Special Issue "Real-World Evidence in Clinical Epidemiology and Public Health"

A special issue of International Journal of Environmental Research and Public Health (ISSN 1660-4601). This special issue belongs to the section "Public Health Statistics and Risk Assessment".

Deadline for manuscript submissions: 31 December 2021.

Special Issue Editors

Dr. Matteo Franchi
E-Mail Website
Guest Editor
1. National Centre for Healthcare Research and Pharmacoepidemiology, Milan, Italy;
2. Laboratory of Healthcare Research & Pharmacoepidemiology, Unit of Biostatistics, Epidemiology and Public Health, Department of Statistics and Quantitative Methods, University of Milano-Bicocca, Milan, Italy
Interests: biostatistics; epidemiology; pharmacoepidemiology; healthcare research; real-world data; real-world evidence; cancer treatments; survival analysis; cost-effectiveness analysis; healthcare research
Dr. Anna Cantarutti
E-Mail Website
Guest Editor
1. National Centre for Healthcare Research and Pharmacoepidemiology, 20126 Milan, Italy;
2. Laboratory of Healthcare Research & Pharmacoepidemiology, Unit of Biostatistics, Epidemiology and Public Health, Department of Statistics and Quantitative Methods, University of Milano-Bicocca, 20126 Milan, Italy
Interests: biostatistics; epidemiology; pharmacoepidemiology; healthcare research; real-world data; real-world evidence; population-based birth cohort study; longitudinal studies; utilization, comparative safety and effectiveness of prescription medications in pregnant women and their offspring
Special Issues and Collections in MDPI journals
Dr. Federico Rea
E-Mail Website
Guest Editor
1. National Centre for Healthcare Research and Pharmacoepidemiology, 20126 Milan, Italy;
2. Laboratory of Healthcare Research & Pharmacoepidemiology, Unit of Biostatistics, Epidemiology and Public Health, Department of Statistics and Quantitative Methods, University of Milano-Bicocca, 20126 Milan, Italy
Interests: healthcare research; cost-effectiveness analysis; epidemiology; meta-analysis; multilevel modelling; pharmacoepidemiology; prognostic score; healthcare utilization database; case-identification algorithms; diagnostic-therapeutic paths

Special Issue Information

Dear Colleagues,

Although randomized clinical trials (RCTs) generate the highest level of evidence on the efficacy of a given treatment, they are insufficient for several reasons: (i) frail individuals, such as children, pregnant women, elderly people and those with comorbidities or co-treatments, are usually excluded from RCTs for both ethical and methodological reasons; (ii) the treatment efficacy is affected by a low rate of adherence and therapeutic inertia in clinical practice with the consequence that guidelines based on RCTs are often disregarded in real life; and (iii) RCTs are not suitable for assessing the efficacy of an integrated healthcare pathway.

Therefore, there is growing interest in observational studies based on real-world data (RWD), i.e., the data relating to patient health status in a non-experimental setting routinely collected from a variety of sources, that could complement the results of RCTs. RWD and real-world evidence (RWE) are playing an increasing role in healthcare decisions, and they are used to monitor post-marketing long-term cost-effectiveness and safety, and to develop guidelines and decision support tools for the use of treatments in clinical practice. According to the Food and Drug Administration, RWE is “the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of real-world data”. Thus, RWE should generate functional knowledge for the decision-making process, continuously improve the quality of care, and be used to assess the cost-effectiveness profiles of treatments observed in clinical practice.

This Special Issue is dedicated to studies that aim to generate RWE for the decision-making process.

