ClinicoEconomics and Outcomes Research最新文献

Background: Saudi Arabia is reforming its healthcare system to promote the use of generic and biosimilar medications to improve spending efficiency. However, concerns regarding therapeutic equivalence persist. This study aims to establish a stakeholder-driven, value-based procurement framework for off-patent pharmaceuticals (OPPs).

Methods: The Saudi chapter of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR) convened stakeholders to define procurement criteria. Participants reviewed 29 potential criteria during a workshop, followed by a multi-criteria decision analysis (MCDA) simple scoring exercise and conjoint analysis to determine the relative weight of essential factors.

Results: Forty-nine stakeholders participated, primarily pharmacists (57%) and regulators (27%). Sixteen criteria were identified as important: published effectiveness data, drug delivery systems, bioequivalence, real-world data generation, drug stability, direct costs, supply track record, labeled indications, drug safety profile, quality assurance of production, interchangeability, contribution to national healthcare priorities, availability in reference countries, manufacturing site quality certification, pharmaceutical equivalence, and pharmacovigilance. Effectiveness data received the highest weight (11.56%), while pharmacovigilance received the lowest (3.23%).

Conclusion: This pilot study establishes the first consensus-based MCDA criteria for Saudi Arabia, prioritizing effectiveness and drug stability over costs. It emphasizes scientific validity over price, serving as a foundation for a national value-based procurement system, though further research is needed to test the framework in healthcare tenders.

Introduction: Prescription drugs are excluded from Canada's federal legislation covering health care. Each provincial government has developed its own drug plan. To get new prescription medicines listed in these plans, developers must pass regulatory review, health technology assessment and price negotiation, and convince individual government plans to list their drugs. The objective of this research is to assess how many reimbursement recommendations issued by Canada's Drug Agency (CDA) have clinical and/or price conditions and what the consequences are.

Methods: Data were obtained on drugs with CDA recommendations issued between January 2020 and December 2024, together with dates of price negotiations between the pan-Canadian Pharmaceutical Alliance (pCPA) and manufacturers by the end of July 2025 and listings in government plans relating to the same drugs by early November 2025.

Results: Of 344 CDA recommendations, only three (0.9%) were unconditional reimbursement, 291 (84.6%) reimbursement with clinical criteria and/or a price condition, and 50 (14.5%) no reimbursement. Median time for CDA reviews was 221 days (interquartile range (IQR): 199-282 days). Where recommended to achieve cost-effectiveness of $50,000/quality-adjusted life-year, median reduction was 74.5% (IQR: 50.0%-90.0%). Median time for the pCPA to decide whether to negotiate was 128 days (IQR: 73-191 days) and median negotiation time was 131 days (IQR: 82-219 days). The median time between submission to CDA and pCPA outcome was 518 days (IQR: 394-633 days). Government drug plan listing rates for drugs successfully negotiated with the pCPA ranged from 58.6% to 91.6%. Five patients had prior-authorization requests to a private insurer for costly drugs denied because the drugs had conditional CDA recommendations.

Conclusion: CDA and pCPA processes take considerable time and listing decisions by government drug plans add extra time before potential access by patients. Nearly all CDA reimbursement recommendations, which are intended for government drug plans (not private payers), are conditional.

Purpose: This study provides one of the few national comparisons of antineoplastic expenditures from 2010 to 2021, offering new insight into how spending patterns have evolved over the past decade. In addition, it examines how out-of-pocket expenses relate to patient quality of life, an essential domain to evaluate healthcare interventions.

Patients and methods: This study provides an updated estimate of the costs of antineoplastic agents using 2021 data and compares them with estimates from 2010. Medical Expenditures Panel Survey (MEPS) files were analyzed for national estimates. Antineoplastic treatment was defined in the MEPS using Multum Lexicon variables from the Cerner Multum. All reported prescriptions and refills were included in the expense and usage estimates. The 2010 costs were adjusted to 2021 figures using the consumer price index for out-of-pocket expenses and gross domestic products for third-party payers. SAS Studio 3.81 (Enterprise Edition) was employed for analysis.

