Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Objectives: Given the limited evidence on UK nurses' preferences for pediatric hexavalent vaccines, we aimed to evaluate their preferences for these vaccines' attributes.

Methods: In a discrete-choice experiment study, 150 nurses chose between 2 hypothetical pediatric hexavalent vaccines with varying attribute levels (device type, plastic in packaging, time on the market, and time the vaccine can stay safely at room temperature) in a series of choice questions. Using random-parameters logit-model estimates, conditional relative attribute importance (CRAI) and odds ratios (ORs) were calculated.

Results: Device type (with associated preparation time and risk of dosage errors) was the most important attribute (CRAI, 61%), followed by years on the market (CRAI, 25%). The odds of choosing a prefilled syringe were nearly 3 times the odds of choosing syringe-and-vial combinations requiring reconstitution (OR, 2.80; 95% confidence interval [CI], 1.93-3.68). Vaccines on the market for < 1 year were less likely to be preferred to vaccines available for > 3 years (OR, 0.66; 95% CI, 0.47-0.84). ORs for time a vaccine can stay at room temperature (3 vs. 6 days) (0.94; 95% CI, 0.71-1.16) and plastic blisters in packaging (1.19; 95% CI, 0.80-1.56) were not significant, indicating that these attributes did not influence choices.

Conclusions: In this survey, nurses' preferences were mainly influenced by device type.

Background: Biosimilar drugs represent a valuable opportunity for healthcare systems worldwide as they offer substantial cost savings. This retrospective observational study aimed to assess the current prescribing trend in order to raise awareness among physicians for more appropriate prescribing.

Research design and methods: A study was conducted to assess the use of major biologic drugs across different therapeutic areas within two Italian healthcare entities. Usage and cost data were obtained from company databases. In addition, a comparison with 2021 data was performed to identify any increases in biosimilar drug utilization during 2022.

Results: The analysis for 2022 revealed that the majority of studied active ingredients have been administered as biosimilar drugs, with percentages close to 100%, except for a few exceptions. In particular, resistance remained in the prescription of biosimilar adalimumab (73.8%) and etanercept (73.3%) in Asl Napoli 3 Sud, erythropoietin (69.5%) and rituximab (72.8%) in Policlinico Umberto I.

Conclusions: A comparison between 2021 and 2022 highlighted the increasing adoption of biosimilar drugs. This positive trend suggested the potential for full utilization in the near future, benefiting significantly the National Health System and the public, ensuring a healthcare approach that is effective and sustainable.

Background: Worldwide, many clinical trials are performed using clinical outcomes and surrogate outcomes as endpoints. Surrogate outcomes are used, for instance, if there is not enough follow-up time to measure the outcome of interest. Surrogate outcomes might not be patient-relevant, however. This study assesses to what extent patient-relevant outcomes are measured in clinical trials for breast cancer drugs.

Research design and methods: A cross-sectional comparative analysis was conducted in which patient preferences for outcomes derived from the literature were compared to outcomes measured in phase III breast cancer trials conducted between 2014 and 2024.

Results: Patients prefer outcomes addressing survival benefits, treatment effectiveness, adverse events and health-related quality of life. Minor improvements in survival benefits are greatly valued. The majority of patients are willing to accept some side effects for a positive outcome. The primary outcome used most frequently in trials is progression-free survival. The most common secondary outcomes are adverse events, mortality, overall response rate, and health-related quality of life.

Conclusion: Phase III trial outcomes appear to align largely with breast cancer patients' preferences. Nevertheless, patients and trial designers emphasize different outcomes. Improvement is therefore needed to enhance the relevance of trial data for patients.

Background: This study compared healthcare resource utilization (HCRU) and cost among patients treated for pit viper envenomation with Fab or F(ab')₂ antivenom in the United States.

Research design and methods: Patients administered Fab or F(ab')₂ between January 2019 and September 2022 were identified from IQVIA's New Data Warehouse and placed into two mutually exclusive cohorts. Inverse probability of treatment weighting (IPTW) was used to adjust for baseline differences between cohorts. All-cause HCRU and cost were assessed in the 6-months following antivenom administration (post-index) and compared between IPTW-adjusted cohorts.

