DRUGS - REAL WORLD OUTCOME
Drugs real world outcomes: Latest results from PubMed
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Unmanned aerial vehicles in prehospital emergency care: current evidence, technological advances, and future perspectives
CONCLUSION: UAVs demonstrate significant potential to enhance the speed, coverage, and coordination of prehospital emergency care. Future research should focus on integrating UAVs within established emergency medical service networks, developing unified policy and airspace regulations, and validating cost-effectiveness and clinical impact through large-scale, real-world studies. -
Treatments for renal cell carcinoma: NICE Pilot Treatment Pathways Appraisal
CONCLUSIONS: This pilot demonstrated the feasibility of producing a reference model, which is open source and available to relevant stakeholders without restriction. This will improve consistency in the National Institute for Health and Care Excellence's decision-making and allow for the evaluation of optimum treatment sequences for advanced renal cell carcinoma. -
Treatment strategies for motor fluctuations in Parkinson's disease: a systematic review of efficacy, functionality, and drug accessibility with a focus on Latin America
CONCLUSION: While multiple pharmacological and device-based interventions effectively reduce OFF-time in Parkinson's disease, their real-world impact is constrained by uneven global access and affordability. The Latin American region exemplifies these disparities, with limited regulatory availability, heterogeneous pricing, and insufficient inclusion of novel agents in national formularies. Integrating efficacy evidence with accessibility analyses highlights the need for coordinated regional... -
Restoring the Sense of Smell: A Systematic Review and Meta-Analysis of Biologic Therapies for Chronic Rhinosinusitis with Nasal Polyps
PURPOSE OF REVIEW: Smell loss is among the most debilitating symptoms of chronic rhinosinusitis with nasal polyps (CRSwNP). We aimed to synthesize current evidence on the smell-restoring efficacy of biologics, focusing on outcomes measured by psychophysical tests. -
Real-world effectiveness and safety of galcanezumab for the treatment of migraine: A systematic review and meta-analysis
CONCLUSION: Galcanezumab appears to be associated with clinically meaningful improvements in migraine and favorable safety outcomes, although the evidence certainty is limited by heterogeneity. -
Machine learning for predicting treatment response to biologic and targeted synthetic disease-modifying antirheumatic drugs in rheumatoid arthritis: a scoping review
CONCLUSION: ML methods are increasingly used to predict RA treatment response, but vary widely in methodology and performance. Standardization, external validation, and transparent reporting are critical for advancing clinical application. -
Comparative outcomes of aflibercept biosimilars and reference aflibercept in nAMD: a systematic review and meta-analysis
CONCLUSION: This systematic review and meta-analysis found no statistically significant differences in functional, anatomical or safety outcomes between aflibercept biosimilars and the reference product for nAMD, based on moderate to high certainty evidence. Functional and anatomical outcomes appeared stable across multiple timepoints. Further long-term pharmacovigilance studies and real-world data beyond 56 weeks are warranted. -
The association of direct-acting antiviral therapy with health care utilization and costs in patients with chronic hepatitis C virus infection: A systematic review of real-world studies in the United States
CONCLUSIONS: This systematic review reveals significant gaps in the real-world economic evaluation of DAA therapy. In particular, longitudinal data, geographic granularity, and state-specific analyses are lacking. Future research should address these limitations and further explore the long-term impacts of DAA therapy and variations in access and costs across different populations and insurance programs. -
Drugs for transthyretin amyloidosis under the microscope: Survival, safety, and a meta-analysis with certainty of evidence assessment
Transthyretin amyloidosis (ATTR) is an infiltrative disease caused by the deposition of misfolded transthyretin (TTR) fibrils in organs and tissues, with incidence and prevalence rapidly increasing worldwide. Current therapeutic strategies fall into two main classes: TTR stabilizers and TTR gene silencers. To date, no comprehensive reviews cover all the available pharmacological treatments for ATTR, both approved and off-label. In addition, previous meta-analyses have often excluded real-world... -
