Use of Real‐World Evidence to Drive Drug Development Strategy and Inform Clinical Trial Design

Diagnostic Delay in Patients with Eosinophilic Gastritis and/or Duodenitis: A Population-Based Study

(2021) Mirna Chehade et al.   Journal of Allergy and Clinical Immunology-In Practice

Cancer Screening Tests and Cancer Diagnoses During the COVID-19 Pandemic

(2021) Ziad Bakouny et al.   JAMA Oncology

Association of SARS-CoV-2 Seropositive Antibody Test With Risk of Future Infection

(2021) Raymond A. Harvey et al.   JAMA Internal Medicine

Drugs in COVID‐19 Clinical Trials: Predicting Transporter‐Mediated Drug‐Drug Interactions Using In Vitro Assays and Real‐World Data

(2021) Sook Wah Yee et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Artificial intelligence in drug discovery: what is realistic, what are illusions? Part 2: a discussion of chemical and biological data

(2021) Andreas Bender et al.   DRUG DISCOVERY TODAY

Artificial intelligence to deep learning: machine intelligence approach for drug discovery

(2021) Rohan Gupta et al.   MOLECULAR DIVERSITY

Evaluating eligibility criteria of oncology trials using real-world data and AI

(2021) Ruishan Liu et al.   NATURE

COVID-19 Evidence Accelerator: A parallel analysis to describe the use of Hydroxychloroquine with or without Azithromycin among hospitalized COVID-19 patients

(2021) Mark Stewart et al.   PLoS One

Real-world evidence and product development: Opportunities, challenges and risk mitigation

(2021) Poobalan Naidoo et al.   WIENER KLINISCHE WOCHENSCHRIFT

Quantitative Image Analysis for Tissue Biomarker Use

(2021) Haydee Lara et al.   APPLIED IMMUNOHISTOCHEMISTRY & MOLECULAR MORPHOLOGY

Demographic diversity of participants in Pfizer sponsored clinical trials in the United States

(2021) Melinda Rottas et al.   Contemporary Clinical Trials

The Contribution of Machine Learning in the Validation of Commercial Wearable Sensors for Gait Monitoring in Patients: A Systematic Review

(2021) Théo Jourdan et al.   SENSORS

TrendyGenes, a computational pipeline for the detection of literature trends in academia and drug discovery

(2021) Guillermo Serrano Nájera et al.   Scientific Reports

A DICOM dataset for evaluation of medical image de-identification

(2021) Michael Rutherford et al.   Scientific Data

Clinical Relevance of a 16-Gene Pharmacogenetic Panel Test for Medication Management in a Cohort of 135 Patients

(2021) David F. Niedrig et al.   Journal of Clinical Medicine

Organoid Technology: A Reliable Developmental Biology Tool for Organ-Specific Nanotoxicity Evaluation

(2021) Minakshi Prasad et al.   Frontiers in Cell and Developmental Biology

Artificial intelligence for pre-operative lymph node staging in colorectal cancer: a systematic review and meta-analysis

(2021) Sergei Bedrikovetski et al.   BMC CANCER

Preclinical tumor organoid models in personalized cancer therapy: Not everyone fits the mold

(2021) Li-Feng Hu et al.   EXPERIMENTAL CELL RESEARCH

Combination ATR and PARP Inhibitor (CAPRI): A phase 2 study of ceralasertib plus olaparib in patients with recurrent, platinum-resistant epithelial ovarian cancer

(2021) Payal D. Shah et al.   GYNECOLOGIC ONCOLOGY

Estimated Research and Development Investment Needed to Bring a New Medicine to Market, 2009-2018

(2020) Olivier J. Wouters et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Opportunities and challenges in using real-world data for health care

(2020) Vivek A. Rudrapatna et al.   JOURNAL OF CLINICAL INVESTIGATION

How Can Law and Policy Advance Quality in Genomic Analysis and Interpretation for Clinical Care?

