Search Results (92)

Background and aim: Advanced Therapy Medicinal Products (ATMPs) are innovative drugs based on genes, tissues, or cells that target rare and severe diseases. ATMPs have shown promising clinical outcomes but are associated with high costs, raising questions about cost-effectiveness. Hence, this systematic review aims to analyze the cost-effectiveness and cost-utility profiles of the European Medicines Agency-authorized ATMPs for treating rare diseases. Methods: A systematic review was conducted following PRISMA guidelines. Studies were identified by searching PubMed, Embase, Web of Science, and ProQuest scientific databases. Economic evaluations reporting incremental cost-effectiveness/utility ratios (ICERs/ICURs) for ATMPs were included. Costs were standardized to 2023 Euros, and a cost-effectiveness plane was constructed to evaluate the results against willingness-to-pay (WTP) thresholds of EUR 50,000, EUR 100,000, and EUR 150,000 per QALY, as part of a sensitivity analysis. Results: A total of 61 studies met the inclusion criteria. ATMPs for rare blood diseases, such as tisagenlecleucel and axicabtagene ciloleucel, were found to be cost-effective in a majority of studies, with incremental QALYs ranging from 1.5 to 10 per patient over lifetime horizon. Tisagenlecleucel demonstrated a positive cost-effectiveness profile in the treatment of acute lymphoblastic leukemia (58%), while axicabtagene ciloleucel showed a positive profile in the treatment of diffuse large B-cell lymphoma (85%). Onasemnogene abeparvovec for spinal muscular atrophy (SMA) showed uncertain cost-effectiveness results, and voretigene neparvovec for retinal diseases was not cost-effective in 40% of studies, with incremental QALYs around 1.3 and high costs exceeding the WTP threshold set. Conclusions: ATMPs in treating rare diseases show promising economic potential, but cost-effectiveness varies across indications. Policymakers must balance innovation with system sustainability, using refined models and the long-term impact on patient outcomes. Full article

Hypercholesterolemia is a major modifiable risk factor for atherosclerotic cardiovascular disease (ASCVD), affecting a significant proportion of the adult population worldwide. This narrative review provides a comprehensive and up-to-date overview of hyperlipidemia management, spanning from epidemiological trends and underlying pathophysiological mechanisms to the limitations of conventional therapies such as statins and ezetimibe. Particular emphasis is placed on cardiovascular risk assessment, current stratification tools, and international guideline-based interventions. The present paper, focusing primarily on the biological mechanisms of dyslipidemia and the clinical use of traditional lipid-lowering agents, serves as the first part of a two-part series, preceding a forthcoming review of novel pharmacological approaches. Our data synthesis is based on a structured literature search conducted across Google Scholar, PubMed, and Scopus, including studies published up to June 2025. The review also includes aspects related to non-pharmacological strategies, pharmacoeconomic considerations, and pharmacogenetic influences on treatment response. Ultimately, this work aims to equip clinicians with evidence-based, nuanced insights essential for optimizing lipid management and reducing cardiovascular risk, while setting the foundation for understanding how emerging therapies may overcome current therapeutic limitations. Full article

Neuromuscular blocking agents such as atracurium and rocuronium are commonly used during anesthetic procedures in laboratory pigs. However, species-specific dosing guidelines remain limited, leading to reliance on data extrapolated from other species. This prospective, blinded study aimed to determine the equipotent dose for atracurium (A) and rocuronium (R) in laboratory pigs receiving propofol and to compare their cost-effectiveness. Twelve healthy animals were randomly distributed according to the drug administered (n = 6 per group). For both drugs, the infusion rate was adjusted following an up-and-down titration to maintain a train-of-four count between 3 and 4. Group differences were analyzed using the Wilcoxon signed-rank test. The bolus induction dose (mg/kg) was comparable between atracurium (2.3 [1.8–2.6]) and rocuronium (2 [2]), while atracurium was associated with higher costs (CHF/kg: A, 1.122 [0.878–1.366] versus R, 0.208 [0.208–0.208]; p = 0.002725). The maintenance infusion rate (mg/kg/h) was approximately 40% lower for atracurium (2.7 [2.5–2.8]) than for rocuronium (4.5 [4.4–4.5]; p = 0.004922), yet the maintenance cost (CFH/kg/h) remained higher for atracurium (A: 1.30 [1.22–1.37] versus R: 0.47 [0.45–0.47]; p = 0.0043). This study reports higher doses for anesthetized pigs compared to other species and demonstrates that rocuronium offers superior cost-effectiveness compared to atracurium under these experimental conditions. Full article

