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Background The number of orphan drug approvals is currently increasing globally. This creates a significant burden on payers and healthcare systems. This study aimed to create a multi-criteria decision analysis (MCDA) tool for evaluating orphan drugs within the United Arab Emirates (UAE). The intended result of the tool is to provide evidence-based guidance to decision-makers in reimbursement and procurement decisions. Methods We conducted a literature search and local expert interviews to identify relevant preliminary criteria for the MCDA tool. Then we conducted a structured consensus-building session for healthcare experts and decision-makers in the UAE to develop the Emirati MCDA tool for orphan drugs. The experts voted for the criteria to be included in the tool and their ranking according to importance, as well as the weight of each criterion and its scoring function. To improve understanding and facilitate the voting process, experts were provided with a brief illustration of similar tools conducted in other countries before the voting sessions. Finally, the tool was developed in a Microsoft Excel sheet (Microsoft Corporation, Redmond, Washington, United States), and it was validated and tested based on real case studies, then it was fine-tuned accordingly based on the experts' discussions. The final tool was provided to the attendees to guide their decisions in the reimbursement and procurement of orphan drugs. Results The created tool provides a score for each analyzed orphan drug based on its value. Ten criteria were included in the final MCDA tool. These were cost-effectiveness (25.1% of the weight), magnitude of health gain (20.1%), availability of therapeutic alternative (14.3%), disease severity (11%), budget impact (7.9%), disease rarity (5.6%), strength of clinical evidence (5.6%), burden on households (4.5%), indication uniqueness (3.2%), and patients' age (2.6%). Conclusions Implementation of evidence-based healthcare necessitates assessing the fair value of each health technology. Addressing the high unmet medical needs and improving healthcare for patients with rare diseases are priorities within the UAE. The created Emirates MCDA tool for orphan drugs has the potential to help decision-makers implement value-based and evidence-based reimbursement decisions for orphan drugs.
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OBJECTIVES: Haemodynamic changes following intravenous acetaminophen are well studied in adults. Limited data are published in critically ill paediatric patients, especially from the Middle East. We aim to investigate haemodynamic effects and incidence of hypotension with intravenous acetaminophen in critically ill children, with a focus on understanding factors influencing these effects. METHODS: We retrospectively reviewed patients who received intravenous acetaminophen between July and December 2022. A haemodynamic event was defined as drop of >15% in systolic blood pressure (SBP) or mean arterial blood pressure (MAP) within 120 min after drug administration. Hypotension was defined as either drop in SBP below the 5th percentile for age, or a haemodynamic event associated with tachycardia, increased lactate or treatment with fluid/vasopressors. Logistic regression was performed to quantify relationships between patients' characteristics and the occurrence of haemodynamic event and hypotension. RESULTS: A haemodynamic event was observed in 50/156 patients (32%) post-acetaminophen. Mean MAP (SD) before and after acetaminophen was 69.6 mm Hg (14.8) and 67.4 mm Hg (13.9), respectively (p=0.001). Mean SBP (SD) before and after acetaminophen was 95.4 mm Hg (18.2) and 92.8 mm Hg (19.2), respectively (p=0.006). Baseline MAP, median (interquartile range (IQR)) was 76.0 (64.0-85.3) and 66.0 (57.0-74.5) in patients with and without haemodynamic events, respectively (p=0.004). Only 38/156 patients (24%) met the definition for hypotension. Baseline MAP, median (IQR) was 62.0 (51.8-79.0) in patients with, and 68.5 (62.0, 79.3) in patients without hypotension (p=0.036). Baseline shock, vasoactives, mechanical ventilation and paediatric sequential organ failure assessment were not significantly associated with hypotension. Only MAP was found to be associated with both haemodynamic event (adjusted odds ratio (AOR) 1.05, 95% CI 1.02-1.10) and hypotension (AOR 1.06, 95% CI 1.02-1.10) even after controlling for other confounders. CONCLUSIONS: Administration of intravenous acetaminophen in critically ill children can lead to haemodynamic changes, including clinically significant hypotensive events.
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Introduction The establishment of second-generation antipsychotics in the early 1900s has shown greater benefits in many outcome domains. As atypical antipsychotics revealed an improvement in the shortcomings of first-generation antipsychotics, they also have significant limitations in terms of side effects. Background As a class, atypical antipsychotics have a more satisfactory profile in terms of extrapyramidal side effects but have other side effects, including metabolic syndrome. Moreover, a patient with severe mental illness has reduced life expectancy compared to the general population and increased dying risk by two to three folds due to the associated risk of metabolic syndrome. Metabolic syndrome induced by second-generation antipsychotics requires routine monitoring, suitable intervention, and management, including lifestyle modification, caregiver education, and the use of other medications like lipid-lowering agents, anti-diabetics, and adjunctive therapies. Method This is a retrospective observational study that involves a review of medical records of adult patients attending the psychiatry outpatient clinics in Shaikh Khalifa Medical City to whom oral second-generation atypical antipsychotics were prescribed between January 2016 and December 2017. All pediatric patients were excluded, as well as those who are on other agents known to induce metabolic syndrome, including first-generation antipsychotics, mood stabilizers, anxiolytics, and anti-depressants. Besides, a patient who already developed metabolic syndrome before starting atypical antipsychotics was excluded as well. IBM SPSS v. 25 (IBM Corp., Armonk, NY) and Jamovi 1.0.4.0 (The jamovi project (2021). [Computer Software]. Retrieved from https://www.jamovi.org) were used for statistical analysis. The Shapiro-Wilk was used to test for normality of data, One-way repeated measure analysis of variance (ANOVA) was used to determine whether there are any statistically significant differences between the means of BMI across the three pre-specified time points, Kendall's tau was used to test the correlation between categorical variables, and the paired t-test was used to determine whether there was a statistically significant mean difference between BMI at baseline and after one year, a p-value above 0.05 is considered non-significant. Result A total of 123 patients were included in this study out of 4123 patients. An olanzapine disintegrating tablet was the most used atypical antipsychotic among the study population, followed by risperidone and quetiapine, respectively. Furthermore, BMI was statistically significantly increased from baseline to six months (M = 2.37 kg/m2, p = .002), and from baseline to 12 months (M =3.26 kg/m2, p < .001) but not from six months to 12 months (M =1.41 kg/m2, p = .346). Due to lack of documentation and monitoring, it was difficult to assess the continuous change in total cholesterol level/high-density lipoprotein, glucose, and HbA1C level. It was observed that nine (7.32%) patients of those who have been included in the study started a treatment to manage the metabolic syndrome. Conclusion Patients on second-generation antipsychotics must be monitored and treated in case of metabolic syndrome development to decrease the high risk of mortality in such patients. Documentation of such complications, patient treatment, and progress is essential for outcome improvement.