Articles & Guides
Explore our in-depth articles and practical guides covering various aspects of pharmacoepidemiology methodologies, data sources, regulatory aspects, and more.
Choosing the Right Study Design in Pharmacoepidemiology Research
A comprehensive guide to selecting appropriate study designs for addressing specific research questions in drug safety and effectiveness research.
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Advanced Techniques for Controlling Confounding in Observational Studies
An exploration of modern approaches to addressing confounding bias in pharmacoepidemiological research, including propensity score methods, disease risk scores, and instrumental variables.
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Leveraging Administrative Claims Data for Pharmacoepidemiological Research
A practical guide to understanding, accessing, and analyzing administrative healthcare claims data for drug safety and utilization studies.
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Navigating Regulatory Requirements for Post-Marketing Safety Studies
An overview of current regulatory expectations for post-authorization safety studies in major markets, including study protocols, reporting requirements, and good pharmacovigilance practices.
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Real-World Evidence in Regulatory Decision Making: Current Status and Future Directions
An examination of how real-world evidence is being integrated into regulatory frameworks for both pre- and post-approval drug evaluation, with case studies and best practices.
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Integrating Electronic Health Records with Other Data Sources: Methods and Challenges
A technical guide to linking electronic health record data with other health information for comprehensive pharmacoepidemiological studies.
Read ArticleResearch Highlights
Stay informed about significant recent studies and publications in pharmacoepidemiology, with expert summaries and commentary on their implications.
Association of ACE Inhibitor and ARB Use with Adverse Outcomes in Hospitalized COVID-19 Patients: A Multinational Study
Key Findings:
This multinational cohort study of over 35,000 hospitalized COVID-19 patients found no significant association between prior ACE inhibitor or ARB use and increased risk of death or severe outcomes, after robust adjustment for confounding factors. The study employed multiple sensitivity analyses and advanced confounding control methods.
Methodological Contribution:
The researchers utilized a distributed network approach across 11 countries, with a common data model and analysis plan. This approach allowed for both country-specific and pooled analyses while addressing data privacy concerns.
Implications:
These findings provide reassurance regarding the continued use of these medications during the COVID-19 pandemic, demonstrating how pharmacoepidemiological research can rapidly address emerging clinical questions of public health importance.
Read Full SummaryValidity of Cancer Diagnoses in Administrative Claims Databases: A Systematic Review and Meta-Analysis
Key Findings:
This systematic review and meta-analysis evaluated the validity of cancer diagnoses in administrative claims data across 47 validation studies. Positive predictive values varied by cancer type (ranging from 65% to 93%) and were highest when algorithms incorporated multiple claims and specific diagnostic codes.
Methodological Contribution:
The authors developed a quality assessment framework for validation studies and provided cancer-specific recommendations for case-finding algorithms in claims data.
Implications:
This work provides essential guidance for researchers using claims data for cancer pharmacoepidemiology studies, highlighting the importance of validated outcome definitions in observational research.
Read Full SummaryComparative Cardiovascular Safety of GLP-1 Receptor Agonists in Type 2 Diabetes: A Population-Based Cohort Study
Key Findings:
In this large cohort study using U.S. claims data, significant differences in cardiovascular outcomes were observed between individual GLP-1 receptor agonists in routine clinical practice, with semaglutide associated with lower rates of major adverse cardiovascular events compared to other agents in the class.
Methodological Contribution:
The study employed an active comparator new-user design with high-dimensional propensity score adjustment to minimize confounding, demonstrating advanced methods for comparing treatments within the same therapeutic class.
Implications:
These findings provide real-world evidence to complement clinical trial data on the cardiovascular effects of GLP-1 receptor agonists, potentially informing treatment selection in clinical practice.
Read Full SummaryGlossary of Terms
A comprehensive glossary of key terms and concepts in pharmacoepidemiology and related fields, designed to support both newcomers and experienced professionals.
