Review
Pharmacology & Pharmacy
Lesley H. Curtis, Oriol Sola-Morales, Julien Heidt, Patrick Saunders-Hastings, Laura Walsh, Deborah Casso, Susan Oliveria, Tiffany Mercado, Robbert Zusterzeel, Rachel E. Sobel, Jessica J. Jalbert, Vera Mastey, James Harnett, Ruben G. W. Quek
Summary: Regulators and HTA bodies are familiar with using external controls derived from RWD to generate RWE. This paper emphasizes the need for more guidance and alignment among regulatory agencies and HTA bodies in terms of operational and methodological aspects. Practical guidelines for the responsible generation of fit-for-purpose RWE are discussed, including early engagement with regulators, consideration of appropriate and comparable external controls, ensuring adequate sample sizes, addressing data quality issues, selecting meaningful endpoints, and conducting sensitivity analyses.
CLINICAL PHARMACOLOGY & THERAPEUTICS
(2023)
Article
Pharmacology & Pharmacy
Nirosha Mahendraratnam, Kerra Mercon, Mira Gill, Laura Benzing, Mark B. McClellan
Summary: Research has shown that RWE has been used to support the regulatory effectiveness of oncology and rare disease treatments, but there is potential for expanding its use to other therapeutic areas for a more patient-centered approach.
CLINICAL PHARMACOLOGY & THERAPEUTICS
(2022)
Article
Health Care Sciences & Services
Afsaneh Barzi, Angela J. Kim, Crystal K. Liang, Howard West, D. Wong, Carol Wright, Nitya Nathwani, Catherine M. Vasko, Vincent Chung, Douglas A. Rubinson, Todd Sachs
Summary: This study identified significant gaps in care delivery for patients with pancreatic adenocarcinoma, particularly in patients with non-metastatic cancer. Deficiencies in germline testing and palliative care were major concerns. Measures should be taken to address these issues in a timely manner.
JOURNAL OF PERSONALIZED MEDICINE
(2023)
Article
Multidisciplinary Sciences
Rasmus Ingemann Tuffveson Jensen, Joras Ferwerda, Kristian Sand Jorgensen, Erik Rathje Jensen, Martin Borg, Morten Persson Krogh, Jonas Brunholm Jensen, Alexandros Iosifidis
Summary: Due to the confidentiality of bank transactions, there is a lack of public data sets for investigating and comparing anti-money laundering methods in banks, which severely limits research in this area. To address this issue, we propose SynthAML, a synthetic data set for benchmarking statistical and machine learning methods for AML, and analyze open problems in the AML literature.
Article
Computer Science, Software Engineering
Linghui Luo, Felix Pauck, Goran Piskachev, Manuel Benz, Ivan Pashchenko, Martin Mory, Eric Bodden, Ben Hermann, Fabio Massacci
Summary: Due to the lack of established real-world benchmark suites for static taint analyses of Android applications, evaluations of these analyses are often restricted and hard to compare. To address this issue, this paper proposes criteria for constructing real-world benchmark suites and introduces TaintBench, the first benchmark suite with documented taint flows for real-world malware. Experiments using TaintBench provide new insights into the effectiveness and accuracy of taint analysis tools.
EMPIRICAL SOFTWARE ENGINEERING
(2022)
Review
Rheumatology
Suchitra Kataria, Vinod Ravindran
Summary: The diversity of diseases in rheumatology and variability in disease prevalence require greater data parity in disease presentation and treatment responses. Real-world data from various resources can provide insights into disease evolution and progression, as well as the early diagnosis of diseases. Digital tools have the potential to generate realistic, non-invasive, patient-friendly real-world evidence in rheumatology.
Review
Biochemical Research Methods
Stefano Castellana, Tommaso Biagini, Luca Parca, Francesco Petrizzelli, Salvatore Daniele Bianco, Angelo Luigi Vescovi, Massimo Carella, Tommaso Mazza
Summary: This study found that hundreds of human proteins interact with degenerated DNA sequences, and identifying these motifs and genomic sites is a challenging research goal in modern molecular biology and bioinformatics. Over the past twenty years, there has been an explosion of computational tools for this task, and sixteen of them were evaluated for their ability to identify known motifs in simulated sequence datasets.