Background/Objectives: Fixed-dose combinations (FDCs) offer significant advantages for patients and healthcare systems by improving adherence and reducing pill burden. However, developing multi-drug formulations remains challenging due to complexities in drug compatibility, stability, and dissolution behavior. The COVID-19 pandemic has necessitated innovative therapeutic approaches. This study aims to develop and evaluate an FDC containing FR (an antiviral drug) and RT (a PDE4 inhibitor) for potential COVID-19 treatment. Methods: The proposed dual-layer FDC was formulated to achieve immediate release of RT using Klucel EXF and controlled release of FR using a combination of Klucel HXF and Compritol ATO888. Critical quality attributes, including drug–excipient compatibility, solid-state properties, tablet uniformity, and dissolution kinetics, were assessed. RT and FR quantification methods were developed and validated per international guidelines. Compatibility studies were conducted by combining excipients in fixed ratios with APIs, followed by stability testing. Results: No degradation or adverse interactions were observed between APIs and excipients. RT exhibited rapid dissolution within 30 min, while FR release was effectively controlled through a gel-forming matrix and lipid barrier. Bulk powder and tablet physical parameters met pharmacopeial standards, and content uniformity between layers was maintained. The formulation demonstrated a stable dissolution profile for both drugs, ensuring consistent drug release. Conclusions: The novel FDC of RT and FR exhibits favorable physicochemical properties, a stable dissolution profile, and potential for improved treatment efficacy in COVID-19 patients. By optimizing drug release mechanisms and ensuring formulation stability, this FDC could serve as a pharmaco-economically viable alternative to existing therapies, enhancing patient compliance and treatment outcomes. Full article

Background and aims: We investigated parenteral nutrition (PN) containing fish oil (FO-PN) vs. standard PN without fish oil (NF-PN) in adult patients hospitalized in the non-intensive care unit (general ward). Methods: Searches in Medline, Embase, and Web of Science (any date to 10 October 2024) were screened, data were extracted, and the quality of the studies was assessed by two independent researchers. Meta-analyses were performed, with outcomes included in random effects models, and heterogeneity for clinical outcomes was explored via subgroup analyses and meta-regression. Pharmacoeconomic analyses used data from the current meta-analysis. Results: In this study, 29 randomized controlled trials (RCTs) were included, with intervention and control groups given FO-PN and NF-PN, respectively, as part of PN covering ≥70% energy provision. Compared to NF-PN, FO-PN was associated with a 37% lower relative risk (RR) of infection (19 RCTs; RR 0.63, 95% confidence interval [CI] 0.50–0.78; p < 0.0001), 2.03 days shorter length of hospital stay (18 RCTs; 95% CI 1.23–2.84; p < 0.00001), and a 51% reduction in the risk of sepsis (10 RCTs; RR 0.49, 95% CI 0.32–0.74; p = 0.0009). There was a non-significant 54% reduction in the 30-day mortality rate (11 RCTs; RR 0.46, 95% CI 0.20–1.08; p = 0.07) for FO-PN. FO-PN was associated with better clinical outcomes and financial savings (i.e., dominance) compared to NF-PN in all five countries studied. Conclusions: FO-PN is a cost-effective option compared to NF-PN for adult patients hospitalized in a general ward across a range of healthcare systems. Full article

