Review Of Real-world Evidence Research Articles

Clinical and Disease Burden of Patients with Generalized Pustular Psoriasis: A Review of Real-World Evidence.

Generalized pustular psoriasis (GPP) is a chronic, rare, and potentially life-threatening disease. There is limited understanding of patient characteristics in GPP and their correlation with disease progression or healthcare resource utilization. Our review aims to examine real-world evidence on these characteristics and the associated disease burden as related to economic and quality of life factors. Results showed that most patients with GPP experienced flares once a year, lasting from 2weeks to 3months, with > 80% of patients having residual disease post-flare, with/without treatment, indicating the long-term nature of GPP. The impact of GPP on patients' daily activities was significant, even in the absence of a flare. GPP adversely affected mental health, and anxiety and depression were reported regularly. Patients with GPP had more comorbidities, were prescribed more medication, and had more inpatient and outpatient visits than in matched plaque psoriasis or general population cohorts. Improving the education of healthcare providers in diagnosing GPP, defining disease flares, and managing the disease, as well as making globally accepted clinical guidelines for GPP treatment available, could help to reduce the burden on patients with GPP. Effective therapies that control and prevent GPP flares and manage chronic disease are needed.

HTA34 A Review of Real-World Evidence in Health Technology Assessment Decision-Making of Gene Therapies in the US, Canada, UK, EU4, and Japan

Evidence submitted for health technology assessments (HTAs) of gene therapies (GTs) are limited and associated with uncertainty. Real-world evidence (RWE) may address these uncertainties, with many health authorities adopting new guidelines and increasingly requesting RWE. Our aim was to evaluate the role of RWE in the HTA decision-making of GTs.

A structured literature review of real world evidence reflecting the current lipid management across Europe

A structured literature review of real world evidence reflecting the current lipid management across Europe

Abstract 15651: The Burden of Nontreatment or Undertreatment Among Non-valvular Atrial Fibrillation Patients With Elevated Stroke Risk: A Systematic Literature Review of Real-world Evidence

Introduction: Global treatment guidelines (e.g., AHA/ACC/HRS, ESC, JCS) recommend treatment with oral anticoagulants (OACs) for patients with non-valvular atrial fibrillation (NVAF) and an elevated stroke risk, defined by CHA 2 DS 2 -VASc score ≥2. However, not all patients with NVAF and an elevated stroke risk receive guideline-recommended therapy. A systematic literature review of observational studies was undertaken based on PRISMA guidelines to identify the body of evidence on untreated and undertreated NVAF among patients with elevated stroke risk, and assess its epidemiological and clinical burden. Methods: An extensive search of MEDLINE, GoogleScholar, the Cochrane Library, relevant conference proceedings, and health technology assessments (HTAs) was conducted for RWE studies. Studies published between 1/2010-4/2020 were included if they evaluated rates of nontreatment or undertreatment in NVAF. Nontreatment was defined as absence of OACs, while undertreatment was defined as treatment with only antiplatelet agents. Studies focusing on valvular AF were excluded. Results: Thirty-nine published studies (13 countries) met inclusion criteria. Rates of nontreatment for patients with elevated stroke risk ranged from 2.0%-51.1% (median: 23.3%), while rates of undertreatment ranged from 10.0%-45.1% (median: 34.6%). Few studies assessed the relationship between nontreatment or undertreatment and clinical outcomes in the evaluated population. Among US outpatients with NVAF and an elevated stroke risk, those undertreated had a higher likelihood of death (HR: 1.22; 95% CI: 1.05-1.41; p=.006) compared to patients treated with OACs. Also, in a separate US study of NVAF patients with varying levels of warfarin exposure, patients with no warfarin exposure had significantly higher rates of ischemic stroke than patients with low adherence (4.41 vs. 1.87 per 100 person-years, p<.001) or high adherence (4.41 vs. 0.72 per 100 person years, p<.001). Conclusion: Rates of nontreatment and undertreatment among NVAF patients with elevated stroke risk remain high and are associated with potentially preventable cardiovascular events and death. Strategies to increase rates of treatment may reduce adverse clinical outcomes.

Evolution of treatment paradigms in neovascular age-related macular degeneration: a review of real-world evidence

The aim of this work was to evaluate the contribution of real-world evidence (RWE) in changing anti-vascular endothelial growth factor (VEGF) therapy treatment practices and improving real-world treatment strategies for...

Calcipotriol/betamethasone dipropionate aerosol foam for the treatment of psoriasis vulgaris: a review of real-world evidence (RWE)

Background Randomized controlled trials (RCTs) demonstrated the advantages of an aerosol foam formulation of calcipotriol/betamethasone dipropionate (Cal/BD) in patients with psoriasis. In this review, we investigated whether such benefits could also be obtained in real-world clinical practice. Methods PubMed was searched for studies reporting real-world data in patients with psoriasis treated with Cal/BD aerosol foam. Results Three large real-world studies with Cal/BD aerosol foam were identified: a prospective non-interventional study from Germany, a medical chart review from the US, and a retrospective non-interventional study from Spain. Some key findings included the following: high levels of adherence to treatment (82–93%); after 4 weeks, about 50% of patients achieved complete/almost complete responses; at final assessment, 85–95% of patients were extremely satisfied/very satisfied/satisfied with Cal/BD aerosol foam; all healthcare providers were satisfied or somewhat satisfied with the efficacy of Cal/BD foam and they reported similar findings for symptom improvements (itch, pain, erythema/redness, flaking, and plaque thickness). Global ratings by investigators/healthcare providers for symptom control, overall efficacy and the emotional status of patients were also very high (75–100%). Conclusion The results from real-world studies undertaken in diverse healthcare systems reinforce the positive findings reported in RCTs with Cal/BD aerosol foam in patients with psoriasis.

