Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Introduction: Hip fractures represent a major global health burden, associated with substantial morbidity, mortality, and economic costs, particularly among older adults. With population aging accelerating worldwide, understanding of the epidemiology, cost drivers, and cost-effectiveness of prevention and management strategies is essential for informed resource allocation.

Areas covered: This review summarizes the global burden and costs of hip fractures, with emphasis on high-risk groups. We searched the literature in MEDLINE (via Ovid), ScienceDirect, Scopus, PubMed, and Google Scholar. Data from the 2023 Global Burden of Disease study indicate 24.3 million new hip fracture cases globally. Burden and costs vary widely across regions, influenced by demographic trends, healthcare access, and system capacity. The review evaluates the cost-effectiveness of pharmacologic and non-pharmacologic interventions, along with broader health system approaches aimed at reducing incidence, improving treatment pathways, and preventing secondary fractures.

Expert opinion: Reducing the growing burden of hip fractures requires a multifaceted approach. While cost-effective interventions exist, their real-world implementation remains uneven. Future priorities include closing care gaps through systematic models, enhancing medication adherence, and tailoring strategies for low-resource settings. Emerging opportunities, such as digital health tools may improve risk stratification and personalize prevention, supporting more sustainable and equitable hip fracture care globally.

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.

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.

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.

Objective: To evaluate the effects of standardized pharmaceutical service (SPS) on medication adherence, anxiety, and depression in patients with chronic obstructive pulmonary disease (COPD).

Research design and methods: A prospective, randomized controlled study design was adopted. 184 COPD patients hospitalized at the First Hospital of Putian City between March and August 2023 were randomly assigned to a control group receiving routine care or observation group receiving SPS provided by clinical pharmacists. SPS included health records establishment, education, medication guidance, psychological support and medication simplification. Patients were followed for 6 months. Eight-Item Morisky Medication Adherence Scale (MMAS-8), the Self-Rating Anxiety Scale (SAS), and the Self-Rating Depression Scale (SDS) were used to assess outcomes.

Results: At 3- and 6-month, the observation group had higher MMAS-8 scores (P < 0.05) and a greater of good-to-moderate adherence at 6 months (66.30% vs.35.87%; P < 0.01). Moderate-to-severe anxiety and depression rates were significantly lower in the observation group (53.26% and 54.35%) than in controls (P < 0.001). Fewer adverse events were recorded in the observation group (3 vs. 8), though not statistically significant.

Conclusion: SPS enhance medication adherence and reduce anxiety and depression in COPD patients, supporting its integration into routine COPD management.