Dr. Matteo Franchi
Dr. Anna Cantarutti
Dr. Federico Rea
Guest Editors

Manuscript Submission Information

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Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2300 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • real-world evidence
  • clinical epidemiology
  • public health
  • healthcare research
  • pharmacoepidemiology
  • frailty
  • chronic diseases

Published Papers (7 papers)

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Research

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Article
Drug–Drug Interactions in Italian Patients with Chronic Hepatitis C Treated with Pangenotypic Direct Acting Agents: Insights from a Real-World Study
Int. J. Environ. Res. Public Health 2021, 18(13), 7144; https://0-doi-org.brum.beds.ac.uk/10.3390/ijerph18137144 - 03 Jul 2021
Viewed by 519
Abstract
This Italian observational real-world study aims to assess in chronic hepatitis C virus (HCV) patients treated with pangenotypic direct acting agents (pDAAs) glecaprevir/pibrentasvir (GLE/PIB) or sofosbuvir/velpatasvir (SOF/VEL) the potential drug–drug interactions (DDIs) with concomitant medications prescribed, with a focus on cardiovascular and system [...] Read more.
This Italian observational real-world study aims to assess in chronic hepatitis C virus (HCV) patients treated with pangenotypic direct acting agents (pDAAs) glecaprevir/pibrentasvir (GLE/PIB) or sofosbuvir/velpatasvir (SOF/VEL) the potential drug–drug interactions (DDIs) with concomitant medications prescribed, with a focus on cardiovascular and system nervous (CNS) co-medications. Data were collected from administrative databases covering 6.9 million health-assisted individuals. All patients prescribed SOF/VEL or GLE/PIB between 11/2017 and 12/2018 were included. Patients were analyzed while on DAA. DDIs were identified according to the Liverpool University tool. Overall, 3181 HCV patients were included: 1619 in the GLE/PIB cohort and 1562 in the SOF/VEL cohort. SOF/VEL patients were generally older than GLE/PIB ones (mean age 58.4 vs. 53.1, p < 0.001) and had more cardiovascular and CNS comorbidities (58% vs. 42%, p < 0.001 and 33% vs. 28%, p = 0.002, respectively). Contraindications due to DDIs in the GLE/PIB cohort affected 9.3% and 3.2% of patients before and on DAA, respectively, while the percentages in the SOF/VEL cohort were 3.2% before and 0.4% after pDAAs initiation. Among GLE/PIB patients, 2.7% had cardiovascular drugs (all statins) contraindicated while on DAA. The potential DDIs between cardiovascular drugs and SOF/VEL were mainly with statins (5%). SOF/VEL was prescribed in patients with older age and with more cardiovascular and CNS comorbidities. Despite this, a proportion of contraindicated drugs lower than that of GLE/PIB was registered. Full article
(This article belongs to the Special Issue Real-World Evidence in Clinical Epidemiology and Public Health)
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Article
Treatment Patterns and Pharmacoutilization in Patients Affected by Rheumatoid Arthritis in Italian Settings
Int. J. Environ. Res. Public Health 2021, 18(11), 5679; https://0-doi-org.brum.beds.ac.uk/10.3390/ijerph18115679 - 26 May 2021
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Abstract
This study aimed to evaluate the treatment patterns and pharmacoutilization of patients with rheumatoid arthritis (RA) in real-world settings in Italy. This retrospective observational analysis was based on administrative databases of selected Italian entities. All adult patients with RA diagnosis confirmed by ≥1 [...] Read more.
This study aimed to evaluate the treatment patterns and pharmacoutilization of patients with rheumatoid arthritis (RA) in real-world settings in Italy. This retrospective observational analysis was based on administrative databases of selected Italian entities. All adult patients with RA diagnosis confirmed by ≥1 discharge diagnosis of RA (ICD-9-CM code = 714.0) or an active exemption code (006.714.0) were enrolled in 2019. Two cohorts were created: one included patients prescribed baricitinib, the other those prescribed biological disease-modifying antirheumatic drugs (bDMARDs). Overall, 47,711 RA patients were identified, most of them without DMARD prescription. As a first-line prescription, 43.2% of patients were prescribed conventional synthetic DMARDs (csDMARDs), 5.2% bDMARDs and 0.3% baricitinib. In 2019, 82.6% of csDMARD users continued with the same DMARD category, 15.9% had a bDMARD, while 1.5% had baricitinib as second-line therapy. Overall, 445 patients used baricitinib during 2019. During follow-up, baricitinib was prescribed as monotherapy to 31% of patients, as cotreatment with csDMARDs and corticosteroids to 27% of patients, with corticosteroids to 28% of patients and with csDMARDs to 14% of patients. In line with previous findings, a trend of bDMARD undertreatment was observed. The treatment patterns of baricitinib patients could help to better characterize patients eligible for new therapeutic options that will be available in the future. Full article
