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Burosumab is an orphan medicinal product (OMP) approved in Europe for the treatment of X-linked hypophosphatemia (XLH). The aim of this study was to assess the value of burosumab versus conventional therapy for the treatment of paediatric XLH, through a multi-criteria decision analysis (MCDA) framework for health technology assessment (HTA) of OMPs in Portugal.
The MCDA framework considered 14 criteria related to disease burden, therapeutic value and economic burden. A multidisciplinary panel of national stakeholders participated in a two-phase exercise. In the first phase, relative weights and part-worth utilities for the criteria and their levels were elicited and a reimbursement likelihood function was calibrated through adaptive conjoint analysis. In the second phase, burosumab and conventional therapy were assessed against the criteria, providing a global value score (0-100) and reimbursement likelihood (0-100%) for both.
Of the 14 criteria, disease burden, therapeutic value and economic burden criteria represented 27.29%, 57.17% and 15.53% of the total weight in the decision, respectively. All disease burden and some therapeutic value criteria, typically not included in traditional HTA, represented 47.88% of the total weight. Burosumab was unanimously considered superior to conventional therapy, with an average (range) global value score of 84.96 (82.48-86.54) against 48.06 (43.37-57.68), and reimbursement likelihood of 97.50% (96.78%-98.32%) against 43.66% (31.48%-68.73%), respectively.
MCDA represents a powerful tool in HTA decision-making for OMPs. The results of this MCDA acknowledge burosumab as a disease-modifying drug, deemed superior to conventional therapy for the treatment of paediatric XLH.
MCDA represents a powerful tool in HTA decision-making for OMPs. The results of this MCDA acknowledge burosumab as a disease-modifying drug, deemed superior to conventional therapy for the treatment of paediatric XLH.Evidence shows that undernutrition during early life is associated with an increased risk of chronic diseases in adulthood. We aimed to investigate whether exposure to the Chinese famine in the fetal or infant stage was associated with self-reported arthritis risk in adulthood. selleck chemicals A total of 3,622 participants were included in the final analysis. Participants were classified into non-, fetal-, and infant-exposed group. Arthritis was self-reported doctor-diagnosed arthritis. A multivariate logistic regression model was used to calculate the odds ratios (ORs) and 95% confidence intervals (CIs) of arthritis. The prevalence of arthritis was 27.07%, 27.74%, and 34.09% among individuals in non-, fetal-, infant-exposed group, respectively. Infant-exposed group (OR 1.32; 95% CI 1.12-1.56) had a higher arthritis risk then non-exposed group after adjustment for gender, age, area, education level, smoking status, drinking status, and physical activity. Participants who experienced severe famine during infant and fetal period had higher (41.46%, OR=1.71 and 32.94%, OR=1.36) arthritis risk than those exposed to less severe famine. Exposure to the Chinese famine in early life was associated with an increased risk of arthritis in adulthood, which was partially influenced by some factors (e.g., gender, area, body mass index, and born in severely affected area or not).
Current evidence indicates that the pharmacokinetic profile of rivaroxaban is not significantly impacted by body weight. However, real-world data are needed to better assess the potential clinical benefits and risks associated with rivaroxaban in non-valvular atrial fibrillation (NVAF) patients with obesity. Thus, our objectives were to assess the real-world effectiveness and safety of rivaroxaban versus warfarin among NVAF patients with obesity in the US nationally representative commercially-insured population.
Health insurance claims data from the IQVIA PharMetrics Plus database (January 2010-September 2019) were used to identify NVAF patients with obesity (based on diagnosis codes) initiated on rivaroxaban or warfarin. Inverse probability of treatment weighting (IPTW) was used to adjust for imbalances between groups. Study outcomes of interest were evaluated up to 36 months post-treatment initiation and included the composite of stroke or systemic embolism (stroke/SE) and major bleeding. Outcomes were compared using Cox proportional hazards regression models with hazard ratios (HR) and 95% confidence intervals (CIs).
A total of 10,555 patients were initiated on rivaroxaban and 5080 patients on warfarin. Following IPTW, the risk of stroke/SE was 26% lower among patients prescribed rivaroxaban relative to warfarin (HR 0.74, 95% CI 0.60, 0.91,
= .004) at 36 months. Rivaroxaban-initiated patients had a risk of major bleeding similar to that of warfarin-initiated patients (HR 0.85, 95% CI 0.71, 1.02,
= .085).
These results suggest that rivaroxaban is an effective and safe treatment option among NVAF patients with obesity in a commercially-insured US population.
These results suggest that rivaroxaban is an effective and safe treatment option among NVAF patients with obesity in a commercially-insured US population.
Misuse/abuse of pregabalin is increasing worldwide. French Poison Control Centers (PCCs) recently received several unusual calls regarding the recreational use of pregabalin in adolescents. This study aims to describe this new and specific population of pregabalin misusers.
We extracted all cases of pregabalin intentional exposures reported to the French National Database of Poisonings (FNDP) from 2004 to 2020. We compared the proportion of recreational exposure to pregabalin between adolescents (10-17 years) and adults (>18 years). We reviewed all cases of pregabalin recreational exposures in adolescent in order to describe the characteristics of this population.
During the study period, 382 cases of acute intentional exposure to pregabalin were reported in adolescents and 1188 in adults, 94/382 (24.6%) and 43/1188 (3.6%) were pregabalin recreational use, respectively (
< .0001). Almost all cases of pregabalin recreational use in adolescent were reported from 2018 (86/94; 91%). Most of those adolescent patients were males (male/female ratio - 5.
Website: https://www.selleckchem.com/
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