Results: Between 2010 and 2021, cancer diagnoses in the United States rose by 20.46%, while patients getting antineoplastic therapy increased by 7.6%. During this time, the cost of these medicines increased by thrice, from $9.78 billion to $35.12 billion. Prescription numbers were steady, with an average of four per patient per year. Males, older patients, and the insured were more likely to use the service. Breast cancer was the most common, with prostate and skin cancers increasing. The average prescription expenditure for cancer patients increased significantly compared to non-cancer individuals. Finally, there was no significant relationship observed between patients' quality of life and their out-of-pocket expenses.

Conclusion: The substantial increase in cancer treatment expenses had no positive impact on patients' physical or mental well-being. This cost-quality gap necessitates a study on spending efficiency and patient well-being.

Introduction: In the treatment of attention-deficit/hyperactivity disorder (ADHD), supplemental use of immediate-release (IR) stimulants can lead to fluctuating plasma levels that impede symptom control and are more prone to diversion. The negative implications of IR stimulant supplementation are broad, leading to extra costs and harm to individuals and society. To quantify the economic impact of IR supplementation from a United States societal perspective, this study evaluated the incremental cost difference between Dyanavel XR and other extended-release (ER) stimulants over a one-year base-case horizon and summarized value using a benefit-cost ratio.

Methods: As a hypothesis-generating approach, a decision-tree model compared these interventions across direct medical, non-medical, and indirect costs. Analysis was conducted for the general ADHD population and stratified by age group to account for differences in medication use, adherence, and costs. Suboptimal response combined IR supplementation and patient-reported end-of-dose crash, with all inputs drawn from the published literature.

Results: The average per-person cost for Dyanavel XR was $43,219 versus $51,071 for other ER stimulants, resulting in savings of $7,852 per person over one year. Nationally, Dyanavel XR resulted in $44.6 billion in aggregate savings. The benefit-cost ratio of Dyanavel XR was 12.59, indicating that benefits outweighed the treatment cost; 16.89 in young adults and 0.89 in children. In one-way deterministic sensitivity analysis, Dyanavel XR remained cost-saving, highlighting the robustness of the economic benefit.

Conclusion: From a United States societal perspective, Dyanavel XR demonstrates an economic advantage over other ER stimulants, with value most pronounced in young adults and lower in children. Dyanavel XR's potential to reduce IR supplementation and end-of-dose crash, thereby mitigating downstream costs, makes it a compelling ADHD treatment option. The model's operational definition of suboptimal response is intended as a pragmatic framework for future research to test its predictive validity for clinical outcomes and quality-of-life measures.

Background: Acute kidney injury (AKI) is common among patients admitted to the intensive care unit (ICU), with 5-15% receiving renal replacement therapy (RRT). Continuous renal replacement therapy (CRRT) and intermittent hemodialysis (IHD) are well-established treatments for severe AKI, but renal recovery is variable and often incomplete, leading to long-term morbidity and mortality. The clinical and cost-effectiveness of either therapy are under active debate. This analysis aims to strengthen the evidence on the cost-utility of CRRT compared with IHD to manage severe AKI in ICU using a US third-party costing perspective.

Methods: The analysis used a 90-day decision tree simulating hospital admission and a semi-Markov process with annual cycles and half-cycle correction to capture lifetime costs and outcomes, discounted at 3% annually. Survivors at 90 days either progressed to ESKD on dialysis (ESKD-D), with some receiving transplants, or became dialysis-independent. In the case of transplant failure, patients returned to ESKD-D. Tunnel states addressed Markov memoryless properties. A US-representative analysis of real-world data applying propensity score matching to control for selection bias informed the probability of lifetime dialysis dependence. Costs and utilities were sourced from peer-reviewed publications or national data. Uncertainty was investigated using deterministic and probabilistic sensitivity analyses.

Results: In the base case, lifetime costs and quality-adjusted life-years (QALYs) were $273,314 and 5.681 for CRRT compared to $268,449 and 5.457 for IHD. CRRT had an 89.6% probability of being cost-effective ($23,860/QALY gained), being associated with 0.269 additional life-years. Long-term CKD management costs, accounting for 50% of CRRT's excess costs, significantly influenced results and were examined in scenario analyses.