(2020) Barbara J. Evans et al.   JOURNAL OF LAW MEDICINE & ETHICS

Low hemoglobin increases risk for cerebrovascular disease, kidney disease, pulmonary vasculopathy, and mortality in sickle cell disease: A systematic literature review and meta-analysis

(2020) Kenneth I. Ataga et al.   PLoS One

Use of real‐world data to emulate a clinical trial and support regulatory decision‐making: assessing the impact of temporality, comparator choice and methods of adjustment

(2020) Devin Abrahami et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Dissecting the journey to breast cancer diagnosis in sub‐Saharan Africa: Findings from the multicountry ABC‐DO cohort study

(2020) Milena Foerster et al.   INTERNATIONAL JOURNAL OF CANCER

Immunostimulation with chemotherapy in the era of immune checkpoint inhibitors

(2020) Lorenzo Galluzzi et al.   Nature Reviews Clinical Oncology

Responsible, practical genomic data sharing that accelerates research

(2020) James Brian Byrd et al.   NATURE REVIEWS GENETICS

Combining PARP with ATR inhibition overcomes PARP inhibitor and platinum resistance in ovarian cancer models

(2020) Hyoung Kim et al.   Nature Communications

A review of computational drug repositioning: strategies, approaches, opportunities, challenges, and directions

(2020) Tamer N. Jarada et al.   Journal of Cheminformatics

Bridging the Gap Between RCTs and RWE Through Endpoint Selection

(2020) Robert J. LoCasale et al.   Therapeutic Innovation & Regulatory Science

Evolving use of real-world evidence in the regulatory process: a focus on immuno-oncology treatment and outcomes

(2020) John C O’Donnell et al.   Future Oncology

Comparative host-coronavirus protein interaction networks reveal pan-viral disease mechanisms

(2020) David E. Gordon et al.   SCIENCE

Improving Transparency to Build Trust in Real-World Secondary Data Studies for Hypothesis Testing—Why, What, and How: Recommendations and a Road Map from the Real-World Evidence Transparency Initiative

(2020) Lucinda S. Orsini et al.   VALUE IN HEALTH

Diagnosis-wide analysis of COVID-19 complications: an exposure-crossover study

(2020) William Murk et al.   CANADIAN MEDICAL ASSOCIATION JOURNAL

Call to Action: Structural Racism as a Fundamental Driver of Health Disparities: A Presidential Advisory From the American Heart Association

(2020) Keith Churchwell et al.   CIRCULATION

Disease course and treatment patterns in progressive supranuclear palsy: A real-world study

(2020) John C. Morgan et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

The prevalence of MS in the United States

(2019) Mitchell T. Wallin et al.   NEUROLOGY

A new way to estimate neurologic disease prevalence in the United States

(2019) Lorene M. Nelson et al.   NEUROLOGY

Current Treatment Patterns of Aplastic Anemia in China: A Prospective Cohort Registry Study

(2019) Xiao-Fan Zhu et al.   ACTA HAEMATOLOGICA

Re‐identifiability of genomic data and the GDPR

(2019) Mahsa Shabani et al.   EMBO REPORTS

Applications of machine learning in drug discovery and development

(2019) Jessica Vamathevan et al.   NATURE REVIEWS DRUG DISCOVERY

Nonrandomized real‐world evidence to support regulatory decision‐making: Process for a randomized trial replication project

(2019) Jessica M. Franklin et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Overcoming Regulatory Aversion to Novel Methods of Evidence Generation

(2019) Elodie Baumfeld Andre et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

The Signaling Pathways Project, an integrated ‘omics knowledgebase for mammalian cellular signaling pathways

(2019) Scott A. Ochsner et al.   Scientific Data

Beyond the Randomized Clinical Trial: Innovative Data Science to Close the Pediatric Evidence Gap

(2019) Sebastiaan C. Goulooze et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Trial designs using real‐world data: The changing landscape of the regulatory approval process

(2019) Elodie Baumfeld Andre et al.   PHARMACOEPIDEMIOLOGY AND DRUG SAFETY

Mechanisms of antimicrobial resistance among hospital-associated pathogens

(2018) Ayesha Khan et al.   Expert Review of Anti-Infective Therapy

Are whole-exome and whole-genome sequencing approaches cost-effective? A systematic review of the literature