Results: In total, 242 and 98 patients receiving Fab and F(ab')₂ were identified, respectively. Pre-IPTW adjustment, cohorts differed on demographics and select comorbidities (standardized mean difference [SMD] ≥0.10). Post-IPTW, most variables were balanced (SMD < 0.01). Post-index total (Fab: $70,994; F(ab')₂: $55,324), medical ($69,162; $52,339), and inpatient costs ($49,466; $29,730) were higher among Fab than F(ab')₂ patients (all p < 0.05). After multivariable adjustment, the total (cost ratio [CR]: 0.83; 95% confidence interval [CI]: 0.70-0.98), inpatient (CR: 0.63; 95% CI: 0.51-0.79), and inpatient pharmacy costs (CR: 0.48; 95% CI: 0.33-0.68) were lower in the F(ab')₂ than Fab cohort.

Conclusions: Patients administered Fab had higher post-index total, inpatient, and inpatient pharmacy costs than patients administered F(ab')₂ AV.

Background: This study aimed to evaluate the cost-effectiveness of ASCT and maintenance therapy strategies for transplant-eligible patients with newly diagnosed multiple myeloma from a Chinese healthcare perspective.

Research design and methods: A short-run decision tree and a long-run Markov model were created to assess the mean costs and quality-adjusted life-years (QALYs) of ASCT plus maintenance therapy over a lifetime horizon. Utility values were sourced from published literature, while healthcare costs were based on a single-center retrospective analysis and national drug bidding data.

Results: The ASCT strategy with two-year daratumumab maintenance had the lowest discounted costs (1,394,183 CNY). Compared to no ASCT with continuous lenalidomide maintenance, ASCT had an ICER of 90,997 CNY/QALY. With one-year lenalidomide maintenance, ASCT provided more QALYs at lower costs. One-year lenalidomide maintenance after ASCT also resulted in higher QALYs and lower costs than no maintenance, while continuous lenalidomide had an ICER of 396,731 CNY/QALY.

Conclusions: Under the WTP threshold of CNY 268,074 per QALY, ASCT strategies were more cost-effective than non-ASCT approaches. One-year lenalidomide therapy after ASCT was more cost-effective than no maintenance or continuous therapy. Among all strategies, ASCT followed by two years of daratumumab maintenance was the most cost-effective option.

Background: This study explores Turkish medical oncologists' perceptions of integrating international treatment guidelines with national reimbursement policies, considering local legal, economic, and healthcare constraints.

Research design and methods: A cross-sectional online survey was conducted from March 24-31, 2024, targeting all 1,096 active oncologists registered with the Turkish Medical Oncology Association, as these specialists are exclusively authorized to prescribe anticancer drugs under national regulations. The survey included 25 questions on demographics, perceptions of guidelines, and integration preferences. Statistical analyses, including chi-square tests and logistic regression, identified factors influencing guideline preferences.

Results: Among 337 respondents (31%), 94% found international guidelines essential, but 62% noted a lack of clear real-world algorithms. Significant predictors for preferring national guidelines included working in public institutions (OR: 3.90, p < 0.001), concerns about pharmaceutical industry influence (OR: 4.38, p = 0.017), legal challenges (OR: 5.89, p < 0.001), and variability among clinical research centers (OR: 2.95, p = 0.019). Despite these challenges, 57% favored national guidelines for their compatibility with local healthcare policies.

Conclusions: The findings highlight the need for hybrid models that merge the evidence-based rigor of international frameworks with local healthcare priorities. Such models can enhance equitable and effective cancer care in Türkiye by addressing both global standards and national realities.

Objectives: Although U.S. Preventive Services Task Force (USPSTF) recommended CRC screenings are effective; patient reluctance reduces adherence. Most cost-effectiveness models assume perfect adherence, yet one-third of eligible individuals aren't current with CRC screening. Our study assesses the cost-effectiveness of Shield, an FDA-approved blood-based CRC screening test, using real-world adherence.

Methods: The CAN-SCREEN (Colorectal cANcer SCReening Economics and adherENce) model, a validated discrete-event simulation, evaluated clinical and economic outcomes of CRC screening under real-world adherence scenarios. We compared the Shield blood-based test administered every 3 years to no screening, considering it cost-effective if the incremental cost-effectiveness ratio (ICER) was under $100,000 per quality-adjusted life-year (QALY) gained.

