Expert Review of Pharmacoeconomics & Outcomes Research最新文献

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.

Background: Patient-reported outcome (PRO) data capture patients' perspectives on their disease and its treatments; however, use of such data in health technology assessments (HTA) and pricing/reimbursement processes is limited. We provide considerations to support the incorporation of PROs in payer decision-making.

Research design and methods: A review of guidance documents released by payers until 2024 regarding requirements for using PRO evidence and scientific literature published during 2015-2024 on the trends of PRO use by payers informed the development of a discussion guide. Using this, 15 experts from seven countries were interviewed (one-on-one) to seek their opinions on PRO use in HTAs and identify potential barriers to its adoption.

Results: PRO-related guidance focused on the validity and reliability of PRO instruments, risk for bias, missing data, and economic modeling. Guidance varied between countries with essential details often missing. Expert interviews revealed that PRO use in payer evaluations may depend on established endpoints for indications and varied decision approaches and frameworks. Barriers to PRO use include the lack of capacity or technical expertise, instrument validity concerns, and data accuracy.

Conclusion: Barriers to the use of PRO data for evaluations require further efforts from all relevant stakeholders to promote PRO incorporation in payer decision-making.

Background: The long-term impact of preventive policies in the Netherlands on the mental health of young adults remains unclear. Therefore, this paper describes the development of a conceptual model of youth mental health that serves as the foundation of a future decision-analytic model.

Research design and methods: Stakeholders were engaged through three rounds of focus group discussions to indicate the factors of youth mental health that affect the likelihood of developing mental disorders later in life and the relationships among them. Findings were discussed with stakeholders and in a study team that included members with diverse backgrounds. Literature was used as an additional information source for the relationships among the selected factors.

Results: In total, 43 stakeholders participated in the focus group discussions. Eleven factors of youth mental health were regarded as most influential, with 13 relationships among them. The final conceptual model was approved by the stakeholders and the study team.

Conclusions: Through integrating stakeholder perspectives and published literature, a conceptual model was created that captures essential factors and relationships affecting (long-term) mental health. Although stakeholder engagement requires extensive planning, it enhanced the model's credibility and validity, and could therefore serve as a complement to other conceptual modeling approaches.

Background: Previous research demonstrated the effectiveness of an add-on module in reducing intrusive suicidal mental images in patients with elevated depression symptoms indicative of a depressive disorder. This study assessed the cost-effectiveness of combining this module with treatment as usual (TAU) versus TAU-alone.

Research design and methods: A randomized controlled trial of 91 participants (46 add-on+TAU, 45 TAU). Cost-effectiveness was assessed over 12 months using quality-adjusted life years (QALYs) as the primary outcome. Healthcare, patient, and work-related costs were measured. Health-related quality of life was assessed using EQ-5D-5 L at baseline, and after 3, 6, 9, and 12 months.

Results: The add-on+TAU group showed slightly higher QALYs (0.674) than TAU-group (0.630) after 12 months. From a societal perspective, the intervention yielded an incremental cost-effectiveness ratio of €137,285 per QALY gained (95% CI: -€7,338 to €15,656; QALY gain: 0.0274). Healthcare costs were higher in the add-on+TAU group, primarily due to increased outpatient mental care costs (including intervention costs of €524.02/participant). The intervention showed a 35% probability of cost-effectiveness at an €80,000 willingness-to-pay threshold.

Conclusions: Although the add-on module reduced suicidal mental images, its cost-effectiveness remains uncertain. Modest QALY improvements did not justify increased healthcare and societal costs. Larger-scale studies with extended follow-up are needed.

Clinical trial registration: The trial was prospectively registered in the Netherlands Trial Registry (No. NTR7563).

Introduction: This study aims to conduct a systematic review of cost-effectiveness studies evaluating later-line treatments for refractory metastatic colorectal cancer.

