ClinicoEconomics and Outcomes Research最新文献

Background: Secukinumab, an IL-17A inhibitor, has been recommended for psoriatic arthritis (PsA) patients with contraindications to TNF-alpha inhibitors (TNFi). However, its budgetary implications in Thailand remain unclear.

Objective: To estimate the 5-year budget impact of introducing secukinumab 150 mg for PsA patients contraindicated to TNFi therapy from the perspective of the Thai healthcare system.

Methods: A budget impact analysis (BIA) was developed following the Thai HTA Guideline. Two treatment scenarios were compared: the current standard of care using csDMARDs and a new scenario incorporating secukinumab 150 mg (auto-injector). The model estimated eligible patients based on national demographics and clinical data. Costs included direct medical costs for medications, administration, monitoring, and complications. Net budget impact (NBI) was estimated. Deterministic sensitivity analyses were conducted to evaluate the impact of varying uptake rates and treatment durations.

Results: Under the base-case scenario over five years, secukinumab use resulted in the NBI of 15.14 million THB (434,965 USD), with an average annual net budget impact of 3.03 million THB (86,993 USD). Drug acquisition accounted for 97.16% of the total budget impact. Sensitivity analyses revealed a higher financial burden with increased uptake or lifetime treatment but remained within a manageable range (up to 18.34 million THB or 526,849 USD).

Conclusion: Introducing secukinumab for PsA patients contraindicated to TNFi is associated with a moderate increase in healthcare expenditure. These findings suggest that inclusion of secukinumab in the National List of Essential Medicines for this specific population may be feasible, conditional on successful price negotiation and/or managed entry arrangements to ensure budgetary sustainability and equitable access.

Purpose: Unit-hour utilization (UHU) is the most common metric for evaluating the productivity of ambulances and crews for ambulance services. Calculating UHU is essential for maximizing profitability and evaluating the number of hours of ambulance utilization during a shift. This has not been examined previously at King Abdulaziz Medical City (KAMC). KAMC is a major tertiary-care medical center in Riyadh, Saudi Arabia, with dedicated ALS (Advanced Life Support) and BLS (Basic Life Support) ambulance units. This study aimed to determine the cost of operating the emergency calls (advanced life support [ALS]) unit and non-emergency calls (basic life support [BLS]) unit and compare the costs of emergency calls (ALS) and non-emergency calls (BLS). The study used a standardized institutional checklist that itemizes expenditures across logistics, equipment, maintenance, salary, pharmacy, and fuel cost components.

Methods: This was a retrospective cross-sectional study. Institutional Review Board approval was obtained from King Abdullah International Medical Research Center. The study examined emergency and non-emergency calls for a period of 12 months. This study was based on a checklist assessing various expenditures to calculate the cost of ambulance utilization at KAMC in 2022.

Results: The total average ambulance utilization cost per year for ALS was 4,806,245.7 SR, and the average ambulance utilization cost per hour was 548.65 SR; these costs were to operate the ALS crew for 24 hours, seven days a week. Conversely, the total average ambulance utilization cost per year for BLS was 3,934,156.92 SR, and the average ambulance utilization cost per hour was 449.10 SR. The main expenses concerned salaries and overhead costs for ALS and BLS.

Conclusion: This study provides insights into the cost of ambulance utilization between ALS and BLS. The higher cost of ALS calls may be attributed to the higher level of training, equipment, and staffing required.

Background: According to an analysis of the catastrophic costs incurred by people with tuberculosis, 56% of households in the Democratic Republic of Congo bear the economic burden of tuberculosis. The objective of this study was to determine the clinical and economic burdens of tuberculosis treatment in vulnerable patients.

Methods: A descriptive, cross-sectional study was conducted at Janson Sendwe Provincial General Referral Hospital between September and December 2023, a four-month period. Data collection was based on a previously developed survey form with several parameters.

Results: In this study, 59 tuberculosis cases with vulnerability factors were recorded. Male sex was the most prevalent with 62.71%, the average patient age was 33.37 ± 9.92 (15-68) years, TB/HIV coinfection was the most prominent with 94.92%, the average total cost was 56.74 ± 20.81 USD, direct costs were 10.61 ± 0.00 and indirect costs were 47.75 ± 19.23 USD. The average direct costs were 1.56 ± 0.19 USD.

Conclusion: Catastrophic costs for tuberculosis patients can be addressed by improving the delivery and financing of tuberculosis services and strengthening social protection for tuberculosis patients.

Purpose: Thiotepa is an intravenous alkylating agent used with other chemotherapy drugs to treat several forms of cancers. Preparation of thiotepa requires reconstitution of powder in sterile water, followed by dilution of the solution with sodium chloride 0.9% solution for injection. The reconstitution of lyophilized powdered drug formulations may pose challenges as it can increase the risk of preparation errors, leading to wastage or potential clinical consequences, and extend preparation time compared to ready-to-dilute formulations; one study found that 15% of thiotepa doses were made out-of-tolerance. Tepylute^® is a ready-to-dilute formulation of thiotepa and has been approved by the United States Food and Drug Administration for the treatment of breast and ovarian adenocarcinoma. The objective of this study was to explore the economic impact to a hospital pharmacy of using ready-to-dilute Tepylute^® versus a lyophilized powdered formulation of thiotepa.