(This article belongs to the Special Issue Real-World Evidence in Clinical Epidemiology and Public Health)
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Article
Significantly Reduced Alanine Aminotransferase Level Increases All-Cause Mortality Rate in the Elderly after Ischemic Stroke
Int. J. Environ. Res. Public Health 2021, 18(9), 4915; https://0-doi-org.brum.beds.ac.uk/10.3390/ijerph18094915 - 05 May 2021
Viewed by 594
Abstract
(1) Background: A significantly reduced alanine aminotransferase (ALT) level is being recognized as a risk factor of increasing mortality in the elderly in relation to frailty. In the elderly, both frailty and ischemic stroke are not only common, but are also associated with [...] Read more.
(1) Background: A significantly reduced alanine aminotransferase (ALT) level is being recognized as a risk factor of increasing mortality in the elderly in relation to frailty. In the elderly, both frailty and ischemic stroke are not only common, but are also associated with mortality. The aim of this research was to investigate whether a significantly reduced ALT level increases the all-cause mortality rate in the elderly with ischemic stroke. (2) Methods: Between February 2014 and April 2019, a retrospective study of 901 patients with ischemic stroke admitted to a university-affiliated hospital was conducted. Cox proportional hazard regression was used to determine whether a significantly reduced ALT level is an independent risk factor for mortality in elderly patients after an ischemic stroke. (3) Results: This study enrolled 323 older adults (age ≥ 65 years) who were first diagnosed with ischemic stroke. The mean age of the participants was 76.5 ± 6.6 years, the mean survival time was 37.1 ± 20.4 months, and the number of deaths was 96 (29.7%). Our results showed that reduced ALT level (less than 10 U/L) increased the risk of all-cause mortality in the elderly after ischemic stroke (adjusted HR: 3.24, 95% CI: 1.95–5.41; p < 0.001). (4) Conclusions: A significantly reduced ALT level at the time of diagnosis (less than 10 U/L) is an independent risk factor that increases the mortality rate in the elderly after ischemic stroke. Full article
(This article belongs to the Special Issue Real-World Evidence in Clinical Epidemiology and Public Health)
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Article
Associated Factors and Survival Outcomes for Breast Conserving Surgery versus Mastectomy among New Zealand Women with Early-Stage Breast Cancer
Int. J. Environ. Res. Public Health 2021, 18(5), 2738; https://0-doi-org.brum.beds.ac.uk/10.3390/ijerph18052738 - 08 Mar 2021
Viewed by 653
Abstract
This study aimed to investigate type of loco-regional treatment received, associated treatment factors and mortality outcomes in New Zealand women with early-stage breast cancer who were eligible for breast conserving surgery (BCS). This is a retrospective analysis of prospectively collected data from the [...] Read more.
This study aimed to investigate type of loco-regional treatment received, associated treatment factors and mortality outcomes in New Zealand women with early-stage breast cancer who were eligible for breast conserving surgery (BCS). This is a retrospective analysis of prospectively collected data from the Auckland and Waikato Breast Cancer Registers and involves 6972 women who were diagnosed with early-stage primary breast cancer (I-IIIa) between 1 January 2000 and 31 July 2015, were eligible for BCS and had received one of four loco-regional treatments: breast conserving surgery (BCS), BCS followed by radiotherapy (BCS + RT), mastectomy (MTX) or MTX followed by radiotherapy (MTX + RT), as their primary cancer treatment. About 66.1% of women received BCS + RT, 8.4% received BCS only, 21.6% received MTX alone and 3.9% received MTX + RT. Logistic regression analysis was used to identify demographic and clinical factors associated with the receipt of the BCS + RT (standard treatment). Differences in the uptake of BCS + RT were present across patient demographic and clinical factors. BCS + RT was less likely amongst patients who were older (75+ years old), were of Asian ethnicity, resided in impoverished areas or areas within the Auckland region and were treated in a public healthcare facility. Additionally, BCS + RT was less likely among patients diagnosed symptomatically, diagnosed during 2000–2004, had an unknown tumour grade, negative/unknown oestrogen and progesterone receptor status or tumour sizes ≥ 20 mm, ≤50 mm and had nodal involvement. Competing risk regression analysis was undertaken to estimate the breast cancer-specific mortality associated with each of the four loco-regional treatments received. Over a median follow-up of 8.8 years, women who received MTX alone had a higher risk of breast cancer-specific mortality (adjusted hazard ratio: 1.38, 95% confidence interval (CI): 1.05–1.82) compared to women who received BCS + RT. MTX + RT and BCS alone did not have any statistically different risk of mortality when compared to BCS + RT. Further inquiry is needed as to any advantages BCS + RT may have over MTX alternatives. Full article