Strategies to Improve PrEP Adherence in People Who Use Drugs: A Systematic Review
People who use drugs (PWUD) face a disproportionately high risk of HIV, accounting for a considerable share of cases in the United States. Pre-exposure prophylaxis (PrEP) has been shown to be effective in preventing HIV transmission through both sexual contact and injection drug use. However, adherence is essential for real-world success. Following PRISMA guidelines, we conducted the first systematic review to examine PrEP interventions among PWUD and identify strategies to enhance adherence.... -
A systematic review of real-world evidence on the clinical relevance, characterization, and utility of CYP2D6 biomarker testing
Pharmacogenomic (PGx) research investigates how an individual's genetic make-up impacts their drug metabolism. PGx testing can therefore inform therapeutic decision-making, especially as compelling evidence develops over time to substantiate its clinical and personal utility across a range of therapeutic areas. PGx biomarker CYP2D6, in particular, is widely implicated in drug metabolism and across several therapeutic areas. Real-world evidence (RWE) derived intentionally using electronic health... -
Current Evidence on Celecoxib Safety in the Management of Chronic Musculoskeletal Conditions: An Umbrella Review
CONCLUSIONS: Celecoxib appears to offer better gastrointestinal safety than non-selective NSAIDs. Although data on cardiovascular, renal, and mortality outcomes suggest possible advantages, the evidence remains limited and of low certainty. Moreover, some real-world evidence raises concerns in specific high-risk populations. Future research should integrate data from both randomized trials and observational studies to better inform long-term safety assessments and guide individualized treatment... -
Safety profiles of the new target therapies-pemigatinib, futibatinib, and ivosidenib-for the treatment of cholangiocarcinoma: a systematic review
CONCLUSION: FGFR2 and IDH1 inhibitors have good tolerability in the population examined. All AEs were optimally managed with dose modulation. Future studies should focus on identifying the most effective dosages to further enhance treatment safety. -
Therapeutic efficacy of thiocolchicoside-nonsteroidal anti-inflammatory drug combination in pain management: A systematic review and cross-sectional real-world study
CONCLUSION: Evidence indicates that multimodal therapy, including parenteral TCC with NSAIDs, provides quicker and effective pain relief, reduces muscle spasms, and improves hand-to-floor distance compared to using NSAIDs or TCC alone. -
Long-Term Safety of Anti-Interleukin-1 Medications in Children with Rheumatic Diseases: a Systematic Review
CONCLUSIONS: Our results are consistent with the known safety profile of anti-IL-1 medications and show that they are generally safe for use in the context of childhood rheumatic and autoinflammatory diseases. This review of clinical trial and real-world data will help inform clinical decision-making and family counselling when initiating anti-IL-1 medications in children. -
Comparative effectiveness and safety of biosimilars versus reference biologics in rheumatoid arthritis during treatment initiation: a systematic review of real-world evidence
CONCLUSION: In real-world settings, biosimilars generally demonstrate comparable effectiveness to originators. Future investigations are warranted to examine the comparative safety profiles of biosimilars and originators. -
Interventions to improve adherence to lipid-lowering drugs: a systematic review and meta-analysis
BACKGROUND: Poor medication adherence to lipid-lowering medications (LLMs) remains a barrier in reducing the burden of cardiovascular disease (CVD). We sought to review interventions aimed at improving adherence to LLMs for the primary or secondary prevention of CVD, and evaluate their effectiveness on patient outcomes. -
Persistence with anti-dementia medications: a systematic review and meta-analysis
CONCLUSION: The meta-analysis demonstrated relatively low persistence to ADDs, which varied according to the evaluation criteria used. Targeted interventions to improve persistence with therapy may lead to better outcomes in patients with dementia. Also, a standardised framework for measuring persistence could improve research reliability. -
Economic and developmental impacts of FDA designations: a systematic review and meta-analysis