Conclusion: CRRT is likely a cost-effective option for managing severe AKI in the ICU compared with IHD. This study builds on existing economic evaluations by incorporating large comparative studies and exploring clinical uncertainty. The model highlights the need to clarify RRT's role in CKD progression and enhance post-AKI care to improve patient outcomes.

Purpose: To assess the cost-effectiveness of biologics in patients with axial spondyloarthritis (axSpA) in a real-world setting.

Patients and methods: This is a non-interventional, registry-based, prospective cohort study included 203 consecutive patients with axSpA who attended the Rheumatology Department of a public hospital in the Emirate of Dubai between July 2018 and September 2020. Demographic and clinical data were collected and disease activity and treatment response were assessed. Patients were grouped according to the treatment received, distinguishing between biologics and non-biologics, and classified them based on their disease activity at baseline and 52 and 104 weeks. The direct and indirect costs associated with their management were collected. The incremental cost-effectiveness ratio (ICER) was calculated and the impact of patient-related factors on its value across subgroups was examined. A state-transition model was used to simulate disease progression across four health states over a lifetime (62 years), and Monte Carlo simulation was applied to address uncertainty.

Results: The total cost of managing the patients was AED 27,532,189, resulting in a gain of 293.7 quality-adjusted life years (QALYs) over a 2-year follow-up period. Biological therapies were associated with higher direct costs, accounting for 83.3% of the total costs of biologics. The overall ICER was AED 253,616 per QALY, influenced by higher medication acquisition costs. Patients with < 5 years of disease duration, female patients, those with radiographic-axSpA, co-existing fibromyalgia, and those receiving a combination of biologic treatments and conventional disease-modifying antirheumatic drugs showed a higher ICER than the median ICER of all biologic therapies.

Conclusion: Biologic therapies are cost-effective for patients with axSpA, especially those not achieving clinical targets. Patients' selection, and targeted cost-containment strategies, such as biosimilars and medication price reductions, can enhance clinical benefits and reduce societal costs.

Background and objectives: Global demand for health economics/pharmacoeconomics (HE/PE) has significantly increased, leading to a greater need for competent and skilled healthcare professionals in this field. However, the current literature indicates a lack of research regarding HE/PE among healthcare professionals, including those in the Kingdom of Saudi Arabia (KSA), particularly among registered nurses. Hence, this study assessed registered nurses' levels of awareness and knowledge of HE/PE and their sociodemographic and HE/PE-related predictors.

Methods: A descriptive-correlational, cross-sectional design was used. The study was conducted on a convenience sample of 324 registered nurses working at the three hospitals within King Saud University Medical City, Riyadh, KSA. Data were collected from May 2025 to June 2025, and analyzed using SPSS v.30. Binary logistic regression analyses were conducted for HE/PE awareness, and multiple linear regression analyses were performed for knowledge about HE/PE. The significance level was established at p <0.05.

Results: Registered nurses demonstrated an overall low level of knowledge (2.39/5), while showing a good level awareness about HE/PE across four awareness items (52.8% to 84.6%) but suboptimal for the remaining two items (38.6% and 38.9%). Several sociodemographic and HE/PE-related factors were identified as significant predictors of the registered nurses' awareness and knowledge regarding HE/PE. Notably, four out of the six awareness items: "have heard about HE/PE" (p =0.040), "read articles related to HE/PE regularly" (p <0.001), "participate in HE/PE decisions at work" (p <0.042), and "implement HE/PE techniques to make decisions" (p <0.001) were revealed as significant predictors of registered nurses' knowledge about HE/PE.

Conclusion: The findings indicate that the knowledge level regarding HE/PE among registered nurses was low; nevertheless, their awareness was acceptable. It is crucial to develop and implement targeted educational strategies and interventions aimed at enhancing both awareness and knowledge about HE/PE among registered nurses in the KSA, while meticulously considering pertinent sociodemographic and HE/PE-related factors.

