Special Issue "Real World Data (RWD) and Real World Evidence (RWE) in Health Care Decisions"

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

Deadline for manuscript submissions: 20 August 2021.

Special Issue Editor

Dr. Valentina Orlando
E-Mail Website
Guest Editor
Department of Pharmacy, University of Naples Federico II
Interests: Big data analytics, Drug Utilization Research, Health Care Decision Making, Regulatory Decision-Making, Medication adherence

Special Issue Information

Dear Colleagues,

Interest in using real-world data (RWD) for providing real-world evidence (RWE) is significantly increasing in healthcare systems worldwide.

Nowadays, real-world evidence is widely considered a useful tool to confirm data collected for regulatory purposes.

In this growing scenario, searchable healthcare databases have been assessed as an excellent opportunity for secondary data used in research and applications. These databases include electronic health records and administrative claims, e.g., hospital discharge, prescribed drugs, and therapeutic procedures.

These data can be subsequently merged at an individual level using unique, anonymized identifiers and make data available for both academic and policy research.

These information are of great interest for making healthcare decisions that enable improvements in the quality, safety, and value of healthcare. Moreover, real-world evidence may provide information regarding the clinical practice of health technologies in real-world supporting decisions taken by stakeholders. This Special Issue is dedicated to the factors related to RWD and RWE as tools from the perspective of digital health in terms of a global vision.

Dr. Valentina Orlando
Guest Editor

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All papers will be peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. International Journal of Environmental Research and Public Health is an international peer-reviewed open access semimonthly journal published by MDPI.

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 data
  • Real-world evidence
  • Health
  • Health care decisions
  • Regulatory decision making
  • Health technology assessment
  • Drug utilization
  • Outcome research
  • Decision makers in health system
  • Data analysis in pharmaceutical therapy
  • Digital health

Published Papers (7 papers)