CONCLUSIONS: FDA designations provide economic advantages, especially for smaller companies, and can expedite approvals for high-priority therapies. Notable heterogeneity, particularly with RMAT, warrants further research to clarify how disease area and company size shape real-world outcomes. -
Dronedarone vs Sotalol Among Patients With Atrial Fibrillation: A Meta-Analysis of Retrospective Observational Databases
CONCLUSIONS: In this retrospective meta-analysis, dronedarone exhibited significantly lower rates of CV hospitalization and ventricular arrhythmias compared with sotalol. These findings provide real-world evidence to support selection of the most appropriate first-line AAD for rhythm control in patients with AF. -
Real-World Evidence for Baricitinib in the Treatment of Rheumatoid Arthritis in Spain: A Systematic Literature Review
CONCLUSION: These results suggest baricitinib is effective in the real-world setting in Spain, with a consistent safety profile, similar to findings reported in clinical studies and in real-world studies conducted in other countries. -
Cardiovascular toxicity induced by immunotherapy in non-small cell lung cancer: a systematic review and meta-analysis of observational studies
CONCLUSIONS: The treatment with ICIs caused a higher rate of CV events compared to non-ICI treatments. Nevertheless, further research is required to elucidate the underlying mechanisms and implications for patient care. This calls for continued research efforts to optimize the cardiovascular health of patients undergoing immunotherapy for lung cancer. -
Investigating the Relationship Between Anti-seizure Medications and Bleeding Disorders: A Comprehensive Review of the Current Literature
Anti-seizure medications (ASMs) are specific types of anticonvulsants used to treat epileptic seizures. However, several studies have shown an association between ASMs and an increased risk of hematological disorders, such as thrombocytopenia, aplastic anemia, and platelet function disorders leading to prolonged bleeding times. This review explores the existing literature on this topic, investigating a wide variety of ASMs, ranging from first-generation medications to newer ones. A comprehensive... -
Evaluation of real-world evidence to assess health outcomes related to deprescribing medications in older adults: an International Society for Pharmacoepidemiology-endorsed systematic review of methodology
Observational studies using real-world data (RWD) can address gaps in knowledge on deprescribing medications but are subject to methodological issues. Limited data exist on the methods employed to use RWD to measure the effects of deprescribing. To describe methodological approaches used in observational studies of deprescribing medications in older adults, we conducted a systematic review in Medline for observational studies published in English (January 1, 2000, to September 14, 2023) that... -
Exploiting blood-based biomarkers to align preclinical models with human traumatic brain injury
Rodent models are important research tools for studying the pathophysiology of traumatic brain injury (TBI) and developing new therapeutic interventions for this devastating neurological disorder. However, the failure rate for the translation of drugs from animal testing to human treatments for TBI is 100%. While there are several potential explanations for this, previous clinical trials have relied on extrapolation from preclinical studies for critical design considerations, including drug dose... -
Comparative Cardiovascular Safety of Gonadotropin-releasing Hormone Antagonists and Agonists Among Patients Diagnosed with Prostate Cancer: A Systematic Review and Meta-analysis of Real-world Evidence Studies
CONCLUSIONS AND CLINICAL IMPLICATIONS: Real-world evidence studies indicate that degarelix, compared with GnRH agonists, is associated with a modest increased risk of MACEs, particularly among patients with a history of cardiovascular disease. However, residual confounding due to the treatment of high-risk patients with degarelix may account for these findings. Additional large studies with detailed data on tumor characteristics and cardiovascular risk factors are needed to confirm these... -
Comparative Effectiveness and Safety of Direct Oral Anticoagulants in Low Body Weight Patients with Atrial Fibrillation: A Systematic Review and Meta-analysis
INTRODUCTION: Direct oral anticoagulant (DOAC) agents are established as the anticoagulation strategy of choice for a variety of clinical risks. Despite this, uncertainty still exists with regard to their efficacy and safety for the prevention of stroke and systemic embolism in some patient populations; most notably those with low body weight (LBW) (