BRIEFINGS IN BIOINFORMATICS
(2021)
Article
Pharmacology & Pharmacy
Rui Zhu, Bianca Vora, Sujatha Menon, Islam Younis, Gaurav Dwivedi, Zhaoling Meng, Amita Datta-Mannan, Pooja Manchandani, Satyaprakash K. Nayak, Brinda Tammara, Parag Garhyan, Shahed Iqbal, Simon Dagenais, Pascal Chanu, Arnab Mukherjee, Cyrus Ghobadi
Summary: Since the enactment of the 21st Century Cures Act in 2016, real-world data (RWD) and real-world evidence (RWE) have gained significant attention in the global healthcare sector. While the potential of RWD/RWE to inform regulatory decisions and drug development has been extensively discussed, a comprehensive review of industry applications from a clinical pharmacology perspective is needed to uncover new insights and identify future opportunities.
CLINICAL PHARMACOLOGY & THERAPEUTICS
(2023)
Article
Infectious Diseases
David Shasha, Ronen Bareket, Fabienne Hershkowitz Sikron, Omer Gertel, Judith Tsamir, David Dvir, David Mossinson, Anthony D. Heymann, Galia Zacay
Summary: In this study, individuals vaccinated with the Pfizer BNT162b2 vaccine were compared with unvaccinated controls, showing no association between vaccination and Bell's palsy, herpes zoster, or GBS. However, vaccinated individuals were more likely to experience symptoms of numbness or tingling. The study provides reassuring data regarding the safety of the BNT162b2 vaccine.
CLINICAL MICROBIOLOGY AND INFECTION
(2022)
Article
Multidisciplinary Sciences
Balint Hartmann, Viktoria Sugar
Summary: The study found that only after the introduction of multiple voltage levels, power grids exhibit small-world behavior; the node distribution of the grid does not show scale-free behavior, and the scaling stabilizes around certain values after the initial phase of grid evolution.
SCIENTIFIC REPORTS
(2021)
Article
Computer Science, Information Systems
Clair Blacketer, Frank J. Defalco, Patrick B. Ryan, Peter R. Rijnbeek
Summary: Advances in standardization of observational healthcare data have enabled methodological breakthroughs, rapid global collaboration, and generation of real-world evidence to improve patient outcomes. Standardization in data structure needs to be coupled with standardized approaches for data quality assessment to ensure confidence in real-world evidence. The implementation of data quality checks and reporting can customize data quality reporting across different observational health data sources to promote transparency and build confidence in the resulting output.
JOURNAL OF THE AMERICAN MEDICAL INFORMATICS ASSOCIATION
(2021)
Article
Gastroenterology & Hepatology
Andrea R. Mospan, Heather L. Morris, Michael W. Fried
Summary: Real-world evidence includes health-related information obtained outside of clinical trials, which can provide insights into disease progression and treatment effectiveness. Global regulatory agencies have utilized real-world data to inform drug development and refine therapeutic regimens post-marketing, especially in underrepresented populations. In the management of hepatocellular carcinoma, early detection offers the best chance for curative therapies, while novel systemic treatments have improved survival rates. Longitudinal observational studies in real-world settings can help assess the long-term safety and effectiveness of these therapies.
LIVER INTERNATIONAL
(2021)
Article
Hematology
William A. Wood, Peter Marks, Robert M. Plovnick, Kathleen Hewitt, Donna S. Neuberg, Sam Walters, Brendan K. Dolan, Emily A. Tucker, Charles S. Abrams, Alexis A. Thompson, Kenneth C. Anderson, Paul Kluetz, Ann Farrell, Donna Rivera, Matthew Gertzog, Gregory Pappas
Summary: The ASH Research Collaborative is a nonprofit organization that aims to support patients with hematologic conditions. It houses two major initiatives: the Data Hub for collecting patient data and the Clinical Trials Network for accelerating clinical trials efficiency, initially developed for sickle cell disease. The organization focuses on enhancing clinical care through data analysis and testing healthcare delivery strategies.
Article
Computer Science, Artificial Intelligence
Zhihang Zhong, Ye Gao, Yinqiang Zheng, Bo Zheng, Imari Sato
Summary: Real-time video deblurring in real world is a challenging task. This paper proposes an improved method that incorporates residual dense blocks into RNN cells and introduces a global spatio-temporal attention module. The authors also contribute a novel benchmark dataset to the community. Experimental results demonstrate the superior deblurring performance of the proposed method.
INTERNATIONAL JOURNAL OF COMPUTER VISION
(2023)
Review
Orthopedics
David J. Hak, John I. Mackowiak, Debra E. Irwin, Molly L. Aldridge, Christina D. Mack
Summary: Real-world data (RWD) are increasingly important in orthopaedics, with each type of RWD source having its strengths and weaknesses. Linking different RWD datasets provides greater utility and value for research. Careful consideration of data quality and appropriate analysis methods can alleviate concerns about the validity of orthopaedic studies using RWD.
