Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Objective: To compare the cost of adverse events (AEs) associated with the acute treatment with rimegepant (RIM) versus lasmiditan (LAS) for migraine in Spain.

Methods: A probabilistic modeling analysis was performed, using second-order Monte Carlo simulations, from the perspective of the Spanish National Health System (SNHS). The cost per patient of all AEs described with RIM or LAS in 12 clinical trials, obtained through a systematic review, was analyzed. Several sensitivity analyzes (among them, a matching adjusted indirect comparison -MAIC- of the two long-term studies) were also performed. The cost of AEs management (€ 2024) was obtained from Spanish sources.

Results: The probabilistic model estimated that RIM compared to LAS would generate savings of €612.79 (95% CI €159.49-1339.43) per treated patient with migraine, in a treatment period of 6.11 ± 3.25 months, €98.54 per month. In the MAIC analysis, a saving per patient of €697.04 (95% CI: €514.44-879.04) was obtained in a treatment period of 12.4 months. The probability of RIM saving in all analysis was 100%.

Conclusions: In accordance with this model, the favorable safety profile of RIM compared to LAS would generate savings for the SNHS in health-care resources in all the scenarios considered.

Background: Psoriatic arthritis (PsA) requires long-term, phenotype-oriented management. This study compared the cost-utility of ixekizumab, tofacitinib, and golimumab using a real-world modeling approach.

Research design and methods: A 10-year Markov model was developed from the perspective of the Spanish National Health System, informed by three real-world PsA cohorts. Transition probabilities were derived from drug-persistence data. Utilities and direct medical costs were obtained from published sources. Approved dosing, standard treatment durations, and annual cycles were modeled. Main outcomes included total costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs). Scenario analyses evaluated a 20% price reduction and phenotype-specific settings (enthesitis, dactylitis, axial PsA, refractory disease, prior TNFi failure, and relevant comorbidity).

Results: All three agents were cost-effective in the base case, with ICERs below the €30,000/QALY threshold. Ixekizumab provided the highest QALYs and was most cost-effective in axial PsA, enthesitis, and refractory disease. Tofacitinib, with the lowest total cost, was dominant in TNFi-failure scenarios. Golimumab offered the best value in dactylitis and comorbid patients and showed marked improvement under reduced-price conditions. Safety-related discontinuations were infrequent across cohorts.

Conclusions: Ixekizumab, tofacitinib, and golimumab are all cost-effective options for PsA. These findings support phenotype-guided therapeutic decisions and highlight the influence of drug pricing.

Background: This study evaluated the health and productivity impact of expanding the use of inhibitors of programmed cell death protein 1 (PD-1) or its ligand (PD-L1), referred to as anti-PD-(L)1 agents, to early-stage cancer treatment in the Czech Republic.

Research design and methods: A four-state Markov-based model compared the use of anti-PD-(L)1 agents for early-stage disease (ESD) treatment of melanoma (stage IIB-C and III), renal cell carcinoma (RCC), and triple-negative breast cancer (TNBC) versus the established use of anti-PD-(L)1 agents only for metastatic disease in the Czech Republic from 2024-2033. Outcomes included recurrence-/event-/disease-free life-years (LYs), total LYs, quality-adjusted LYs (QALYs), productive work years, events/recurrences, metastatic disease treatments, and deaths.

Results: Of 16,410 eligible patients, 13,745 initiated ESD treatment with anti-PD-(L)1 agents. ESD treatment was estimated to increase recurrence-/event-/disease-free LYs (+10.6%), QALYs (+4.1%), and productive years for both patients (+22.1%) and caregivers (+21.0%), while decreasing events/recurrences (-22.2%), metastatic treatments (-17.6%), and total deaths (-23.2%).

Conclusions: Expanding the use of anti-PD-(L)1 agents for early-stage cancer treatment in the Czech Republic is expected to reduce events/recurrences and metastatic treatments while also extending survival, quality-of-life, and productive work years, supporting the clinical and societal value of earlier use of innovative immunotherapies.

Objective: Providing an analytical framework to evaluate critiques by Canada's Drug Agency (CDA) and to compare the manufacturers' economic analyses versus CDA's reanalyses for oncology medications.

Methods: We reviewed CDA oncology reports from 2018 to 2025. Clinical data and methodological critiques were compared between submissions receiving positive versus negative recommendations.