Risk of cardiovascular events in patients with hypertriglyceridaemia: A review of real-world evidence.

AimsTo describe the real‐world prevalence and consequences of hypertriglyceridaemia.Materials and methodsWe searched two large patient databases, the National Health and Nutrition Examination Survey (NHANES) database (2007–2014) and the Optum Research Database, as well as electronic medical records from two Kaiser Permanente regions.ResultsThe NHANES data showed that ~26% of US adults, including nearly one‐third of statin users, had at least borderline hypertriglyceridaemia (triglycerides [TGs] ≥1.69 mmol/L), and ~40% of adults with diabetes had levels of ≥150 mg/dL despite statin use. The Optum analyses demonstrated that those with TG levels ≥1.69 mmol/L who were on statins had a significantly increased risk of composite initial major cardiovascular (CV) events (hazard ratio [HR] 1.26, 95% confidence interval [CI] 1.19–1.34; P < 0.001 vs. patients with TGs <150 mg/dL). This was accompanied by increased healthcare utilization and direct healthcare costs (HR 1.12, 95% CI 1.08–1.16; P < 0.001). In the analyses of the Kaiser Permanente records, patients with diabetes and TG levels 2.26–5.64 mmol/L had significantly higher adjusted incidence rates of non‐fatal myocardial infarction (rate ratio 1.30, 95% CI 1.08–1.58; P = 0.006), non‐fatal stroke (rate ratio 1.23; 95% CI 1.01–1.49; P = 0.037) and coronary revascularization (rate ratio 1.21; 95% CI 1.02–1.43; P = 0.027), but not unstable angina (rate ratio 1.33; 95% CI 0.87–2.03; P = 0.185) compared with patients with TG levels <1.69 mmol/L.ConclusionsReal‐world analyses suggest that elevated TGs are prevalent and commonly associated with increased CV risk. CV outcomes trials in patients with established hypertriglyceridaemia will clarify whether strategies to reduce TG levels can ameliorate residual CV risk in patients taking statins.

Seizure management and prescription patterns of anticonvulsants in Dravet syndrome: A multicenter cohort study from Germany and review of literature

ObjectiveThe aim of this study was to describe the treatment pattern of patients with Dravet syndrome (DS) in Germany with routine antiepileptic drugs (AEDs) and emergency medication, and to review the literature of real-world evidence on medicine utilization of patients with DS in Europe. MethodsPatient use of routine AEDs and emergency medications over 3–6 months was analyzed from a 2018 multicenter survey of 93 caregivers of patients with DS throughout Germany. Results were contextualized in a review of real-world evidence on medicine utilization of patients with DS in Europe. ResultsThe variety of medications and the most frequent combinations routinely used by patients with DS (AEDs and others) are described. Patients use a large number of pharmaceutical treatments to manage seizures. The five most commonly used AEDs were sodium valproate (66% of the patients; mean daily dose: 660 mg; 24.5 mg per kg bodyweight), bromide (44%; 1462 mg; 51.2 mg per kg), clobazam (41%; 10.4 mg; 0.32 mg per kg), stiripentol (35%; 797 mg; 27.6 mg per kg), and topiramate (24%; 107 mg; 3.5 mg per kg). Ninety percent had reported using emergency medications in the last 3 months;, with the most common medications being Buccolam (40%, an oromucosal form of midazolam) and diazepam (20%, mostly rectal application). No discernable relationships between current medication and age or seizure frequency were observed. SignificanceThis is the first comprehensive report of routine AEDs and emergency medication use in a large sample of patients with DS in Germany over a period of 3–6 months and shows that despite the most common AED combinations being in line with clinical guidelines/best practice, there is no discernable impact of best treatment on seizure frequency. We find a higher use of bromide in Germany compared with other real-world evidence in Europe.

The Seriousness of Chronic Venous Disease: A Review of Real-World Evidence.

Chronic venous disease (CVD) is a prevalent condition that tends to worsen with age. Patients initially seek treatment to relieve symptoms of leg pain, discomfort, heaviness and swelling, all of which impact their quality of life. As the disease increases in severity to include varicose veins, skin changes, and venous ulcer, the demand for treatment increases while the quality of life further diminishes. The prevalence of CVD is highest in Western countries where it already consumes up to 2% of healthcare budgets. With the aging of the global population, the prevalences of CVD and severe CVD are projected to increase substantially, foretelling unsustainably large increases in the healthcare resources and costs needed to treat CVD patients in the coming decades. Effective venoactive drug treatments and ablation procedures are available that provide symptom relief, improve quality of life, slow disease progression, and promote ulcer healing. In addition, venoactive drug treatments may be highly cost-effective. However, there is evidence that physician awareness of CVD is suboptimal and that many patients with CVD are not being treated or referred to specialists according to established guidelines. To decrease this treatment gap and prevent unnecessary disease progression, international guidelines are available to help physicians consider CVD treatment options and refer patients when warranted. Improved disease awareness and appropriate early treatment may help reduce the coming burden of CVD.Funding: Servier.

PDB92 - Economic Impact of Hypoglycemic Events in Type 2 Diabetes Patients: A Review of Real-World Evidence

PDB92 - Economic Impact of Hypoglycemic Events in Type 2 Diabetes Patients: A Review of Real-World Evidence