The global burden of end-stage kidney disease (ESKD) is rising, compelling patients to increasingly confront the choice of initiating kidney replacement therapy (KRT). Cost-utility analysis (CUA) has the advantage of incorporating patient-reported outcomes regarding KRT. The aim of this study is to summarize the CUA of hemodialysis (HD), peritoneal dialysis (PD), and kidney transplantation in patients with ESKD. A systematic search was conducted to identify relevant articles in three primary databases (PubMed, SCOPUS, and ProQuest). CUA studies of dialysis for ESKD patients from 2000 to 2023 in all countries were included. Non-full-text, non-English language, review articles, systematic reviews, and studies that did not compare distinct dialysis methods were excluded. All the information was summarized narratively. Out of the 130 studies identified, 13 met the inclusion criteria and were included in this review. Most studies demonstrated good reporting quality, with CHEERS checklist scores ranging from 75% to 96%. Kidney transplantation was consistently found to be the most cost-effective KRT, offering the highest quality-adjusted life years (QALYs) and the lowest cost per QALY in the long term, despite higher initial expenses. PD showed better cost-effectiveness than HD in several studies. HD generally incurred the highest costs with lower QALY gains. Cost-effectiveness trends varied depending on regional context, healthcare perspective, and patient comorbidities. This study found that kidney transplantation is consistently the most cost-effective kidney replacement therapy, with lower cost per QALY and improved quality-adjusted life years (QALY) in most settings. In particular, transplantation showed favorable long-term outcomes despite higher initial costs, whereas peritoneal dialysis emerged as a more cost-effective alternative to hemodialysis, especially in resource-limited settings. These findings offer practical implications for treatment prioritization and policy-making in both high- and low-resource countries.

Purpose: Malnutrition in cancer patients may stem from both tumor progression and treatment regimens. Nutritional interventions have shown potential for enhancing treatment efficacy and overall quality of life, but their cost-effectiveness requires exploration. Accordingly, this study systematically investigated research on economic evaluations of nutritional interventions implemented as either preventive or therapeutic strategies in cancer patients.

Methods: PubMed and Scopus databases were searched from inception to September 9, 2025. Following study selection and data extraction, the reporting quality of included studies was evaluated using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist, while risk of bias was assessed using the ECOBIAS checklist.

Results: Eight studies, published between 1989 and 2022, primarily focused on gastrointestinal cancers and were conducted in high-income countries. Most of these investigations (6 out of 8) performed cost-utility analyses. Four indicated that combining oral supplements or parenteral nutrition with nutritional counseling was more cost-effective, although these studies differed in terms of time horizons, types of interventions evaluated, and types of comparators used. Most of the studies adhered to the CHEERS standards, but no reported on characterizing heterogeneity, and none discussed approaches to stakeholder involvement in study design. Based on the ECOBIAS framework, the three most frequently encountered risks of bias were limited sensitivity analysis bias, bias related to quality-of-life weights, and limited scope bias.

Conclusion: The findings offer physicians valuable guidance for optimizing treatments and potentially support policy decision-making.

Purpose: Uganda's digital health transformation anchored on District Health Information Software 2 (DHIS2) and the FAIR (Findable, Accessible, Interoperable, Reusable) Data Principles has reshaped health data governance. Nevertheless, systemic constraints in privacy, infrastructure, and human resources threaten sustainability and equity.

Objective: To compare DHIS2 and FAIR-based approaches on (i) privacy protection, (ii) interoperability and data usability, and (iii) regulatory/institutional readiness for privacy-preserving health data analytics in Uganda.

Methods: Systematic review of 84 peer-reviewed and grey-literature sources (2010-2025) following PRISMA 2020; extracted indicators on reuse, interoperability, privacy, and institutional readiness.

Results: 36% of included studies were Uganda-specific; 50% were published in 2020-2024. DHIS2 reached near-national coverage, ~12,000 trained users, and integration across >20 programmes. Persistent gaps include limited rural internet (≈12% of facilities with stable connectivity), high staff turnover (~35%), and low analytics literacy (~25% with intermediate skills). FAIR efforts (since ~2019) remain early: ~10% of institutions with formal policies; low dataset reuse (~22%), machine-readable metadata (~18%), and documented digital consent (<10%). Privacy infrastructure is weak: <30% of facilities with formal privacy frameworks/secure platforms and <10% with Data Protection Officers.

Conclusion: DHIS2 improved reporting and availability, while FAIR initiatives began enabling governed, interoperable reuse. To achieve ethical analytics at scale, priorities are legal enforcement, secure rural ICT, standardized machine-readable metadata/consent, and workforce development.