Patients and methods: A cost offset model was created in Microsoft Excel™, the analysis compared a current scenario (thiotepa: 100% market share) with a future scenario (Tepylute^®: 100% market share) for a hospital pharmacy treating 96 cancer patients annually. Costs (2024 USD) included drug acquisition, compounding time, drug wastage, and clinical consequences due to reconstitution errors. Incremental costs, cost offsets, and pharmacy time saved were evaluated.

Results: For a hypothetical hospital pharmacy, use of Tepylute^® versus a lyophilized powdered formulation of thiotepa resulted in savings of $339,777 over 1-year ($3539 per patient), primarily from reduced drug acquisition costs, as well as a reduction in drug wastage, clinical consequences due to reconstitution errors, and compounding time. Results were consistent across sensitivity and scenario analyses.

Conclusion: Modeled results demonstrate that ready-to-dilute Tepylute^® may save time, reduce acquisition and preparation costs, and lower clinical risks from preparation errors. This study is among the first to quantify potential savings from switching to a ready-to-dilute formulation.

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.

Purpose: To describe real-world patient characteristics and outcomes after initiation of Acthar Gel (repository corticotropin injection, RCI) in the management of nephrotic syndrome (NS). This real-world study characterized patients with membranous nephropathy (MN) who initiated RCI therapy and evaluated the impact of higher versus lower adherence to RCI on NS-related outcomes, healthcare resource utilization, and medical costs.

Patients and methods: We retrospectively analyzed data from the Integrated Dataverse from Symphony Health database. Adult (≥18 years) patients were included if they had ≥1 inpatient or ≥2 outpatient claims for an NS and MN diagnosis (January 1, 2016-December 31, 2022); ≥1 prescription claim for RCI; and continuous eligibility ≥12 months before and ≥12 months following and including the index date. Patients with ≥2 RCI claims and a calculated proportion of days covered (PDC) were included. Those with a PDC above the sample mean were classified as "above-average" adherence and those below the mean as "below-average" adherence in that period.

Results: The analyzed population comprised 28 patients with below-average and 34 with above-average RCI adherence. The above-average adherence cohort showed a noticeable reduction compared with the below-average adherence cohort in corticosteroid use, extended corticosteroid use, opioid use, NS-related renal biopsy procedures, all-cause and NS-related inpatient hospitalizations, and NS-related office visits. From baseline to follow-up, inpatient hospitalization rates were similar in the below-average adherence cohort for all-cause (50.0% vs 53.6%) and NS-related (35.7% vs 28.6%) admissions but markedly lower in the above-average cohort for all-cause (50.0% vs 23.5%) and NS-related (41.2% vs 8.8%) hospitalizations. Despite similar baseline medical costs, all-cause ($131,306 vs $50,048) and NS-related costs ($111,743 vs $29,868) were noticeably higher at follow-up in the below-average adherence cohort than the above-average adherence cohort.

Conclusion: This real-world study shows that greater adherence to RCI during treatment improves overall outcomes studied.

Purpose: This study aimed to explore the impacts and economic burden experienced by patients and families affected by Pompe disease in Thailand.

Patients and methods: A mixed-methods analysis was employed. To estimate the direct medical cost, databases and/or medical records of the seven rare disease (RD) centers in Thailand from 2010 to 2021 were reviewed. Structured interviews were also conducted to examine direct non-medical and indirect costs. The economic burden was presented in 2024 US dollars (USD) value (1 USD = 35.29 THB). In-depth interviews were conducted with four patients and two caregivers to explore the impacts of this disease.

Results: Frequently reported symptoms included fatigue, muscle weakness, and difficulty breathing during sleep. These symptoms had significant and profound impacts on patients' functioning, family and social roles, self-esteem, emotion, and financial status. Frequent visits to the RD centers, along with the referral process, were reported as a substantial burden to the patients and families. The mean annual direct medical cost per patient, excluding the cost of enzyme replacement therapy, ranged between 2,505 and 14,042 USD from the provider's perspective and 1,484 USD from the patient's perspective. Direct non-medical costs were highly significant, with the annual cost of informal care of 1,878-1,992 USD per patient. Around 43% of the patients reported requiring informal care. On average, each patient required 2.6 ± 3.5 hours of informal care per day.

Conclusion: Our findings revealed substantial impacts of Pompe disease on individuals' physical, social, emotional, and functional capacities as a result of its symptoms. Coordinated care, where patients can be treated by clinicians at local hospitals who operate in close liaison with specialists from the RD centers, is warranted. Psychosocial, welfare, and transportation supports are clearly justified to alleviate the burden and improve the quality of life of the patients.

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.