JOURNAL OF ORTHOPAEDIC TRAUMA
(2021)
Editorial Material
Public, Environmental & Occupational Health
Cynthia J. Girman, Mary E. Ritchey, Wei Zhou, Nancy A. Dreyer
PHARMACOEPIDEMIOLOGY AND DRUG SAFETY
(2019)
Review
Public, Environmental & Occupational Health
Elodie Baumfeld Andre, Robert Reynolds, Patrick Caubel, Laurent Azoulay, Nancy A. Dreyer
PHARMACOEPIDEMIOLOGY AND DRUG SAFETY
(2020)
Article
Medical Informatics
Jennifer B. Christian, Emily S. Brouwer, Cynthia J. Girman, Dimitri Bennett, Kourtney J. Davis, Nancy A. Dreyer
THERAPEUTIC INNOVATION & REGULATORY SCIENCE
(2020)
Review
Medical Informatics
Nancy A. Dreyer, Marni Hall, Jennifer B. Christian
THERAPEUTIC INNOVATION & REGULATORY SCIENCE
(2020)
Article
Health Care Sciences & Services
Kristin M. Sheffield, Nancy A. Dreyer, James F. Murray, Douglas E. Faries, Megan N. Klopchin
JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH
(2020)
Article
Economics
Dony Patel, Fiona Grimson, Elena Mihaylova, Peter Wagner, Joss Warren, Anke van Engen, Joseph Kim
Summary: The study found that the number of SAT-based HTA submissions increased 13-fold between 2011 and 2019, with around 65% focused on oncology or hem-oncology. Of the 226 submissions, approximately 52% used some form of EC data, with an overall acceptance rate of 48% and 59% when including RWD EC data. The use of RWD ECs increased over time while prior trial ECs decreased, impacting acceptance rates.
Article
Medicine, General & Internal
Nancy Dreyer, Natalia Petruski-Ivleva, Lisa Albert, Damir Mohamed, Emma Brinkley, Matthew Reynolds, Stephen Toovey
Summary: Individuals with underlying autoimmune conditions who use medications for these conditions are more likely to experience persistent COVID-19 symptoms, including shortness of breath and fatigue, after 30 days compared to those not using such medications. This suggests that early intervention may be necessary for this vulnerable population.
INTERNATIONAL JOURNAL OF GENERAL MEDICINE
(2021)
Editorial Material
Engineering, Biomedical
Jennifer Price, Noah Goodson, Edward J. Warren, Paul Wicks, John Reites
EXPERT REVIEW OF MEDICAL DEVICES
(2021)
Article
Health Care Sciences & Services
Matthew W. Reynolds, Alex Secora, Alice Joules, Lisa Albert, Emma Brinkley, Tom Kwon, Christina Mack, Stephen Toovey, Nancy A. Dreyer
Summary: This study assessed the real-world effectiveness of three COVID-19 vaccines and found all three to be highly effective.
JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH
(2022)
Article
Infectious Diseases
Matthew W. Reynolds, Yiqiong Xie, Kendall B. Knuth, Christina D. Mack, Emma Brinkley, Stephen Toovey, Nancy A. Dreyer
Summary: This study used adult community volunteers to obtain systematic information on vaccine effectiveness and breakthrough infections, showing a lower breakthrough infection rate in fully vaccinated individuals, with most cases being asymptomatic or having mild symptoms compared to unvaccinated cases.
INFECTION AND DRUG RESISTANCE
(2022)
Article
Medicine, General & Internal
Nancy A. Dreyer, Christina Mack
Summary: Real-world evidence (RWE) is derived from real-world data (RWD) generated by clinicians and patients in everyday care settings. RWE is used to evaluate the real-world performance of medical interventions, services, and diagnostics, with a focus on diverse patient groups that may not be typically studied in traditional randomized clinical trials (RCT). This article provides guidance on study design and execution for clinicians interested in using RWE, including considerations about study participant representativeness, data transparency, and potential sources of bias. The authors also highlight the growing role of RWE in clinical, regulatory, and payer decision-making.
PRAGMATIC AND OBSERVATIONAL RESEARCH
(2023)
Article
Public, Environmental & Occupational Health
Nancy A. Dreyer, Matthew Reynolds, Christina DeFilippo Mack, Emma Brinkley, Natalia Petruski-Ivleva, Kalyani Hawaldar, Stephen Toovey, Jonathan Morris
TRAVEL MEDICINE AND INFECTIOUS DISEASE
(2020)
Article
Medical Informatics
Raymond A. Hum, Jill Dawson, Karen Lipworth, Luis Rojas, Edward J. Warren, Charu Manaktala, Jonathan R. Huml
THERAPEUTIC INNOVATION & REGULATORY SCIENCE
(2020)