A
Active Comparator New-User Design
A study design that compares patients who are new users of one medication with patients who are new users of an alternative medication for the same indication. This approach aims to reduce confounding by indication and other biases associated with prevalent user designs.
Adverse Drug Reaction (ADR)
A response to a medicinal product that is noxious and unintended, occurring at doses normally used in humans for the prophylaxis, diagnosis, or therapy of disease, or for the restoration, correction, or modification of physiological function.
Administrative Claims Data
Healthcare data generated through the billing process for medical services, hospitalizations, procedures, and prescription drugs. These data are primarily collected for reimbursement purposes but are widely used in pharmacoepidemiological research.
B
Bias
A systematic error in the design, conduct, or analysis of a study that results in a mistaken estimate of an exposure's effect on the risk of disease. Common types in pharmacoepidemiology include selection bias, information bias, and confounding.
Biological Plausibility
The consistency of an observed association with existing biological and medical knowledge. In pharmacoepidemiology, this refers to whether an observed drug-outcome association is consistent with known pharmacological mechanisms.
C
Case-Crossover Design
A study design in which each case serves as its own control. Exposure frequency during a time period immediately before the outcome event (case period) is compared with exposure frequency during one or more control periods for the same individual.
Channeling Bias
A form of allocation bias that occurs when drugs with similar therapeutic indications are prescribed to groups of patients with different prognostic characteristics, based on factors such as disease severity, comorbidities, or perceived risk of adverse events.
Confounding by Indication
A type of confounding that occurs when the clinical indication for selecting a particular treatment also affects the outcome being studied, independently of the treatment effect. This is one of the most important sources of bias in observational studies of treatment effects.
External Resources
A curated collection of high-quality external resources including journals, databases, organizations, and tools relevant to pharmacoepidemiology research and practice.
Journals
Pharmacoepidemiology and Drug Safety
The official journal of the International Society for Pharmacoepidemiology, publishing original research, reviews, and methodological papers on the effects and uses of medicines in populations.
Visit WebsiteDrug Safety
A journal focusing on promoting the safe and proper use of medicines through the publication of high-quality research on pharmacovigilance and medication errors.
Visit WebsiteValue in Health
The official journal of ISPOR (International Society for Pharmacoeconomics and Outcomes Research), publishing research on pharmacoeconomics, health outcomes, and comparative effectiveness research.
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International Society for Pharmacoepidemiology (ISPE)
A global professional organization dedicated to advancing the health of the public by providing a forum for the exchange of scientific and educational information among members and by promoting pharmacoepidemiology.
Visit WebsiteCouncil for International Organizations of Medical Sciences (CIOMS)
An international non-governmental organization that publishes influential guidelines on pharmacovigilance and drug safety, including the classification and reporting of adverse drug reactions.
Visit WebsiteFDA Sentinel Initiative
A FDA-led program that uses electronic healthcare data to monitor the safety of FDA-regulated medical products, including drugs, biologics, and medical devices.
Visit WebsiteDatabases and Data Networks
OHDSI (Observational Health Data Sciences and Informatics)
A multi-stakeholder, interdisciplinary collaborative that brings out the value of health data through large-scale analytics using a common data model and standardized analytics tools.
Visit WebsiteCNODES (Canadian Network for Observational Drug Effect Studies)
A distributed network of Canadian researchers and data centers that uses administrative health care data to provide rapid answers to questions about drug safety and effectiveness.
Visit WebsiteEU-ADR (Exploring and Understanding Adverse Drug Reactions)
A European initiative that aims to design, develop, and validate a computerized system for detecting adverse drug reactions, using electronic healthcare records from several European countries.
Visit WebsiteRegulatory Resources
EMA Good Pharmacovigilance Practices
A set of measures drawn up by the European Medicines Agency to facilitate the performance of pharmacovigilance activities in the European Union.
Visit WebsiteFDA Guidance on Real-World Evidence
Guidance documents from the U.S. Food and Drug Administration on using real-world evidence to support regulatory decision-making for drugs and biologics.
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