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Real-World Evidence to Assess Medication Safety or Effectiveness in Children: Systematic Review.

ABSTRACT: BACKGROUND:The promise of real-world evidence (RWE) is especially relevant to pediatrics, where medicines prescribed for children are often used without evidence derived from randomized clinical trials. OBJECTIVES:The aim of this systematic review was to describe the state of RWE in pediatrics by identifying observational studies published during 2016 that used RWE to assess medication safety or effectiveness in children. METHODS:An electronic search of PubMed was combined with an extended search of references within systematic reviews and expert suggestions. Studies were included if they reported on an infant or child under 18 years with exposure to medications; assessed safety or effectiveness; specified a comparison or control group, and were published in English in 2016. Data extraction was conducted by one team member using a standardized form and reviewed by a second team member. Study quality was assessed using the GRACE checklist for rating the quality of observational studies. RESULTS:After removing duplicates, 915 citations were screened and 29 studies met the eligibility criteria. Most of the eligible studies relied on primary data collection or chart review at a single institution and did not use the growing number of administrative or electronic health record databases available. One-quarter of the studies did not use well-established statistical methods to control for confounders. No single disease group or medication predominated, and age groups ranged from infants to adolescents. CONCLUSIONS:A small body of observational studies published in 2016 were categorized by the study team as using real-world data to assess medication safety or effectiveness in children. Studies varied in age groups, diseases or conditions, and methods, and may not have fully met the FDA definition of RWE. Our review indicates that the use of RWE is not fully developed in pediatrics, and suggests an opportunity to further develop capabilities and more fully leverage administrative and electronic health record databases to study medication safety and effectiveness in children. Our systematic review appears generalizable to pediatrics broadly, and documents that the high level of activity in RWE in general has had less of an impact on pediatrics.

SUBMITTER: Lasky T

PROVIDER: S-EPMC7221095 | biostudies-literature | 2020 Jun

REPOSITORIES: biostudies-literature

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Real-World Evidence to Assess Medication Safety or Effectiveness in Children: Systematic Review.

Lasky Tamar T Carleton Bruce B Horton Daniel B DB Kelly Lauren E LE Bennett Dimitri D Czaja Angela S AS Gifkins Dina D Osokogu Osemeke U OU McMahon Ann W AW

Drugs - real world outcomes 20200601 2

<h4>Background</h4>The promise of real-world evidence (RWE) is especially relevant to pediatrics, where medicines prescribed for children are often used without evidence derived from randomized clinical trials.<h4>Objectives</h4>The aim of this systematic review was to describe the state of RWE in pediatrics by identifying observational studies published during 2016 that used RWE to assess medication safety or effectiveness in children.<h4>Methods</h4>An electronic search of PubMed was combined ...[more]

PMID: 32112359

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Project description:BackgroundTo date, coronavirus disease 2019 (COVID-19) becomes increasingly fierce due to the emergence of variants. Rapid herd immunity through vaccination is needed to block the mutation and prevent the emergence of variants that can completely escape the immune surveillance. We aimed to systematically evaluate the effectiveness and safety of COVID-19 vaccines in the real world and to establish a reliable evidence-based basis for the actual protective effect of the COVID-19 vaccines, especially in the ensuing waves of infections dominated by variants.MethodsWe searched PubMed, Embase and Web of Science from inception to July 22, 2021. Observational studies that examined the effectiveness and safety of SARS-CoV-2 vaccines among people vaccinated were included. Random-effects or fixed-effects models were used to estimate the pooled vaccine effectiveness (VE) and incidence rate of adverse events after vaccination, and their 95% confidence intervals (CI).ResultsA total of 58 studies (32 studies for vaccine effectiveness and 26 studies for vaccine safety) were included. A single dose of vaccines was 41% (95% CI: 28-54%) effective at preventing SARS-CoV-2 infections, 52% (31-73%) for symptomatic COVID-19, 66% (50-81%) for hospitalization, 45% (42-49%) for Intensive Care Unit (ICU) admissions, and 53% (15-91%) for COVID-19-related death; and two doses were 85% (81-89%) effective at preventing SARS-CoV-2 infections, 97% (97-98%) for symptomatic COVID-19, 93% (89-96%) for hospitalization, 96% (93-98%) for ICU admissions, and 95% (92-98%) effective for COVID-19-related death, respectively. The pooled VE was 85% (80-91%) for the prevention of Alpha variant of SARS-CoV-2 infections, 75% (71-79%) for the Beta variant, 54% (35-74%) for the Gamma variant, and 74% (62-85%) for the Delta variant. The overall pooled incidence rate was 1.5% (1.4-1.6%) for adverse events, 0.4 (0.2-0.5) per 10 000 for severe adverse events, and 0.1 (0.1-0.2) per 10 000 for death after vaccination.ConclusionsSARS-CoV-2 vaccines have reassuring safety and could effectively reduce the death, severe cases, symptomatic cases, and infections resulting from SARS-CoV-2 across the world. In the context of global pandemic and the continuous emergence of SARS-CoV-2 variants, accelerating vaccination and improving vaccination coverage is still the most important and urgent matter, and it is also the final means to end the pandemic.