(This article belongs to the Special Issue Real-World Evidence in Clinical Epidemiology and Public Health)
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Article
Association between Adherence with Recommended Antenatal Care in Low-Risk, Uncomplicated Pregnancy, and Maternal and Neonatal Adverse Outcomes: Evidence from Italy
Int. J. Environ. Res. Public Health 2021, 18(1), 173; https://0-doi-org.brum.beds.ac.uk/10.3390/ijerph18010173 - 29 Dec 2020
Viewed by 703
Abstract
Antenatal care (ANC) aims of monitoring wellbeing of mother and foetus during pregnancy. We validate a set of indicators aimed of measuring the quality of ANC of women on low-risk, uncomplicated pregnancy through their relationship with maternal and neonatal outcomes. We conducted a [...] Read more.
Antenatal care (ANC) aims of monitoring wellbeing of mother and foetus during pregnancy. We validate a set of indicators aimed of measuring the quality of ANC of women on low-risk, uncomplicated pregnancy through their relationship with maternal and neonatal outcomes. We conducted a population-based cohort study including 122,563 deliveries that occurred between 2015 and 2017 in the Lombardy Region, Italy. Promptness and appropriateness of number and timing of gynaecological visits, ultrasounds and laboratory tests were evaluated. We assessed several maternal and neonatal outcomes. Log-binomial regression models were used to estimate prevalence ratio (PR), and corresponding 95% confidence interval (95% CI), for the exposure→outcome association. Compared with women who adhered with recommendations, those who were no adherent had a significant higher prevalence of maternal intensive care units admission (PR: 3.1, 95%CI: 1.2–7.9; and 2.7, 1.1–7.0 respectively for promptness of gynaecological visits, and appropriateness of ultrasound examinations), low Apgar score (1.6, 1.1–1.2; 1.9, 1.3–2.7; and 2.1, 1.5–2.8 respectively for appropriateness and promptness of gynaecological visits, and appropriateness of ultrasound examinations), and low birth weight (1.8, 1.5–2.3 for appropriateness of laboratory test examinations). Benefits for mothers and newborn are expected from improving adherence to guidelines-driven recommendations regarding antenatal care even for low-risk, uncomplicated pregnancies. Full article
(This article belongs to the Special Issue Real-World Evidence in Clinical Epidemiology and Public Health)
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Article
Real World Use of Antidiabetic Drugs in the Years 2011–2017: A Population-Based Study from Southern Italy
Int. J. Environ. Res. Public Health 2020, 17(24), 9514; https://0-doi-org.brum.beds.ac.uk/10.3390/ijerph17249514 - 18 Dec 2020
Cited by 1 | Viewed by 842
Abstract
Diabetes mellitus is a metabolic disease characterized by chronic hyperglycemia. The availability of new antidiabetic drugs (ADs) has led to complex treatment patterns and to changes in the patterns of specific drug utilization. The aim of this population-based study was to describe the [...] Read more.
Diabetes mellitus is a metabolic disease characterized by chronic hyperglycemia. The availability of new antidiabetic drugs (ADs) has led to complex treatment patterns and to changes in the patterns of specific drug utilization. The aim of this population-based study was to describe the pattern of antidiabetic drugs (ADs) use in Southern Italy in the years 2011–2017, in relation to the updated type 2 diabetes mellitus (T2DM) therapy guidelines. A retrospective cohort study was conducted on T2DM patients using data from the Palermo Local Health Unit (LHU) claims database and diabetologist registry. The first-line treatment was investigated and incident treatments were identified and characterized at baseline in terms of demographics, complications, comorbidities, concomitant drugs and clinical parameters. Persistence to AD treatment was also evaluated. During the study period, one-third of first ever ADs users started the treatment with ADs other than metformin, in contrast to guideline recommendations. Among 151,711 incident AD treatments, the male to female ratio was 1.0 and the median age was 66 (57–75) years. More than half (55.0%) of incident treatments discontinued the therapy during the first year of treatment. In Italy, general practitioners (GPs) can only prescribe first-generation ADs, while the prescription of more recently marketed ADs, such as GLP-1RA, DPP4i and SGLT2i, is restricted to diabetologists only, based on a therapeutic plan. The role of GPs in the management of T2DM in Italy should be re-evaluated. Full article
(This article belongs to the Special Issue Real-World Evidence in Clinical Epidemiology and Public Health)
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Review