Results: Shield increased QALYs by 154 and raised costs by $7.5 million per 1,000 individuals, with an ICER of $48,662 per QALY, meeting the $100,000/QALY threshold. Shield remained cost-effective up to a unit cost of $3,241 (at $100,000/QALY) and $4,942 (at $150,000/QALY). Sensitivity analyses confirmed cost-effectiveness with lower adherence to diagnostic colonoscopy (56.1%) and annual screenings.

Conclusion: The CAN-SCREEN model shows that Shield is cost-effective compared to no screening. Including real-world adherence improves accuracy in assessing screening strategies. Shield's noninvasive approach offers a promising, cost-effective way to increase adherence and reduce CRC mortality.

Background: Atopic dermatitis (AD) imposes a hidden burden through its negative effects on quality of life and productivity. We aim to estimate this hidden burden in adults and adolescents in Central and Eastern European (CEE) countries.

Methods: We created a burden of disease model to quantify AD's hidden burden. Humanistic burden was calculated by estimating the monetary value of quality-adjusted life years (QALYs) lost, using prevalence data from the Global Burden of Disease study and gross domestic product (GDP) per capita for each country. Indirect economic burden was estimated based on productivity loss from absenteeism and presenteeism, adjusted for labor force participation and unemployment rates. Total hidden burden was determined by combining productivity losses and QALYs lost.

Results: QALY loss due to AD ranged from 1,832 to 58,596 annually in CEE countries, equating to 38 million to approximately 1 billion Euros per country. Productivity losses ranged from 3.6 to 148.9 million Euros annually. The total hidden burden of AD represents 0.11% to 0.43% of the GDP.

Conclusions: Our estimates reflect significant differences in population size, prevalence, and economic strength among CEE countries. Adjusting findings to country-specific GDP provided insights into AD's true hidden burden, offering valuable information for decision-making.

Introduction: Rheumatoid Arthritis (RA) is among the most prevalent chronic inflammatory diseases affecting millions of people with a significant expenditure of resources by National Healthcare Systems. This study aimed to analyze all available treatments exclusively for RA to highlight the costs of each treatment type and raise awareness of the use of biosimilar drugs.

Methods: In an Italian healthcare authority, all prescriptions made for the diagnosis of RA were extracted to verify consumption expressed in Defined Daily Dose (DDD) and the expenditure incurred expressed in euros. Consequently, a grouping into three major drug categories has been performed: anti - tumor necrosis factor alpha agents (TNFα), other injectable formulations, and novel oral formulations.

Results: Prescriptions for the second half-year 2022 and 2023 have been analyzed, with a total cost of almost 7 million euros in the sample considered. All pharmaceutical categories showed an increase in consumption, but only anti- TNFα recorded a decrease in costs from 25% in 2022 to 22% in 2023, thanks to the lower cost of the biosimilar drug.

Conclusion: The costs of RA may represent a significant spending commitment for central governments. As a result, actions are needed to encourage the preferential use of biosimilar drugs.

Objectives: To estimate the cost-utility of duloxetine compared with that of a placebo, common traditional nonsteroidal anti-inflammatory drugs (NSAIDs) and cyclooxygenase-2 (COX-2) inhibitors for the treatment of osteoarthritis (OA) from a Chinese healthcare perspective.

Methods: A Markov model was constructed. The costs and utility inputs were obtained from the database and published literature. Incremental cost-effectiveness ratio (ICER) was the main model outputs. Subgroup analyses were also conducted for patients at high risk of gastrointestinal (GI) or cardiovascular (CV) AEs. Deterministic and probabilistic sensitivity analyses were performed.

Results: The model estimated an ICER of $3409.21/QALY for duloxetine compared with etoricoxib, with duloxetine dominating other active treatment strategies in patients at a low risk of GI and CV AEs. The ICER for duloxetine over etoricoxib was $322.21/QALY in patients at high risk of GI and CV AEs. These results were consistent with the sensitivity analyses; 53.64% and 53.93% of the patients were willing to use duloxetine comparing with etoricoxib, for which the thresholds were 1.0 and 3.0 per capita gross domestic product (GDP), respectively.

Conclusions: Duloxetine is a valuable option for patients with OA; however, uncertainties exist in the model, and these suggestions can be adopted with caution.