Methods: This study conducted a literature search using PubMed, Cochrane, and Embase databases. Search terms included keywords related to colorectal cancer, regorafenib, trifluridine-tipiracil, fruquintinib, and cost-effectiveness analysis. Extracted data encompassed cost-effectiveness outcomes, methodological characteristics, and the seven parameters exerting the greatest influence on incremental cost-effectiveness ratio (ICER) results.

Results: Among 183 studies screened, a total of 11 studies met the inclusion criteria. Most studies concluded that later-line treatments for refractory metastatic colorectal cancer were not cost-effective compared to best supportive care (BSC). However, trifluridine-tipiracil was found to be cost-effective compared to BSC in some studies, and several studies reported it as a dominant option over regorafenib. Nonetheless, the cost-effectiveness outcomes between trifluridine-tipiracil and regorafenib were inconsistent across studies. Overall, drug costs and health utility values in the progressed disease state were identified as having the greatest impact on ICER outcomes.

Conclusion: Later-line treatments were generally not found to be cost-effective compared to BSC, mainly due to high drug costs. No treatment showed consistently favorable results across studies, with outcomes varying by comparator, country, and model settings.

Protocol registration: PROSPERO (CRD420251243616).

Background: Chronic obstructive pulmonary disease (COPD) imposes a substantial clinical and economic burden in Colombia, particularly among older adults. Despite widespread use of dual therapy, many patients remain at high risk of exacerbations. Our objective was to evaluate the long-term cost-effectiveness of single-inhaler triple therapy (FF/UMEC/VI) versus dual therapy (FF/VI) in patients with moderate-to-severe COPD in Colombia.

Research design and methods: A Markov model was developed to simulate disease progression, healthcare costs, and quality-adjusted life years (QALYs) over a lifetime horizon, from the perspective of the Colombian healthcare system. Clinical inputs were based on randomized controlled trial data, and cost/resource use data were derived from national sources. Deterministic and probabilistic sensitivity analyses were performed.

Results: FF/UMEC/VI provided an additional 2.34 QALYs at an incremental cost of $3,013 USD versus FF/VI, resulting in an incremental cost-effectiveness ratio (ICER) of $1,287 per QALY, below the national willingness-to-pay threshold of $5,180 per QALY. Sensitivity analyses confirmed the robustness of results, with FF/UMEC/VI remaining cost-effective in >60% of simulations.

Conclusions: FF/UMEC/VI is a cost-effective strategy for managing moderate-to-severe COPD in Colombia, offering improved clinical outcomes at an acceptable cost and supporting its adoption in treatment guidelines and reimbursement decisions.

Objective: To evaluate the cost-effectiveness of tisotumab vedotin (TV) versus single-agent chemotherapy in recurrent or metastatic cervical cancer (r/mCC) after failure of first-line treatment, from the perspective of the Spanish National Health System.

Methods: Cost-utility analysis was conducted using a partitioned survival model with three health states, over a 60-month time horizon. Efficacy data ‒overall survival (OS) and progression free survival‒ were derived from innovaTV-301 trial and modeled using parametric survival functions. Costs and utilities were obtained from national databases and literature, with outcomes expressed in quality-adjusted life years (QALYs). Incremental cost-utility ratio (ICUR) was calculated, and sensitivity analyses were performed to explore uncertainty.

Results: TV provided an additional 0.341 QALYs over chemotherapy (1.229 vs. 0.889) at an incremental cost of €12,138, resulting in an ICUR of €35,600/QALY, assuming a cost equivalent to cemiplimab. Sensitivity analyses showed ICURs ranging from €33,483-€37,564/QALY depending on modeling assumptions. Drug price and OS benefit were the most influential parameters. Using the foreign list price for TV, the ICUR increased to €950,017/QALY.

Conclusions: TV offers a modest benefit over chemotherapy in r/mCC and reaches or surpasses the upper bounds of ICER applied in Spain when priced comparably to cemiplimab. The magnitude of clinical benefit remains uncertain.