Backgrounds: Designed to measure symptoms and quality-of-life impacts, the chronic rhinosinusitis patient-reported outcome (CRS-PRO) questionnaire is a novel instrument tailored to CRS patients. This study aimed to translate the CRS-PRO into Hebrew, adapt it cross-culturally, and assess its reliability and validity. Methods: A prospective study was conducted with 127 participants across three groups: CRS, functional endoscopic sinus surgery (FESS), and control groups (healthy individuals). Participants completed the Hebrew CRS-PRO at baseline and one month later. The Hebrew version was developed according to the International Society for Pharmacoeconomics and Outcomes Research guidelines for translation and cross-cultural adaptation. Results: Of the 127 participants (mean age 47.3 ± 17.7 years, range 18–93), 77 were males (60.6%), and 50 were females (39.4%). The Hebrew CRS-PRO demonstrated high internal consistency (Cronbach’s alpha 0.936) and strong discriminant validity among the three groups. Baseline mean scores were 7.2 for the control group, 25.2 for the FESS group, and 27.1 for the CRS group, which subsequently decreased to 6.5, 12.9, and 20.4, respectively, after one month (ANOVA, p < 0.001). Test–retest reliability, supported by Pearson’s correlation (p < 0.01) and intraclass correlation (p < 0.0001), demonstrated the questionnaire’s effectiveness in identifying CRS-related symptoms and monitoring improvement after FESS. Conclusions: The adaptation and validation of the CRS-PRO into Hebrew resulted in a reliable instrument in patients with CRS. It exhibited robust reliability, internal consistency, and strong discriminant validity, effectively differentiating between healthy individuals and CRS patients and those who are pre- and post-FESS. Additionally, the Hebrew CRS-PRO questionnaire may be effective for evaluating patients before and after FESS surgery. Full article

Background/Objectives: Bempedoic acid (BA) is a novel lipid-lowering agent that reduces low-density lipoprotein cholesterol (LDL-c) and cardiovascular events. Limited real-world data on its effectiveness and safety are available. This study aimed to evaluate the utilization and clinical performance of BA in routine clinical practice. Moreover, an explorative pharmacoeconomic analysis was performed. Methods: We prospectively enrolled consecutive patients with dyslipidemia who started 180 mg BA, alone or with 10 mg ezetimibe, across five outpatient clinics in Campania Region, Italy from September to December 2023. Clinical and laboratory assessments, including lipid profile, hepatic function, and creatine phosphokinase levels, were performed at baseline and at least after one month follow-up. Side effects were recorded. Results: 111 patients (age 65 ± 9 years, 61% male) were included. At BA initiation, 70.3% were on maximally tolerated statin dosage and ezetimibe, 16.2% on ezetimibe alone, and 13.5% on PCSK9 inhibitors due to statin intolerance. BA significantly reduced LDL-c serum levels (89.9 ± 33.0 vs. 56 ± 27.6 mg/dL; p < 0.0001), with 46% achieving therapeutic targets. LDL-c decreased by 28% in patients on intensive statins/ezetimibe and by 45% in statin-intolerant patients, with reduced healthcare costs. Side effects were infrequent (10%) and reversible. Adherence was 99%, and persistence 90%. Conclusions: In our clinical pratice, BA was primarily used in high-risk patients with dyslipidemia who failed to reach LDL-c therapeutic target with statins/ezetimibe, and to a lesser extent, in statin-intolerant individuals. BA treatment enabled 54% to reach LDL-c therapeutic target. BA was well tolerated, and showed high adherence and persistence, contributing to cost savings. Full article

Background: The chronic rhinosinusitis patient-reported outcome (CRS-PRO) is a newly developed, disease-specific questionnaire designed for patients with CRS. This study focused on translating the CRS-PRO into Arabic, conducting cross-cultural adaptation and validation of the questionnaire, and assessing its reliability and validity. Methods: This prospective study involved 112 patients divided into CRS, functional endoscopic sinus surgery (FESS), and control groups. Participants completed the questionnaire at enrollment and again after one month. The Arabic version of the CRS-PRO was created following the International Society for Pharmacoeconomics and Outcomes Research guidelines for translation and cross-cultural adaptation. Results: This study included 74 males (66.1%) and 38 females (33.9%), with an average age of 37.4 ± 14.8 years. The Arabic CRS-PRO questionnaire has high internal consistency and reliability (Cronbach’s alpha 0.97). It also has strong discriminant validity in distinguishing between groups (ANOVA, p < 0.001). The assessment of test/retest symptom scores and their consistency over time confirmed the reliability of the CRS-PRO in differentiating CRS patients from healthy individuals and in monitoring surgical outcomes. This was validated through Pearson’s correlation coefficients (p < 0.01) and intraclass correlation (p < 0.0001). Conclusions: The Arabic version of the CRS-PRO proved simple, reliable, and valid. It showed high internal consistency, reliability, and strong discriminant validity in distinguishing between healthy individuals, CRS patients, and those pre- and post-FESS. Full article

