-down sequence (out of 72) starts with combination therapy (infliximab + azathioprine). After two lines of treatment, differences in cost-effectiveness between biologics become smaller. To be equally cost-effective as anti-tumor necrosis factor (TNF) combination therapy, a price decline for ustekinumab (biosimilars) of 81% is required or 50% to become the preferred option after combination therapy. Validation against external data suggested good predictive capabilities of the model.

Conclusions: Integrating NMA and registry data improves the quality of cost-effectiveness models for treatment sequences in CD. This open-source model can be easily updated for future therapies and holds the potential to become a standard model for use in clinical guideline development and the economic evaluation of new drugs.

Objective: To understand the application of productivity-adjusted life years (PALYs) as an outcome measure across various disease contexts.

Methods: We conducted a scoping review of studies published between 2018 and April 2025 that utilised PALYs to illustrate their potential applications and identify methodological approaches that have been applied. Using a citation-based search, we selected studies that applied PALYs to quantify societal health burdens in specific diseases or contexts. Extracted data included health conditions, country, timeframe, model type, outcomes, productivity index components, gross domestic product and sensitivity analysis. Findings were summarised through narrative synthesis.

Results: A total of 41 studies conducted between 2018 and 2025 were reviewed, including chronic diseases such as diabetes and cardiovascular diseases, as well as environmental factors. Conditions such as breast cancer, leukaemia, kidney disease, mental health, knee osteoarthritis, epilepsy and sleep apnoea had the lowest productivity indices. Most of these studies originated from high-income countries (n = 27), followed by upper-middle-income (n = 10), and lower-middle-income (n = 4) settings. Life table models were the most common methodological approach adopted (n = 26), followed by dynamic models (n = 10). Studies focused on disease prevention (n = 21) outnumbered those addressing disease management (n = 18). Most studies accounted for both absenteeism and presenteeism (n = 30). Estimates of productivity loss per person using gross domestic product ranged from US$1137 to AU$217,983 annually.

Conclusions: PALYs have been utilised in diverse diseases and contexts, highlighting their utility in measuring societal health impacts. However, adding unpaid and informal work makes burden estimates more accurate. The increasing emphasis on prevention indicates a strategic change in health policy and economic assessment.

Background: Advances in the availability and regimen optimization of highly effective disease-modifying treatments (DMTs) for relapsing-remitting multiple sclerosis (RRMS) have led to questions about their comparative worth.

Objectives: This study evaluates the costs and effects of natalizumab versus other highly effective DMTs and the impact, in terms of times and costs, of the new subcutaneous natalizumab formulation versus the intravenous formulation in patients with RRMS in Italy.

Methods: This is a cost-consequence analysis from the Italian national health service and societal perspectives. A Markov model was developed to assess clinical and cost outcomes related to disease and DMTs. The model simulated two scenarios: one comparing natalizumab extended-dose regimen and ofatumumab and ocrelizumab, focusing on efficacy outcomes and costs, and one comparing intravenous and subcutaneous natalizumab with a focus on administration resource consumption, times, and costs. Model input data came from the literature.

Results: DMTs had similar clinical and social outcomes: natalizumab slightly reduced disease progression, increased quality-adjusted life-years, and reduced the impact on days of productivity loss and informal care. Natalizumab also resulted in statistically significant 5-year cost reductions compared with ocrelizumab and ofatumumab. Subcutaneous natalizumab improved resource consumption compared with intravenous natalizumab, saving the time of healthcare professionals, patients, and caregivers and reducing administration costs. The subcutaneous formulation was associated with statistically significant total direct and indirect cost reductions at 5 years.

Conclusion: 6-week dosing regimen of natalizumab showed a slight improvement of clinical and social outcomes and a statistically significant cost reduction compared with ocrelizumab and ofatumumab over a 5-year simulation. Moreover, subcutaneous administration reduced administration times and costs.