(2018) Katharina Schwarze et al.   GENETICS IN MEDICINE

Treatment patterns, adherence, and persistence among psoriasis patients treated with biologics in a real-world setting, overall and by disease severity

(2018) Mwangi J. Murage et al.   JOURNAL OF DERMATOLOGICAL TREATMENT

Progress and potential in organoid research

(2018) Giuliana Rossi et al.   NATURE REVIEWS GENETICS

The inclusion of real world evidence in clinical development planning

(2018) Reynaldo Martina et al.   Trials

Incidence and prevalence of neuroendocrine tumors of the lung: analysis of a US commercial insurance claims database

(2018) Michael S. Broder et al.   BMC Pulmonary Medicine

Predictive biomarkers of response for immune checkpoint inhibitors in non–small-cell lung cancer

(2018) Arsela Prelaj et al.   EUROPEAN JOURNAL OF CANCER

Using electronic health records to estimate the prevalence of agitation in Alzheimer disease/dementia

(2018) Rachel Halpern et al.   INTERNATIONAL JOURNAL OF GERIATRIC PSYCHIATRY

Measuring prevalence and incidence of chronic conditions in claims and electronic health record databases

(2018) Jeremy A Rassen et al.   Clinical Epidemiology

Reporting to Improve Reproducibility and Facilitate Validity Assessment for Healthcare Database Studies V1.0

(2017) Shirley V. Wang et al.   PHARMACOEPIDEMIOLOGY AND DRUG SAFETY

Good practices for real-world data studies of treatment and/or comparative effectiveness: Recommendations from the joint ISPOR-ISPE Special Task Force on real-world evidence in health care decision making

(2017) Marc L. Berger et al.   PHARMACOEPIDEMIOLOGY AND DRUG SAFETY

Beyond imaging: The promise of radiomics

(2017) Michele Avanzo et al.   Physica Medica-European Journal of Medical Physics

Good Practices for Real‐World Data Studies of Treatment and/or Comparative Effectiveness: Recommendations from the Joint ISPOR‐ISPE Special Task Force on Real‐World Evidence in Health Care Decision Making

(2017) Marc L. Berger et al.   VALUE IN HEALTH

Pragmatic randomized clinical trials: a proposal to enhance evaluation of new cancer therapies with early signs of exceptional activity

(2016) M. Koehler et al.   ANNALS OF ONCOLOGY

Natural history of diseases: Statistical designs and issues

(2016) Nicholas P. Jewell   CLINICAL PHARMACOLOGY & THERAPEUTICS

Innovation in the pharmaceutical industry: New estimates of R&D costs

(2016) Joseph A. DiMasi et al.   JOURNAL OF HEALTH ECONOMICS

Defining SOD1 ALS natural history to guide therapeutic clinical trial design

(2016) Taha Bali et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Changing R&D models in research-based pharmaceutical companies

(2016) Alexander Schuhmacher et al.   Journal of Translational Medicine

De-identification of Medical Images with Retention of Scientific Research Value

(2015) Stephen M. Moore et al.   RADIOGRAPHICS

Prevalence of Prostate Cancer Clinical States and Mortality in the United States: Estimates Using a Dynamic Progression Model

(2015) Howard I. Scher et al.   PLoS One

Prevalence of Eosinophilic Esophagitis in the United States

(2013) Evan S. Dellon et al.   Clinical Gastroenterology and Hepatology

A Proposal for Integrated Efficacy-to-Effectiveness (E2E) Clinical Trials

(2013) H P Selker et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Potential endpoints for clinical trials in premanifest and early Huntington's disease in the TRACK-HD study: analysis of 24 month observational data

(2011) Sarah J Tabrizi et al.   LANCET NEUROLOGY

How to improve R&D productivity: the pharmaceutical industry's grand challenge

(2010) Steven M. Paul et al.   NATURE REVIEWS DRUG DISCOVERY