Background/Objectives: Psoriasis is a chronic inflammatory condition mediated by the immune system with various manifestations. The increased prevalence of subclinical joint involvement has led to the development of early diagnostic methods for psoriatic arthritis, including several instruments that have been validated and used in clinical practice. The aim of this study was to perform the Romanian translation, cultural adaptation, and validation of three assessment tools: the Early Arthritis for Psoriatic Patients (EARP) Questionnaire, Psoriasis Epidemiology Screening Tool (PEST), and Toronto Psoriatic Arthritis Screen 2 (TOPAS 2), which are designed to evaluate early-stage arthritis in patients with psoriasis. Methods: All the activities were carried out in accordance with the internationally recognized methodology recommended by the International Society for Pharmacoeconomics and Outcome Research (ISPOR), the recommendations of the World Health Organization (WHO) regarding the translation process and the validation of instruments, and data from the international literature. These three questionnaires were administered to 29 patients with psoriasis diagnosed by biopsy. A descriptive study was conducted and the data were analyzed with appropriate statistical tests using the PSPP program. A reliability test was assessed using Cronbach’s alpha coefficient. Results: The obtained values were significant for the first two questionnaires, with a value of 0.89 for the EARP and 0.63 for the PEST, but the value was not as significant for ToPAS2, at 0.40. Conclusions: This pilot study revealed that the Romanian and original versions of the three questionnaires are similar. Full article

Background: Clostridioides difficile infection (CDI) represents a prevalent and potentially severe health concern linked to the usage of broad-spectrum antibiotics. The aim of this study was to evaluate a new lyophilized product based on human fecal microbiota for transplant, including cost–benefit analysis in the treatment of recurrent or refractory CDI. Methods: The product for fecal microbiota transplant was obtained from two donors. Microbiological, viability, and genomic analysis were evaluated. After validation, a clinical pilot study including recurrent or refractory CDI with 24 patients was performed. Clinical response and 4-week recurrence were the outcome. Cost–benefit analysis compared the fecal microbiota transplant with conventional retreatment with vancomycin or metronidazole. Results: The microbiota for transplant presented significant bacterial viability, with and adequate balance of Firmicutes and Bacteroidetes. The clinical response with the microbiota transplant was 92%. In financial terms, estimated expenditure for CDI solely related to recurrence, based on stochastic modeling, totals USD 222.8 million per year in Brazil. Conclusions: The lyophilized human fecal microbiota for transplant is safe and can be an important step for a new product with low cost, even with genomic sequencing. Fecal microbiota transplantation emerges as a more cost-effective alternative compared to antimicrobials in the retreatment of CDI. Full article

Breast cancer is the leading cause of cancer-related death in women worldwide and the fifth most common cause of cancer death overall. Most women with breast cancer have a good prognosis if the cancer is detected at an early stage and the patients have access to the appropriate treatment and disease management. This study aims to evaluate the impact of pharmacist-led interventions on breast cancer management and health outcomes. A literature review was carried out through the scientific databases PubMed, Scopus, and Web of Science using predefined keywords. Only full-text original articles written in English that investigated the role of the pharmacist in the management of breast cancer were included in the final analysis. No publication date limits were set. A total of 1625 articles were retrieved from the electronic databases, of which 14 met the inclusion criteria. The current scoping review consists of different study types, including randomized controlled trials, cross-sectional studies, pre-post studies, retrospective cohort studies, quality improvement projects, case-control studies, and one pharmacoeconomic study. Pharmacists commonly provided the following interventions: consultations regarding chemotherapy treatment, risk assessment and patient education, adverse drug reactions and drug-drug interactions detection, and adherence assessment. This scoping review highlights the beneficial effects of the involvement of pharmacists in breast cancer management, such as better quality of life, reduced drug interaction risk, greater adherence rates, and improved patient knowledge. This confirms the importance of including the pharmacist in the oncology team caring for patients with breast cancer. Full article

