Expert Review of Pharmacoeconomics & Outcomes Research最新文献

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: 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.

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.

This Key Paper Evaluation examines a recent modeling study of return-to-work (RTW) interventions comparing inpatient multimodal occupational rehabilitation (I-MORE) with outpatient Acceptance and Commitment Therapy (ACT). The authors link a randomized trial to seven years of registry follow-up and project costs and effects over a 25-year horizon from healthcare and societal perspectives. We commend the clear intervention definitions, transparent multistate-to-Markov approach, and probabilistic analyses. However, the study's headline finding - lifetime dominance of I-MORE when productivity is counted - hinges on structural assumptions: transition intensities are frozen beyond four years, and within-state absence-day differences are held constant thereafter, generating persistent productivity gains even after state prevalences converge. Health-related quality of life is observed only to 14 months and then modeled as state-specific constants, compressing incremental quality-adjusted life years and leaving the healthcare-only incremental cost-effectiveness ratio unfavorable. We argue that lifetime savings should be treated as upper-bound estimates and that decision relevance would be strengthened by waning-effect scenarios, mid-term horizons, and tests of the absorbing disability state. We also highlight the need for diagnosis-by-treatment analyses and stepped-care sequences given the heterogeneous population and fixed-dose ACT comparator. Overall, the paper advances RTW modeling while underscoring how perspective and structure shape long-run conclusions.

Introduction: The risk of recurrent ischemic strokes in young to middle-aged patients who have experienced a recent cryptogenic ischemic stroke is reduced by percutaneous closure of a patent foramen ovale (PFO) in comparison to medical therapy alone. 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. The quality of life gained from closing the PFO varied between 3.25 and 4.55 among countries. Additionally, the results for the incremental cost-effectiveness ratio per year of life gained were reported to be between $3,882.54 and $248,590. Based on the available evidence, the percutaneous closure of the PFO was found to be cost-effective in preventing a cryptogenic stroke in patients with a PFO in most countries, with the exception of Argentina, compared to medical therapy.

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: Multiple myeloma (MM) is a hematologic cancer with rising incidence worldwide. Autologous stem cell transplantation (ASCT) is a key treatment for eligible patients, but mobilization failure remains a major obstacle. Plerixafor enhances stem cell mobilization, but its high costs and lack of standardized protocol prevent its widespread use.

Objective: To evaluate the cost-utility of preemptive versus rescue use of plerixafor for hematopoietic stem cell mobilization inMM patients.

Methods: A Markov model was developed using real-world data from 196 MM patients undergoing stem cell mobilization at two SãoPaulo transplant centers in Brazil. The model compared two strategies: (1) preemptive plerixafor and (2) rescue plerixafor use following mobilization failure. Transition probabilities, utilities, and costs were informed by clinical data and literature. Deterministic and probabilistic sensitivity analyses were performed.

Results: The preemptive strategy led to higher rates of successful mobilization and ASCT, resulting in greater quality-adjusted life years (QALYs), but also higher costs. Still, it demonstrated favorable results compared to the rescue approach and passed the Brazilian willingness to pay thresholds of acceptability.

Conclusion: Preemptive plerixafor passes the cost-utility guidelines to be used in MM patients in Brazil, potentially guiding policy decisions on resource allocation within the national health system.

Background: Lebrikizumab and tralokinumab significantly treat moderate-to-severe atopic dermatitis. Nevertheless, pharmacoeconomic analyses and head-to-head clinical studies for both are lacking. This study aimed to compare the cost per responder of lebrikizumab and tralokinumab in these patients.

Research design and methods: To indirectly compare the efficacy of lebrikizumab and tralokinumab for in the treatment of moderate-to-severe atopic dermatitis based on published clinical trial data. The number needed to treat and the cost per responder in comparison to a placebo were used to compare the cost-effectiveness of the two therapies. The cost per responder was calculated based on United States drug acquisition costs by multiplying the cost of treatment by the number needed to treat for each therapy.