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Research

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Article
Real-World Data and Machine Learning to Predict Cardiac Amyloidosis
Int. J. Environ. Res. Public Health 2021, 18(3), 908; https://doi.org/10.3390/ijerph18030908 - 21 Jan 2021
Viewed by 1081
Abstract
(1) Background: Cardiac amyloidosis or “stiff heart syndrome” is a rare condition that occurs when amyloid deposits occupy the heart muscle. Many patients suffer from it and fail to receive a timely diagnosis mainly because the disease is a rare form of restrictive [...] Read more.
(1) Background: Cardiac amyloidosis or “stiff heart syndrome” is a rare condition that occurs when amyloid deposits occupy the heart muscle. Many patients suffer from it and fail to receive a timely diagnosis mainly because the disease is a rare form of restrictive cardiomyopathy that is difficult to diagnose, often associated with a poor prognosis. This research analyses the characteristics of this pathology and proposes a statistical learning algorithm that helps to detect the disease. (2) Methods: The hospitalization clinical (medical and nursing ones) records used for this study are the basis of the learning and training techniques of the algorithm. The approach consisted of using the information generated by the patients in each admission and discharge episode and treating it as data vectors to facilitate their aggregation. The large volume of clinical histories implied a high dimensionality of the data, and the lack of diagnosis led to a severe class imbalance caused by the low prevalence of the disease. (3) Results: Although there are few patients with amyloidosis in this study, the proposed approach demonstrates that it is possible to learn from clinical records despite the lack of data. In the validation phase, the algorithm first acted on data from the general study population. It then was applied to a sample of patients diagnosed with heart failure. The results revealed that the algorithm detects disease when data vectors profile each disease episode. (4) Conclusions: The prediction levels showed that this technique could be useful in screening processes on a specific population to detect the disease. Full article
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Article
Community Pharmacist’s Role in Detecting Low Back Pain, and Patient Attitudes—A Cross-Sectional Observational Study in Italian Community Pharmacies
Int. J. Environ. Res. Public Health 2020, 17(16), 5965; https://doi.org/10.3390/ijerph17165965 - 17 Aug 2020
Cited by 1 | Viewed by 841
Abstract
Background: Low back pain (LBP) is one of the most frequent diseases for which patients seek advice in a community pharmacy. The study aimed to evaluate the feasibility of the administration by community pharmacists of questionnaires to assess the LBP intensity and disability [...] Read more.
Background: Low back pain (LBP) is one of the most frequent diseases for which patients seek advice in a community pharmacy. The study aimed to evaluate the feasibility of the administration by community pharmacists of questionnaires to assess the LBP intensity and disability degree in patients entering community pharmacies and the attitudes they have toward pain management by pharmacological and non-pharmacological strategies. Methods: An explorative, cross-sectional, observational, and quantitative study was performed. Twelve Italian community pharmacists were asked to submit a questionnaire on LBP to patients visiting their pharmacies. The questionnaire included a pain intensity scale, and two validated tools: the Roland and Morris Disability Questionnaire (RMDQ) and the Start Back Screening Tool (SBST) to determine the degree and risk of patient disability, respectively. Results: 872 patients filled out the questionnaires in 6 months. No statistical differences between genders (p > 0.30) were recorded for pain intensity (Female: median score 6, IQR 4–7; Male: median scores 5, IQR 4–7; p > 0.30) and disability associated with LBP (RMDQ high-disability level: Females, 14.7%, Males, 15.0%; p > 0.90). Most of the patients (69%) reported a low degree of disability, but the risk of disability was medium and high in 36% and 18% of them, respectively (p < 0.05). About 14% of patients declare to never seek for physician’s advice despite their medium-high degree of disability. Conclusion: The study demonstrated the feasibility of validated tools for assessing the degree and risk of disability in LBP patients administrable in community pharmacies. Moreover, the community pharmacy resulted in an important care portal for patients suffering from moderate LBP and for intercepting patients who suffered from severe LBP but had never reported their problem to their physician. Full article
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Article
Gender Differences in Medication Use: A Drug Utilization Study Based on Real World Data
Int. J. Environ. Res. Public Health 2020, 17(11), 3926; https://doi.org/10.3390/ijerph17113926 - 01 Jun 2020
Cited by 1 | Viewed by 853
Abstract
A gender-specific drug utilization study was performed in the Campania region, Southern Italy. Data were based on outpatient drug prescriptions collected from administrative databases. The study population included all patients with at least one drug prescription in 2018. Prevalence was used as a [...] Read more.
A gender-specific drug utilization study was performed in the Campania region, Southern Italy. Data were based on outpatient drug prescriptions collected from administrative databases. The study population included all patients with at least one drug prescription in 2018. Prevalence was used as a measure to estimate the degree of exposure to drugs. A total of 3,899,360 patients were treated with at least one drug (54.2% females). The number of prescriptions was higher in females than males (55.6% vs. 44.4%). Females recorded higher prevalence for the majority of therapeutic groups (ATC II—anatomical therapeutic chemical), as well as for anti-inflammatory and antirheumatic products drugs (M01) (25.6% vs. 18.7%, risk ratio (RR): 0.73), beta blocking agents (C07) (14.5% vs. 11.6%, RR: 0.80), psychoanaleptics (N06) (7.1% vs. 3.7%, RR: 0.52), and antianemic preparations (B03) (2.8% vs. 6.7%, RR: 0.4). Higher prevalence was identified for males only for drugs used in diabetes (A10) (6.8% vs. 6.2%, RR: 1.1), particularly for biguanides (A10BA). Conversely, treatment duration was longer among males, explaining the higher mean cost per treated patient. This real-world study showed substantial gender differences in terms of medication use and duration of treatment and costs. These results are relevant to promoting and supporting the emerging role of precision and personalized medicine. Full article
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Article
Initial Therapy, Regimen Change, and Persistence in a Spanish Cohort of Newly Treated Type 2 Diabetes Patients: A Retrospective, Observational Study Using Real-World Data
Int. J. Environ. Res. Public Health 2020, 17(10), 3742; https://doi.org/10.3390/ijerph17103742 - 25 May 2020
Cited by 2 | Viewed by 1012
Abstract
The World Health Organization considers the non-adherence to medication a significant issue with global impact, especially in chronic conditions such as type 2 diabetes. We aim to study antidiabetic treatment initiation, add-on, treatment switching, and medication persistence. We conducted an observational study on [...] Read more.
The World Health Organization considers the non-adherence to medication a significant issue with global impact, especially in chronic conditions such as type 2 diabetes. We aim to study antidiabetic treatment initiation, add-on, treatment switching, and medication persistence. We conducted an observational study on 4247 individuals initiating antidiabetic treatment between 2013 and 2014 in the EpiChron Cohort (Spain). We used Cox regression models to estimate the likelihood of non-persistence after a one-year follow-up, expressed as hazard ratios (HRs). Metformin was the most frequently used first-line antidiabetic (80% of cases); combination treatment was the second most common treatment in adults aged 40–79 years, while dipeptidyl peptidase-4 inhibitors were the second most common in individuals in their 80s and over, and in patients with renal disease. Individuals initiated on metformin were less likely to present addition and switching events compared with any other antidiabetic. Almost 70% of individuals initiated on monotherapy were persistent. Subjects aged 40 and over (HR 0.53–0.63), living in rural (HR 0.79) or more deprived areas (HR 0.77–0.82), or receiving polypharmacy (HR 0.84), were less likely to show discontinuation. Our findings could help identify the population at risk of discontinuation, and offer them closer monitoring for proper integrated management to improve prognosis and health outcomes. Full article
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Article
Initiation and Single Dispensing in Cardiovascular and Insulin Medications: Prevalence and Explanatory Factors
Int. J. Environ. Res. Public Health 2020, 17(10), 3358; https://doi.org/10.3390/ijerph17103358 - 12 May 2020
Viewed by 787
Abstract
Background: Adherence problems have negative effects on health, but there is little information on the magnitude of non-initiation and single dispensing. Objective: The aim of this study was to estimate the prevalence of non-initiation and single dispensation and identify associated predictive factors for [...] Read more.
Background: Adherence problems have negative effects on health, but there is little information on the magnitude of non-initiation and single dispensing. Objective: The aim of this study was to estimate the prevalence of non-initiation and single dispensation and identify associated predictive factors for the main treatments prescribed in Primary Care (PC) for cardiovascular disease (CVD) and diabetes. Methods: Cohort study with real-world data. Patients who received a first prescription (2013–2014) for insulins, platelet aggregation inhibitors, angiotensin-converting enzyme inhibitors (ACEI) or statins in Catalan PC were included. The prevalence of non-initiation and single dispensation was calculated. Factors that explained these behaviours were explored. Results: At three months, between 5.7% (ACEI) and 9.1% (antiplatelets) of patients did not initiate their treatment and between 10.6% (statins) and 18.4% (ACEI) filled a single prescription. Body mass index, previous CVD, place of origin and having a substitute prescriber, among others, influenced the risk of non-initiation and single dispensation. Conclusions: The prevalence of non-initiation and single dispensation of CVD medications and insulin prescribed in PC in is high. Patient and health-system factors, such as place of origin and type of prescriber, should be taken into consideration when prescribing new medications for CVD and diabetes. Full article
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Article
Effectiveness of Maintenance Immunosuppression Therapies in a Matched-Pair Analysis Cohort of 16 Years of Renal Transplant in the Brazilian National Health System
Int. J. Environ. Res. Public Health 2020, 17(6), 1974; https://doi.org/10.3390/ijerph17061974 - 17 Mar 2020
Cited by 3 | Viewed by 970
Abstract
The maintenance of patients with renal transplant typically involves two or more drugs to prevent rejection and prolong graft survival. The calcineurin inhibitors (CNI) are the most commonly recommended medicines in combinations with others. While immunosuppressive treatment regimens are well established, there is [...] Read more.
The maintenance of patients with renal transplant typically involves two or more drugs to prevent rejection and prolong graft survival. The calcineurin inhibitors (CNI) are the most commonly recommended medicines in combinations with others. While immunosuppressive treatment regimens are well established, there is insufficient long-term effectiveness data to help guide future management decisions. The study analyzes the effectiveness of treatment regimens containing CNI after renal transplantation during 16 years of follow-up with real-world data from the Brazilian National Health System (SUS). This was a retrospective study of 2318 SUS patients after renal transplantion. Patients were propensity score-matched (1:1) by sex, age, type and year of transplantation. Kaplan–Meier analysis was used to estimate the cumulative probabilities of survival. A Cox proportional hazard model was used to evaluate factors associated with progression to graft loss. Multivariable analysis, adjusted for diabetes mellitus and race/color, showed a greater risk of graft loss for patients using tacrolimus plus mycophenolate compared to patients treated with cyclosporine plus azathioprine. In conclusion, this Brazilian real-world study, with a long follow-up period using matched analysis for relevant clinical features and the representativeness of the sample, demonstrated improved long-term effectiveness for therapeutic regimens containing cyclosporine plus azathioprine. Consequently, we recommend that protocols and clinical guidelines for renal transplantation should consider the cyclosporine plus azathioprine regimen as a potential first line option, along with others. Full article
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Review