Project description:IntroductionBelimumab is a recombinant human monoclonal antibody that binds to soluble B-lymphocyte stimulator and inhibits its biological activity. Since receiving approvals for the treatment of systemic lupus erythematosus (SLE), several observational studies have investigated the effectiveness of belimumab in the real-world setting. This study reports a systematic review and meta-analysis of the literature to evaluate the real-world effectiveness of belimumab for the treatment of SLE.MethodsA literature search following PRISMA Guidelines and limited to studies in English was performed (2014-2020) to identify relevant studies reporting effectiveness outcomes of belimumab in patients with SLE. A modified version of the Newcastle-Ottawa Scale was used to assess study quality. Outcomes, including SLE Disease Activity Index (SLEDAI) score, prednisone-equivalent use, and SLE flare were pooled and analyzed using statistical aggregation methods.ResultsThe literature search identified 514 articles for initial review. Of these, 17 articles were suitable for data extraction and summary. Baseline characteristics of patients in real-world studies were generally similar to those of relevant clinical trials, including age, sex, disease duration, SLEDAI score, and prednisone-equivalent use. Real-world use of belimumab was associated with reductions in SLEDAI score (mean baseline score to month 6: 10.1-4.4; 57% reduction), prednisone-equivalent dosing (mean baseline dose to month 6: 12.1 mg/day to 6.9 mg/day; 43% reduction), and flare frequency (12 months prior to belimumab to 12 months after belimumab: 1.15-0.39 mean flares per patient per year; 66% reduction). Long-term data (up to 2 years post-treatment initiation) for SLEDAI score and prednisone-equivalent dose indicated that improvements in both outcomes continue over time among patients remaining on therapy.ConclusionsIn the real-world setting, observed outcomes with belimumab for the treatment of SLE are consistent with those reported from randomized clinical trials. Improvements persist long-term for SLEDAI activity and prednisone-equivalent use with belimumab.

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Project description:BACKGROUND:Childhood obesity is associated with a multitude of co-morbidities, including hypertension, hyperlipidaemia, cardiovascular disease and type 2 diabetes. Childhood obesity can also affect a young person's social, emotional and mental health if they encounter negative prejudice and social marginalisation. Given the prevalence of overweight and obese children globally, it is imperative that effective interventions are developed. Children are receptive to information conveyed via digital means, therefore, the use of technology may play a crucial role in interventions to reduce childhood obesity. This systematic review aimed to review and critically appraise the literature published to date in relation to the effectiveness of technology-based interventions, employed as secondary prevention, in addressing childhood obesity. METHODS:An electronic search strategy was undertaken in Medline and Embase, covering publications up to and including 12th July 2018. Randomised controlled trials assessing the effectiveness of technology-based interventions on weight-related outcomes in children, aged 8 to 18, published only in the English language, were included. RESULTS:From an initial search total of 1012 studies, 11 met the inclusion criteria. They were assessed for methodological quality using the Cochrane Risk of Bias Tool for Randomised Controlled Trials and were analysed using a narrative approach. The findings of this review showed a limited potential of technology-based interventions, employed as secondary prevention, to address childhood obesity. Of the eleven studies reviewed, three (27%) showed a positive relationship between technology-based interventions and weight-related outcomes in overweight or obese children. CONCLUSIONS:This review suggests that technology-based interventions, primarily active video games, as well as internet or web-based interventions and mobile phone communications, may, with further research, have the potential to impact positively on weight-related outcomes. It is difficult to determine the degree of efficacy of these technology-based interventions, as only two databases were searched, selecting only English language articles. Moreover, the included studies demonstrated a lack of high-quality evidence. The lack and heterogeneity of studies with technology-based interventions is a further limitation.

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Real-World Evidence to Assess Medication Safety or Effectiveness in Children: Systematic Review.

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Real-World Evidence to Assess Medication Safety or Effectiveness in Children: Systematic Review.

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