Results: At Week 16, the cost per responder for lebrikizumab and tralokinumab was $67,932 versus $130,655 for EASI-75 and $109,412 versus $176,622 for IGA 0/1.For key secondary endpoints, the cost per responder was consistently lower for lebrikizumab compared to tralokinumab, including 16-week ∆NRS ≥4 ($95,282vs$156,262), and 4-week IGA 0/1 ($68,955vs$151,874).

Conclusions: In comparison to tralokinumab, lebrikizumab had a much lower number needed to treat and cost per responder. These results, based on US pricing, indicate that lebrikizumab appears to be more cost-effective treatment option based on indirect comparisons.

Background: This study evaluates the cost-effectiveness of larotrectinib compared to the standard of care for treating metastatic NTRK fusion colorectal cancer (CRC) from the perspective of healthcare payers in China.

Research design and methods: An economic evaluation utilizing a 3-state partitioned survival model assessed the cost-effectiveness of larotrectinib therapy vs regorafenib therapy or larotrectinib therapy vs trifluridine/tipiracil therapy.

Results: When the time horizon was 10 years, the anticipated expenditure for larotrectinib therapy exceeded the cost for regorafenib therapy or trifluridine/tipiracil therapy (21,588.50 USD vs 1579.09 USD; 21,588.50 USD vs 2411.16 USD). The estimated utility of larotrectinib therapy was also greater compared to that of regorafenib therapy or trifluridine/tipiracil therapy (1.14 QALYs vs 0.28 QALYs; 1.14 QALYs vs 0.29 QALYs). The ICER of larotrectinib therapy vs regorafenib therapy or larotrectinib therapy vs trifluridine/tipiracil therapy was calculated at 23,321.46 USD/QALY or 22,585.39 USD/QALY.

Conclusions: From the perspective of healthcare payers in China, larotrectinib was cost-effective compared to standard of care as a second-line treatment or subsequent treatment for advanced or metastatic CRC patients with NTRK gene fusion-positive.

Background: The review of healthcare costs in multiple sclerosis (MS) has been of increased interest. In this study, we aimed to estimate the economic burden of MS patients, treated with dimethyl fumarate (DMF) in Greece.

Research design and methods: Four hundred and fifty-six participants were followed every 6 months after DMF initiation. Healthcare resource utilization, out-of-pocket expenses, and productivity loss data were recorded. Treatment cost and effectiveness of DMF were compared with those of previous treatments.

Results: From a societal perspective, the total mean biennial burden was 13,113 € per patient, mainly attributed to direct healthcare costs (10,818 €, with medication accounting for almost 96%). From the payer perspective, the cost per patient was estimated at 10,462 €. The main driver of indirect cost was early retirement (87.8%). The treatment cost in the 2 years before the study was ~25% higher than the biennial DMF cost. A total of 43.9% of the patients experienced adverse events, mostly mild/moderate.

Conclusions: DMF displayed a societal cost mainly driven by medication, disease severity, disease duration and patients' age. There was evidence of beneficial effects on disease activity and quality of life with no new safety signals emerging.

Trial registration: The trial is registered at ClinicalTrials.gov (ID: NCT03101735).

Background: Functional genomics approaches, such as transcriptomics and proteomics, can provide valuable insights into rare diseases when genomic sequencing fails to yield informative findings. This study estimated the monetary value that parents, carers and individuals with undiagnosed rare diseases place on functional genomics testing.

Research design and methods: A triple-bounded dichotomous choice contingent valuation survey was completed by carers and individuals with suspected rare monogenic disorders recruited as part of the Australian Undiagnosed Disease Network. A multilevel interval regression model was used to analyze response data and estimate the monetary value of functional genomics, in terms of willingness to pay (WTP).

Results: There was a total of 57 respondents (48%), primarily carers (95%). The mean WTP for functional genomics testing was estimated to be $2,522 (95% CI: $817-$4,228) [US $1,568 (95% CI: $508-$2,629)].

Conclusions: Our findings indicate that individuals with undiagnosed rare diseases and their parents or caregivers place high value on functional genomics testing. The estimated WTP is comparable to findings from contingent valuation studies of other genomic interventions and exceeds the expected economic cost of proteomics testing. These insights can inform a preference-based evaluation of the diagnostic outcomes and net benefits achieved through functional genomics, thereby guiding decision-making and clinical implementation.