Results: Positive recommendations were significantly more frequent in submissions that included randomized trials (90.1% vs. 61.9%), trials with a control arm (90.8% vs. 65.4%), and trials incorporating phase III or phase IV evidence (79.6% vs. 41.9%) (p < 0.001). Although CDA was generally receptive to indirect treatment comparisons (ITC), studies using naïve ITC (73.0% for naïve ITC vs. 88.1% for others, p = 0.031) or matching-adjusted indirect comparison (MAIC) (75.0% for MAIC vs. 91.2% for other ITCs, p = 0.009) had significantly lower rates of positive recommendations. When comparing submissions with positive versus negative recommendations, certain critiques were significantly more frequent in the latter. These included not having enough evidence available (82.2% vs. 100%, p = 0.011), and using surrogate outcomes (23.9% vs. 51.6%, p = 0.002).

Conclusion: Using MAIC or naïve ITC, submitting a dossier that includes limited clinical evidence, and surrogate outcomes are associated with a negative recommendation.

Background: Little is known about the healthcare burden of Chronic liver disease (CLD) post-introduction of newer antiviral therapies for hepatitis. Therefore, this national study examined healthcare expenditures, productivity loss, and health-related quality of life (HRQoL) of patients with CLD.

Methods: This retrospective, cross-sectional analysis utilized the 2014-2021 Medical Expenditure Panel Survey involving patients with CLD ≥18 years. Two-part model was employed to estimate incremental direct medical costs. Multivariable models were used to evaluate missed workdays and HRQoL.

Results: Annually, 1.63 million patients (95%CI=1.47-1.81) were diagnosed with CLD, with a prevalence of 0.59%. The mean annual incremental healthcare expenditure attributable to CLD was $11,711 (95%CI=$6,867.048-$16,555.36). Patients with CLD have 0.12 (95%CI=0.09-0.15) more missed workdays, translating into a national productivity loss of $51.6 million. Additionally, HRQoL was significantly lower in CLD patients, with mean differences of -3.72 (95%CI = -4.6 to -2.84) for physical component summary score and -1.83 (95%CI = -2.68 to -0.97) fore mental component summary score.

Conclusions: CLD was associated with a significant healthcare burden, as evidenced by healthcare expenditure, productivity loss, and HRQoL. Therefore, concerted efforts are needed to improve the prevention and management of CLD to reduce the disease burden.

Background: Depression imposes a substantial clinical and economic burden, and evaluating the cost-effectiveness of prevention and treatment strategies is essential for informed mental health policy. The original DepMod model was developed to support such evaluations, but evolving evidence and modeling standards have created the need for an updated version.

Research design and methods: This study introduces DepMod 2.0, an updated health-economic Markov model for depression. The model was recalibrated using recent epidemiological data on incidence, prevalence, population size, and severity distribution. In addition, DepMod was transitioned from an Excel-based implementation to an open-source R package, providing both a stand-alone R function and an interactive Shiny interface. Model validation includes assessment using the TECH-VER checklist, validation against population-level reference parameters, and comparison with outcomes from the original DepMod model.

Results: DepMod 2.0 produced outcomes consistent with published reference values and the original model, supporting its technical validity. Differences observed in prevention scenarios, were attributable to updated epidemiological inputs, including higher prevalence and a greater proportion of severe cases.

Conclusions: DepMod 2.0 is an updated and validated R-based model that supports economic evaluation of depression prevention and treatment strategies across diverse healthcare settings.

Introduction: A patent foramen ovale (PFO) closure reduces the risk of recurrent cryptogenic ischemic strokes in young to middle-aged patients. We aimed to review the studies on the cost-effectiveness of percutaneous closure of a PFO in comparison to medical therapy in the prevention of cryptogenic strokes in patients with a PFO.

Methods: This systematic review searched PubMed, EMBASE, Web of Science, Cochrane, and Scopus from 1990 to 2025.

Results: Twelve articles qualified for inclusion. Closed PFOs resulted in a quality-of-life gain of 3.25 to 4.55 years for most countries. According to the study, the incremental cost-effectiveness ratio per year of life gained was between $3,882.54 and $248,590. Percutaneous closure of the PFO has been found to be cost-effective compared to medical therapy in most countries, with the exception of Argentina.