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Review
Economic Evaluations Informed Exclusively by Real World Data: A Systematic Review
Int. J. Environ. Res. Public Health 2020, 17(4), 1171; https://doi.org/10.3390/ijerph17041171 - 12 Feb 2020
Cited by 3 | Viewed by 1181
Abstract
Economic evaluations using Real World Data (RWD) has been increasing in the very recent years, however, this source of information has several advantages and limitations. The aim of this review was to assess the quality of full economic evaluations (EE) developed using RWD. [...] Read more.
Economic evaluations using Real World Data (RWD) has been increasing in the very recent years, however, this source of information has several advantages and limitations. The aim of this review was to assess the quality of full economic evaluations (EE) developed using RWD. A systematic review was carried out through articles from the following databases: PubMed, Embase, Web of Science and Centre for Reviews and Dissemination. Included were studies that employed RWD for both costs and effectiveness. Methodological quality of the studies was assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist. Of the 14,011 studies identified, 93 were included. Roughly half of the studies were carried out in a hospital setting. The most frequently assessed illnesses were neoplasms while the most evaluated interventions were pharmacological. The main source of costs and effects of RWD were information systems. The most frequent clinical outcome was survival. Some 47% of studies met at least 80% of CHEERS criteria. Studies were conducted with samples of 100–1000 patients or more, were randomized, and those that reported bias controls were those that fulfilled most CHEERS criteria. In conclusion, fewer than half the studies met 80% of the CHEERS checklist criteria. Full article
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