ReviewClinical pharmacology and therapeutics2022
When Can Nonrandomized Studies Support Valid Inference Regarding Effectiveness or Safety of New Medical Treatments?
Review in Clinical pharmacology and therapeutics, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
19 citing papers in PubMed.
- Neuropsychiatric outcomes after montelukast versus inhaled corticosteroids in pediatric asthma: a pooled birth cohort study.BMC medicine · 2026Article
- Evaluating the Return on Investment of U.S. Army Holistic Health and Fitness Performance Teams: A Matched Difference-in-Differences Study of Readiness and Economic Outcomes.Sports medicine (Auckland, N.Z.) · 2026Article
- GLP-1 Receptor Agonists in Metabolic Dysfunction-Associated Steatotic Liver Disease: Bridging Hepatic and Cardiovascular Outcomes.Chronic diseases and translational medicine · 2026Review
- Emulating Six Cardiovascular Outcome Trials of Antidiabetic Drugs in Individuals with Type 2 Diabetes: Analyses Based on Real-World Databases from Korea and Taiwan.Clinical pharmacology and therapeutics · 2026Article
- Application of the Target Trial Emulation Framework to Studies in the Pediatric Population.The Journal of pediatrics · 2026Article
- Pharmacoepidemiology unpacked: a roadmap for junior researchers.Frontiers in pharmacology · 2026Review
- General Practitioner Coordinated Multidisciplinary Care Improves Long-Term Survival following Stroke with Variation by Impairment.Neuroepidemiology · 2026Observational
- Methodological Concerns Regarding the Partially Randomized Design and Statistical Inference in Preoperative TEAS for Breast Cancer Patients: A Letter to the Editor [Letter].Journal of pain research · 2026Article
- Partnering with Family Organizations in Research: Experiences from the Field.International journal of environmental research and public health · 2025Article
- Analyzing missingness patterns in real-world data using the SMDI toolkit: application to a linked EHR-claims pharmacoepidemiology study.BMC medical research methodology · 2024Article
- Article
- Treating COVID-19: Targeting the Host Response, Not the Virus.Life (Basel, Switzerland) · 2023Review
- Roles of real-world evidence in severe asthma treatment: challenges and opportunities.ERJ open research · 2023Article
- Inclusion of nonrandomized studies of interventions in systematic reviews of interventions: updated guidance from the Agency for Health Care Research and Quality Effective Health Care program.Journal of clinical epidemiology · 2022Article
- Article
- An Overview of Phase 2 Clinical Trial Designs.International journal of radiation oncology, biology, physics · 2022Review
- Remodeling an existing rare disease registry to be used in regulatory context: Lessons learned and recommendations.Frontiers in pharmacology · 2022Article
- Clinical Evidence Generation During a Pandemic: Lessons Learned for Sustaining Progress.Cancer journal (Sudbury, Mass.)Review
- Generating fit-for-purpose real-world evidence in Asia: How far are we from closing the gaps?Perspectives in clinical researchReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors.
Funding
Abstract
The randomized controlled trial (RCT) is the gold standard for evaluating the causal effects of medications. Limitations of RCTs have led to increasing interest in using real-world evidence (RWE) to augment RCT evidence and inform decision making on medications. Although RWE can be either randomized or nonrandomized, nonrandomized RWE can capitalize on the recent proliferation of large healthcare databases and can often answer questions that cannot be answered in randomized studies due to resource constraints. However, the results of nonrandomized studies are much more likely to be impacted by confounding bias, and the existence of unmeasured confounders can never be completely ruled out. Furthermore, nonrandomized studies require more complex design considerations which can sometimes result in design-related biases. We discuss questions that can help investigators or evidence consumers evaluate the potential impact of confounding or other biases on their findings: Does the design emulate a hypothetical randomized trial design? Is the comparator or control condition appropriate? Does the primary analysis adjust for measured confounders? Do sensitivity analyses quantify the potential impact of residual confounding? Are methods open to inspection and (if possible) replication? Designing a high-quality nonrandomized study of medications remains challenging and requires broad expertise across a range of disciplines, including relevant clinical areas, epidemiology, and biostatistics. The questions posed in this paper provide a guiding framework for assessing the credibility of nonrandomized RWE and could be applied across many clinical questions.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.