Conclusions: On the basis of the results of the present study, percutaneous closure of the PFO was cost-effective compared to medical therapy in preventing a cryptogenic stroke in patients with a PFO. A majority of studies were conducted in countries with high incomes. Further studies on the cost-effectiveness of percutaneous closure of the PFO are required in low- and middle-income countries.

Registration: PROSPERO (CRD42024544835).

Background: Epidermal growth factor receptor (EGFR) mutations represent the primary driver alterations in advanced non-small cell lung cancer (NSCLC). Rezivertinib has emerged as a promising targeted therapy for EGFR-mutated NSCLC. This study aimed to evaluate the cost-effectiveness of rezivertinib versus gefitinib as first-line treatment in Chinese patients with EGFR-mutated advanced NSCLC.

Research design and methods: A cost-effectiveness analysis was conducted using a 10-year Markov model based on data from the REZOR clinical trial (NCT03866499). Primary outcomes included total costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs).

Results: Rezivertinib demonstrated superior clinical outcomes with a mean QALY of 2.14 compared to 1.79 for gefitinib. However, rezivertinib incurred higher treatment costs ($43390.34 vs $31202.03). The ICER of $35,215.97 per QALY for rezivertinib falls below the willingness-to-pay (WTP) threshold ($40334, three times the per capita GDP), confirming its cost-effectiveness. Sensitivity analysis revealed that rezivertinib had a 71.5% probability of being cost-effective versus 28.5% for gefitinib. Threshold analysis established cost-effective pricing thresholds for rezivertinib ($605.02 at 3×GDP per capita, $408.66 at 1.5×GDP per capita).

Conclusions: Rezivertinib demonstrates significantly better cost-effectiveness compared to gefitinib for first-line treatment of EGFR-mutated advanced NSCLC in China.

Introduction: The pharmaceutical industry has delivered innovative therapies that substantially improved health outcomes. However, concerns persist regarding high drug prices and delays in the entry of competitors. This article provides a comprehensive and transdisciplinary overview of the pharmaceutical market. References were identified and selected from Medline, Lilacs, Embase, and Google Scholar until thematic saturation. Particular attention is devoted to the market for monoclonal antibodies (mAbs), given their high prices and expanding commercial relevance.

Areas covered: The industry is heavily dependent on innovation, with firms investing significant resources in risky R&D and charging high prices for new technologies. Although companies justify elevated prices based on R&D expenditures, additional market dynamics also sustain high pricing. Operating under imperfect competition, pharmaceutical products often exhibit characteristics of merit and credence goods, while practices such as evergreening and reverse payments reinforce market power. Patients' willingness to pay, especially for life-saving treatments, remains high despite limited understanding of expected outcomes due to informational asymmetry.

Expert opinion: Drug prices reflect the interaction of market structure, R&D investment, licensing arrangements, and patient behavior. Biopharmaceuticals entail added complexity and cost. Monopolistic or oligopolistic conditions can result in suboptimal pricing and unmet demand, highlighting the need for stronger regulatory oversight.

Background: Denmark's publicly funded routine HPV vaccination program has included boys born from 2005 onward, leaving earlier birth cohorts of young men potentially unprotected.

Methods: A published deterministic dynamic transmission metapopulation model was adapted to evaluate the impacts of a 3-year male catch-up vaccination program on the cases, deaths, and costs of HPV-associated diseases in Denmark over a 100-year time horizon. Routine gender-neutral HPV vaccination of adolescents with a nonavalent vaccine was modeled with and without a male catch-up program, at 4 catch-up vaccination coverage rates (VCRs) from 40% to 70%.

Results: Adding a temporary catch-up program for men born in 1997-2005 was projected to avert 253 HPV-associated cancer cases and 89 deaths at a VCR of 40%. Increasing coverage to 70% was estimated to avert 359 cases and 128 deaths. Catch-up vaccination may be considered cost-effective at all modeled VCRs, with incremental cost-effectiveness ratios of €35,584-35,755 per quality-adjusted life year compared to routine adolescent vaccination alone.

Conclusions: Expanding Denmark's male catch-up HPV vaccination program to include all men born in 1997-2005 would reduce the burden of HPV-associated cancers and diseases and may represent a cost-effective public health strategy.