Background: Given the potential of artificial intelligence (AI) and the increasing importance of understanding AI's economic impact, this study aims to provide insights into the potential cost-effectiveness of an AI tool in the response prediction to neoadjuvant chemoradiotherapy (nCRT) of Stage II-III LARC patients in comparison to usual care (UC).

Methods: This study included a state-transition Markov model from a Dutch societal perspective. Quality-adjusted life years (QALY) and costs were simulated over a 10-year horizon. Sensitivity analyses and a threshold analysis were performed. Results were presented as incremental cost-effectiveness ratios.

Results: With incremental cost savings of -€2,530,000 per QALY gained per 1000 patients, the AI is dominant in the base-case. Main drivers of cost-effectiveness were the clinical complete response incidence and specificity of the tool. Cost-effectiveness was maintained at a cost of €1,100 and €2,100 for an AI performance of 0.85 and 0.90.

Discussion: Findings of this study present the economic impact of a hypothetical AI-based approach to treatment response prediction in Stage II-III LARC patients who receive nCRT and are eligible for consecutive surgery. The results of this study highlight the complexity of healthcare decision-making in tools that could be cost-saving yet yield lower effectiveness when parameters are uncertain.

Introduction: Mechanical thrombectomy (MT) is a proven treatment for acute ischemic stroke (AIS), yet its cost-effectiveness across global settings remains unclear. This systematic review synthesizes cost-effectiveness evidence and introduces a framework for evaluating MT across different health systems.

Methods: Following PRISMA 2020 guidelines, comprehensive searches were conducted across four major databases to identify full economic evaluations comparing MT with intravenous thrombolysis (IVT). Methodological quality was assessed using the Quality of Health Economic Studies (QHES) checklist, and findings were synthesized qualitatively. Incremental cost-effectiveness ratios (ICERs) were converted to 2024 US dollars. Studies were categorized by income level, modeling approach, perspective, and real-world vs. trial-based data.

Results: Of 4550 records screened, 56 met the inclusion criteria. The mean QHES score was 0.83, indicating high quality. MT was consistently cost-effective or dominant across diverse healthcare contexts. Sensitivity analyses indicated that ICER variability was primarily influenced by device costs, patient age, time horizon, acute care costs, and input data used for functional outcomes and utility weights.

Conclusion: MT is a high-value intervention, but its cost-effectiveness is context-dependent. Our classification framework supports localized decision-making. Future research should address geographic inequities in existing economic evaluations and expand evidence from low-resource settings and also integrate real-world data.

Registration: PROSPERO (CRD42024496552).

Background: Patients with overweight or obesity (OW/OB) are at increased risk for multimorbidity (≥2 obesity-related complications [ORCs]) and accompanying increases in mortality and excess costs.

Research design and methods: A retrospective, observational cohort study using the Komodo Health claims database assessed healthcare resource utilization and medical costs in patients with OW/OB and multimorbidity who received semaglutide compared with propensity-score matched obesity medication non-users (controls).

Results: Patients taking semaglutide (mean follow-up 101 days) had 27% lower all-cause total medical costs ($891 vs $1,213 per patient per month [PPPM]), 59% lower inpatient costs ($115 vs $283) and 18% lower outpatient costs ($746 vs $906) vs. controls (all p < 0.0001). ORC-related total medical costs were 36% lower ($522 vs. $812 PPPM), inpatient costs were 59% lower ($107 vs $259) and outpatient costs were 26% lower ($399 vs $540) among patients taking semaglutide than controls (all p < 0.0001). Use of semaglutide was associated with significant improvements in almost all cardiometabolic markers assessed from baseline to Weeks 52 and 104.

Conclusions: Patients treated with semaglutide had lower all-cause and ORC-related total medical costs than non-users, with a yearly reduction of $3,870 and $3,482, respectively. Cost reductions were driven by significantly lower inpatient hospitalization rates and costs.