Expert Review of Pharmacoeconomics & Outcomes Research最新文献

Background: Cannabis-based medicinal products (CBMPs) are increasingly demonstrating effectiveness in treating a wide range of conditions, with a relatively high safety profile in clinical usage compared to other prescription pain medications and few contraindications. Consultation and other prescription-related costs are, at present, higher for CBMPs than for some other treatment options, leading to some concern around wider prescribing.

Research design and methods: An early cost-effectiveness model was developed to estimate the impact of prescribing CBMPs alone and/or in addition to analgesics, physiotherapy, and cognitive behavioral therapy for chronic pain in the UK for 1 year.

Results: Due to their comparative effectiveness, CBMPs were found to be cost saving. Various scenarios were model tested; in all scenarios where CBMPs decrease pain-level states, less resource use is required. Increased efficacy of 5% was conservatively assumed based on current Real-World Evidence. In this scenario, CBMPs were significantly more cost-effective, and as costs relating to the prescribing of these continue to fall, relative savings are predicted to increase.

Conclusion: These findings highlight the substantial cost saving that CBMPs may represent for the treatment of chronic pain patients, and the benefits for healthcare providers as a treatment for this often hard-to-treat population.

Objective: This study compared the survival outcomes of non-traumatic intracerebral hemorrhage (ICH) patients with different famotidine administration routes and explored related risk factors.

Methods: Data from ICH patients between 2008-2019 were extracted from the MIMIC-IV database. Survival differences between patients with intravenous (IV) and non-intravenous (Non-IV) famotidine administration were analyzed using Cox analysis and Kaplan-Meier survival curves.

Results: The study included 351 patients, with 109 in the IV group and 84 in the Non-IV group after PSM. Cox analysis revealed that survival was significantly associated with age (HR = 1.031, 95%CI:1.011-1.050, p = 0.002), chloride ions (HR = 1.061, 95%CI:1.027-1.096, p < 0.001), BUN (HR = 1.034, 95%CI:1.007-1.062, p = 0.012), ICP (HR = 1.059, 95%CI:1.027-1.092, p < 0.001), RDW (HR = 1.156, 95%CI:1.030-1.299, p = 0.014), mechanical ventilation (HR = 2.526, 95%CI:1.341-4.760, p = 0.004), antibiotic use (HR = 0.331, 95%CI:0.144-0.759, p = 0.009), and Non-IV route (HR = 0.518, 95%CI:0.283-0.948, p = 0.033). Kaplan-Meier curves showed a significantly higher 30-day survival rate in the Non-IV group (p = 0.011), particularly in patients with normal ICP (HR = 0.518, 95%CI:0.283-0.948, p = 0.033).

Conclusion: Non-IV famotidine administration significantly improves 30-day survival of ICH patients, especially for those with normal ICP, compared to IV administration.

Objective: Despite the beneficial effects of DOACs, suboptimal adherence is widely documented, and real-world adherence is lower than in clinical trials. The objective of this study is to compare the cost-effectiveness of apixaban versus rivaroxaban for stroke prevention by incorporating real-world adherence from the US payer's perspective.

Methods: We developed a Markov model with three health states to evaluate the total costs and quality-adjusted life years (QALY) at a willingness to pay threshold of $100,000. The population was a hypothetical cohort of 65-year-old patients with moderate to high stroke risk. The transition probabilities of healthy adherent, nonadherent, and stroke were obtained from a Medicare Advantage Plan. The utilities and costs were obtained from prior clinical studies. Deterministic and probabilistic sensitivity analyses were conducted.

Results: Apixaban was cost-effective than rivaroxaban at a willingness to pay threshold of $100,000. Apixaban yielded an additional 0.12 QALYs at a cost of $1904.39, resulting in an incremental cost-effectiveness ratio (ICER) of $16,279.25 per QALY gained. The Monte Carlo simulations indicated that apixaban was cost-effective at 89.67% of simulations. The ICER results were impacted by the medical costs among nonadherent patients.

Conclusion: After incorporating adherence, apixaban 5 mg was a cost-effective alternative to rivaroxaban 20 mg for stroke prevention among elderly atrial fibrillation (AF) patients.

Background: Estimate the costs of inpatient and outpatient care for people with Cerebral Palsy (CP) in Brazil.