Pharmacogenomics (PGx) is the hope for the full optimization of drug therapy while minimizing the accompanying adverse drug events that cost billions of dollars annually. Since years before the century, it has been known that inter-individual variations contribute to differences in specific drug responses. It is the bridge to what is well-known today as “personalized medicine”. Addressing the drug’s pharmacokinetics and pharmacodynamics is one of the features of this science, owing to patient characteristics that vary on so many occasions. Mainly in the liver parenchymal cells, intricate interactions between the drug molecules and enzymes family of so-called “Cytochrome P450” occur which hugely affects how the body will react to the drug in terms of metabolism, efficacy, and safety. Single nucleotide polymorphisms, once validated for a transparent and credible clinical utility, can be used to guide and ensure the succession of the pharmacotherapy plan. Novel tools of pharmacoeconomics science are utilized extensively to assess cost-effective pharmacogenes preceding the translation to the bedside. Drug development and discovery incorporate a drug-gene perspective and save more resources. Regulations and laws shaping the clinical PGx practice can be misconceived; however, these pre-/post approval processes ensure the product’s safety and efficacy. National and international regulatory agencies seek guidance on maintaining conduct in PGx practice. In this patient-centric era, social and legal considerations manifest in a way that makes them unavoidable, involving patients and other stakeholders in a deliberate journey toward utmost patient well-being. In this comprehensive review, we contemporarily addressed the scientific leaps in PGx, along with various challenges that face the proper implementation of personalized medicine in Egypt. These informative insights were drawn to serve what the Egyptian population, in particular, would benefit from in terms of knowledge and know-how while maintaining the latest global trends. Moreover, this review is the first to discuss various modalities and challenges faced in Egypt regarding PGx, which we believe could be used as a pilot piece of literature for future studies locally, regionally, and internationally. Full article

According to data from the World Health Organization (WHO), cancer is considered to be one of the leading causes of death worldwide, and new therapeutic approaches, especially improved novel cancer treatment regimens, are in high demand. Considering that many chemotherapeutic drugs tend to have poor pharmacokinetic profiles, including rapid clearance and limited on-site accumulation, a combined approach with tumor-homing peptide (THP)-functionalized magnetic nanoparticles could lead to remarkable improvements. This is confirmed by an increasing number of papers in this field, showing that the on-target peptide functionalization of magnetic nanoparticles improves their penetration properties and ensures tumor-specific binding, which results in an increased clinical response. This review aims to highlight the potential applications of THPs in combination with magnetic carriers across various fields, including a pharmacoeconomic perspective. Full article

Viral pathogens pose a substantial threat to public health and necessitate the development of effective remediation and antiviral strategies. This short communication aimed to investigate the antiviral efficacy of disinfectants on the surface proteins of human pathogenic viruses. Using in silico modeling, the ligand-binding energies (LBEs) of selected disinfectants were predicted and combined with their environmental impacts and costs through an eco-pharmaco-economic analysis (EPEA). The results revealed that the binding affinities of chemical disinfectants to viral proteins varied significantly (p < 0.005). Rutin demonstrated promising broad-spectrum antiviral efficacy with an LBE of −8.49 ± 0.92 kcal/mol across all tested proteins. Additionally, rutin showed a superior eco-pharmaco-economic profile compared to the other chemicals, effectively balancing high antiviral effectiveness, moderate environmental impact, and affordability. These findings highlight rutin as a key phytochemical for use in remediating viral contaminants. Full article

Background: The main aim of this study is to perform a cross-cultural adaptation and validation of the Visual Analogue Scale for the Foot and Ankle (VASFA) questionnaire, creating a Spanish-language version (VASFA-Sp), and to determine the measurement properties of this instrument. Methods: VASFA was cross-culturally translated into Spanish following the guidelines of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). The study sample was composed of 228 participants who were recruited from February to May 2022. All were at least 18 years old, gave signed informed consent to take part and properly completed the Foot and Ankle Ability Measures-Sp and VASFA-Sp questionnaires. Cronbach’s alpha and test/re-test reliability values were calculated. Structural validity was assessed via exploratory factor analysis. Results: The 228 patients included in the final analysis presented the following characteristics: 35.53% were male and 64.47% were female; the mean age was 35.95 (18–81) years; and the mean body mass index was 23.79. Internal consistency was excellent. The Cronbach’s alpha for VASFA-Sp was 0.96 and the intraclass correlation coefficient was 0.932 (95% CI; 0.84 to 0.97). Exploratory factor analysis identified one main factor. Conclusions: VASFA-Sp is a reliable, valid and sensitive questionnaire that is suitable for measuring perceived foot and ankle function impairment in a Spanish-speaking population. Full article