Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Introduction: The integration of digital endpoints into Health Technology Assessment (HTA) marks a significant advancement in modern healthcare evaluation, especially in the context of post-pandemic growth in telehealth and remote patient monitoring.

Areas covered: Digital endpoints, which are defined by their use of sensor-generated data collected in non-clinical settings, provide a comprehensive, real-time view of patient health. This enhances the precision of HTA by uncovering nuanced aspects of disease burden and improving the evaluation of health technologies. Examples of digital endpoints include smartphone-based diagnostics for cognitive impairment and wearable devices that measure the impact of diseases, such as nocturnal activity in patients with sickle cell disease. Earlier diagnoses, and cost reductions in drug discovery, their integration into HTA faces challenges- data privacy, standardization, and methodological validation.

Expert opinion: This paper explores the potential of digital endpoints to revolutionize HTA by enabling more dynamic and patient-centered evaluations, underscoring the need for established frameworks and standards to guide their effective incorporation. Initiatives such as the Digi-HTA process and the Digital Endpoints Ecosystem and Protocols (DEEP) highlight emerging frameworks that could shape the future of digital health assessments, ultimately enhancing healthcare decision-making and policy. Collaborative efforts across healthcare, technology, and regulatory bodies are essential to overcome these barriers.

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.

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.

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: 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.

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: This inquiry assesses the willingness to pay (WTP) for a COVID-19 vaccination in Vietnam, and uses a mixed-method approach to investigate how economic, social, and health factors influence acceptance of a vaccine and payment preferences.

Methods: The research agenda was implemented via a Discrete Choice Experiment (DCE) with 685 Vietnamese respondents aged 18 years and older from June to September 2024, to measure preferences based on vaccine attributes such as cost, safety, side effects, and duration of protection. WTP was estimated using Binary Logistic Regression. In addition, semi-structured interviews were carried out with 26 community members to explore perceptions, beliefs, and trust in government health messages.

Results: Quantitative findings indicated that increased WTP was significantly associated with income, education, and perceived vaccine safety. The qualitative findings also raised concerns regarding vaccine side effects and trust in public health communications. Citizens who are higher in socioeconomic status are more likely to accept and pay for the vaccine.

Conclusions: The study shows that there are behavioral as well as economic factors influencing vaccinations. The findings may inform targeted strategies to increase vaccine acceptance among poor communities. Limitations include a cross-sectional design and self-reported attitudes, which may be subject to recall bias.

Background: Cystoscopy is a high-frequency endoscopic procedure requiring sustainable and evidence-based device management strategies.

Research design and methods: This institutional cost-opportunity analysis compared disposable and reusable cystoscope devices in a high-volume oncology center. Retrospective real-world data on disposable use were combined with theoretical modeling for reusable systems under three operational scenarios: disposable, reusable with standard sterilization, and reusable with urgent sterilization, each analyzed in optimized and maximal-cost conditions.

Results: Disposable cystoscopes cost €188.75 per procedure, halved procedural time (15 vs. 30 minutes), and ensured workflow continuity. Reusable systems were economically sustainable only under optimized reprocessing (two cystoscopes per cycle, €180.88 per procedure). Under urgent or single-scope sterilization, per-procedure costs increased to €198.88, with annual excesses of up to €79,820.88 compared to disposable systems. Disposable devices minimized downtime and logistical complexity, particularly in high-demand settings.

Conclusions: Reusable cystoscopes achieve economic sustainability only when supported by standardized, high-efficiency workflows, while disposables provide flexibility and reliability in routine and urgent care. Despite its single-center, retrospective design, this framework provides transferable insights for healthcare institutions aiming to align cost management, service quality, and sustainability in cystoscopic practice. Future multicentric studies are warranted to validate these findings across diverse organizational settings.

Background: One of the novel methods of blood glucose monitoring is continuous glucose monitoring (CGM). This study aims to evaluate the cost-effectiveness of CGM versus self-monitoring of blood glucose (SMBG) in patients with type 2 diabetes (T2D) in Iran.

Methods: A cost-effectiveness analysis with a lifetime horizon was conducted to compare the two monitoring strategies. The patient-level United Kingdom Prospective Diabetes Study (UKPDS) Outcomes Model was employed, and clinical and demographic data from Iranian patients enrolled in the Diabetes Care study were extracted for the analysis. Cost-effectiveness was evaluated by calculating incremental cost-effectiveness ratios (ICERs), expressed as cost per QALY gained. Uncertainty in the model was addressed through probabilistic sensitivity analysis using 100 bootstrap iterations.

Results: The cost-effectiveness analysis showed that the expected cost of CGM compared to SMBG was $40,444.2. The incremental gain in QALYs was 0.096, resulting in an ICER of $397,644.7 per QALY gained. Based on bootstrap analysis, the 95% CI for the ICER ranged from $271,157.3 to $600,185.2.

Conclusion: CGM does not represent a cost-effective alternative to SMBG for patients with T2D in Iran. This finding was supported by bootstrap analysis, which demonstrated a lack of cost-effectiveness across all simulated scenarios.

Background: Low back pain (LBP) is a disabling condition affecting all age groups globally. Exercise is safe and cost-effective for managing LBP. Pilates is an effective exercise modality recommended for LBP but is not reimbursed by the Brazilian Public Health System. This study aimed to estimate the budget impact of implementing Pilates for LBP patients within the Brazilian public health system compared to usual care.

Research design and methods: A budget impact analysis (BIA) was conducted over a five-year horizon, from healthcare and societal perspectives. Costs were extracted in local currency and converted to international dollars using purchasing power parities. Deterministic sensitivity analyses were performed.

Results: Over five years, healthcare costs in the usual care were Int$860.8 million, while Pilates reached Int$958 million, resulting in an incremental impact of Int$97.2 million. From the societal perspective, the usual care cost was Int$4.32 billion, and the Pilates scenario was Int$4.15 billion, indicating savings of Int$172.2 million. The incremental budget impact was negative, meaning that Pilates would provide savings of Int$172,203,216.

Conclusion: Implementing a group-based Pilates program for LBP in the Brazilian public health system would increase healthcare costs by Int$97 million but generate societal savings of Int$172 million over five years.