Research design and methods: Health records of people with CP in the Hospital and Outpatient Information Systems of Brazil between 2015 and 2019 were analyzed. Variables analyzed were gender, age, ICD, Intensive Care Unit (ICU) use, total cost, and ICU cost. Costs were adjusted for inflation and converted to dollars. Linear regression analysis was performed to investigate the association between social and clinical variables and direct costs.

Results: A total direct cost of approximately $166 million to the National Health System was identified, with $7.08 million/year and $26.1 million/year of inpatient and outpatient costs, respectively. The healthcare was primarily for children up to 14 years of age. The ICD 'spastic quadriplegic CP' received the most attendance. Rehabilitation was responsible for 75% of the outpatient care, with physiotherapy standing out. Increased age, use of ICU, and the types of CP are related to increased cost.

Conclusions: Healthcare for people with CP produced expressive costs for the Brazilian public health system, mainly with outpatient procedures and rehabilitation, with children being the most attended. Estimating these costs assist in better resource allocation for more effective healthcare provision.

Background: As rheumatoid arthritis (RA) is a chronic and progressive disease that requires lifelong therapeutic intervention, it represents a considerable economic burden on those affected. This study investigated whether tofacitinib is a cost-effective therapeutic alternative to other DMARDs for treating moderate-to-severe RA.

Research design and methods: All economic evaluation studies of tofacitinib compared to other DMARDs were identified. Using random-effects meta-analysis, we pooled incremental net benefit (INB) in (purchasing power parity) adjusted US$ with 95% confidence intervals. The modified economic evaluation bias checklist and Grading of Recommendation, Assessment, Development, and Evaluation (GRADE) instrument for quality appraisal were used. The subgroup analysis was done based on the comparator regimen.

Results: Of the selected 11 studies, the number of studies from high-, upper-middle- and lower-middle-income countries was 7, 3, and 1, respectively. The subgroup analysis showed that tofacitinib with an INB of 19,180 US$ [95% CI, -34520 to -3840; p-value = 0.01] was not statistically cost-effective compared with cDMARDs (p-value > 0.0001). Compared to other DMARDs, the estimated pooled INB of tofacitinib was US$ 7260 [95% CI, 3030 to 11,480; p-value < 0.001], but there was substantial heterogeneity among the included studies, and the observed publication bias.

Conclusion: While tofacitinib shows potential as a cost-effective treatment, tailored economic evaluations are needed to account for the diverse and evolving contexts of RA treatment.

Registration: PROSPERO: CRD42023405970.

Background: This study evaluates the cost-effectiveness of adding ocrelizumab to supportive care for primary progressive multiple sclerosis (PPMS) in Iran.

Research design and methods: Using a lifetime horizon from the payer's perspective, we developed a decision analytic model with Expanded Disability Status Scales (EDSS) as Markov health states while taking transition probabilities and treatment effects into account. Data were sourced from clinical trials and other literature. The target population was PPMS patients receiving either supportive care or ocrelizumab. We assessed cost- effectiveness through total costs, quality-adjusted life-years (QALYs), and the incremental cost- effectiveness ratio (ICER). Sensitivity analyses addressed uncertainties.

Results: The addition of ocrelizumab to supportive care provided an incremental gain of 0.89 QALYs and an additional cost of US$76,771.34, resulting in an ICER of US$86,220.35 compared to supportive care, which is 5.2 times Iran's GDP per capita (US$16,557). Thus, ocrelizumab is not cost-effective at the threshold of one time GDP per capita. However, the probability of cost-effectiveness increases at higher thresholds. Sensitivity analyses confirmed the robustness of the results.

Conclusion: While ocrelizumab is not cost-effective at the threshold of one-time GDP per capita, its clinical benefits are significant. Formulating healthcare policies for high-cost medications with low alternatives like ocrelizumab is essential.

Objectives: Our study assessed the budget impact and cost per responder of upadacitinib15mg and 30 mg for moderate to severe atopic dermatitis (MS-AD) treatment from social security and private health sector perspective in Argentina.

Methods: A budget impact model was adapted to depict clinical and economic aspects of treatment over a 5-years horizon time. Scenario analyses and deterministic sensitivity analyses were performed. A 16-weeks cost per responder model was adapted based on a network meta-analysis. Primary analyses assessed the cost per Eczema Area and Severity Index 50, 75 and 90 at week 16.