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Review
The Risk of Gastrointestinal Bleeding between Non-Vitamin K Antagonist Oral Anticoagulants and Vitamin K Antagonists in the Asian Atrial Fibrillation Patients: A Meta-Analysis
Int. J. Environ. Res. Public Health 2021, 18(1), 137; https://0-doi-org.brum.beds.ac.uk/10.3390/ijerph18010137 - 27 Dec 2020
Viewed by 963
Abstract
Background: Non-vitamin K antagonist oral anticoagulants (NOACs) are more commonly used to prevent atrial fibrillation (AF) patients from thromboembolic events than vitamin K antagonists (VKAs). However, the gastrointestinal bleeding (GIB) risk in the Asian AF patients associated with NOACs in comparison with VKAs [...] Read more.
Background: Non-vitamin K antagonist oral anticoagulants (NOACs) are more commonly used to prevent atrial fibrillation (AF) patients from thromboembolic events than vitamin K antagonists (VKAs). However, the gastrointestinal bleeding (GIB) risk in the Asian AF patients associated with NOACs in comparison with VKAs remained unaddressed. Materials and Methods: A systematic search of studies on NOACs and VKAs in the Asian AF patients was conducted in PubMed, Cochrane Library, and ClinicalTrials.gov. The primary outcome was the hazard ratio (HR) of any GIB associated with NOACs versus VKAs. The secondary outcome was the GIB risks in different kinds of NOACs compared with VKAs. Results: This meta-analysis included two randomized controlled trials (RCTs) and four retrospective studies, comprising at least 200,000 patients in total. A significantly lower HR of GIB risks was found in all kinds of NOACs than VKAs in the Asian AF patients (HR: 0.633; 95% confidence interval: 0.535–0.748; p < 0.001). Additionally, the GIB risks of different NOACs were apixaban (HR: 0.392), edoxaban (HR: 0.603), dabigatran (HR: 0.685), and rivaroxaban (HR: 0.794), respectively. Conclusions: NOACs significantly reduced the risk of GIB in the Asian AF patients compared with VKAs. In the four NOACs compared with VKAs, apixaban probably had a trend of the least GIB risk. We need further head-to-head studies of different NOACs to confirm which NOAC is the most suitable for Asian AF patients and to know the optimal dosage regimen of different NOACs. Full article
(This article belongs to the Special Issue Real-World Evidence in Clinical Epidemiology and Public Health)
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