Results: The inclusion of upadacitinib 15 mg and 30 mg in the biological treatment mix for MS-AD was associated with an average budget saving per-member per-month ofU$S0.062 (social security) and U$S0.064 (private sector). Percentage of patients with access to treatment, acquisition cost of upadacitinib 30 mg and prevalence of MS-AD were the most influential parameters in the budget impact results. At week 16, upadacitinib 30 mg was associated with the lowest number needed to treat and the lowest cost per responder for all outcomes.

Conclusion: The introduction of upadacitinib in MS-AD treatment was associated with modest savings for the social security and private payer budget in Argentina.

Introduction: The Human Immunodeficiency Virus (HIV) is one of the greatest public health challenges still facing communities worldwide, and until this moment, no vaccine is available for its prevention. In Brazil, the Rio de Janeiro State has stood out regarding the prevalence of this disease. As a result, an important state to consider the Willingness to Pay (WTP) for a hypothetical HIV vaccine to help with future pricing.

Methods: A cross-sectional study was conducted to assess the acceptability and WTP of individuals from Rio de Janeiro State for a hypothetical HIV vaccine with a 70% efficacy.

Results: 600 individuals were interviewed and the acceptability for this hypothetical vaccine was 77.2%. In addition, 452 participants were eligible for the WTP analysis and would accept a WTP US$79.37 (400 BRL) for this vaccine, a higher value than that found in another study (200 BRL) conducted in the Northern region of Brazil under the same methodological conditions.

Conclusion: Economic studies such as WTP can contribute to discussions regarding the prices and specifications for future vaccines, particularly for a HIV vaccine in countries such as Brazil with over 5,000 municipalities spread across regions with diverse characteristics and challenges in terms of socioeconomic, epidemiological and cultural differences.

Introduction: Parkinson's Disease (PD) is a progressive, chronic neurodegenerative disease, representing significant economic and social burdens. It is typically defined by motor symptoms (MSs), however, this does not reflect the full patient burden. Non-motor symptoms (NMSs) are increasingly recognized as central characteristics of PD. However, they still lack recognition in research. Therefore, this study aims to identify relevant NMSs, their prevalence, and the effect they have on Quality-of-Life (QoL) and Cost-of-Illness (COI). Secondly, it aims to identify gaps in the current body of knowledge and propose possible ways future research could bridge those gaps.

Methods: The study employed a scoping review, identifying 60 records for inclusion, using PubMed and Web of Science. It included studies from Spain or Italy, including data on People with Parkinson's Disease. A comparative analysis was performed using Microsoft Excel.

Results: It showed that the body of evidence relevant to NMSs, their prevalence, QoL, and COI is limited, or that estimates vary to an extent where interpretation is difficult.

Conclusion: Most studies suffer from generalization, representation, and standardization issues, stemming from their designs and methodological decisions. Although the findings of this study should be interpreted with caution, several recommendations are made for future research.

Background: Viscosupplementation is a viable alternative for managing knee osteoarthritis, showing potential to delay the need for total joint replacement in affected patients.

Methods: We constructed an economic model that compared viscosupplementation with hylan G-F 20, with available hyaluronic acids, and no viscosupplementation over a 5-year period, from the perspective of the Colombian general health system. Time until total knee replacement, sourced from published literature, informed the model. Costs, including acquisition, administration, and knee replacement surgery, were sourced from the local drug prices database and the Ministry of Health's integral information system and expressed in USD.

Results: Over 5 years, hylan G-F 20 demonstrated a total cost of USD$45,188,523 compared to hyaluronic acids (USD$50,247,826) and no viscosupplementation (USD$27,345,736). Hylan G-F 20 yielded 39,400 total knee replacement-free years, exceeding hyaluronic acid (30,741) and no viscosupplementation (4,280). Cumulative costs per surgery-free patient showed substantial savings, ranging from USD$1,158 to $6,847 for hylan G-F 20, compared to $1,164 to $74,662 for hyaluronic acid and $5,426 to $6,389 for no viscosupplementation.

Conclusions: Hylan G-F 20 showed reduced cost per surgery-free patient compared to hyaluronic acids and no viscosupplementation, enhancing knee replacement-free years.