ArticleClinical epidemiology2019
Observational studies of treatment effectiveness: worthwhile or worthless?
Article in Clinical epidemiology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers, 5 of them syntheses that pooled it.
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
29 citing papers in PubMed, 5 syntheses or guidelines pooled it.
- Mucoactive agents in bronchiectasis: a systematic review and meta-analysis.European respiratory review : an official journal of the European Respiratory Society · 2026Pooled it
- Manipulation under Anesthesia versus Non-Surgical Treatment for Patients with Frozen Shoulder Contracture Syndrome: A Systematic Review.International journal of environmental research and public health · 2022Pooled it
- Methodological evaluation of bias in observational coronavirus disease 2019 studies on drug effectiveness.Clinical microbiology and infection : the official publication of the European Society of Clinical Microbiology and Infectious Diseases · 2021Pooled it
- The Impact of Antiretroviral Therapy on Neurocognitive Outcomes Among People Living with HIV in Low- and Middle-Income Countries (LMICs): A Systematic Review.AIDS and behavior · 2021Pooled it
- Pooled it
- Effectiveness of Galcanezumab and Traditional Oral Migraine Preventive Medications: Interim 3-Month Japan Subgroup Findings from the TRIUMPH Study.Advances in therapy · 2026Observational
- Results from TRIUMPH [EUROPE], a Real-World Observational Study on the 3-Month Effectiveness of Galcanezumab Versus Traditional Oral Migraine Preventive Medications.Pain and therapy · 2026Article
- Observational
- REAL-WORLD EFFECTIVENESS OF HIGH-DOSE DUAL THERAPY VS OPTIMIZED CLARITHROMYCIN TRIPLE THERAPY FOR HELICOBACTER PYLORI: A PROSPECTIVE COHORT STUDY WITH TEST-OF-CURE SENSITIVITY ANALYSES.Arquivos de gastroenterologia · 2026Observational
- Methotrexate associates with ischemic cardiovascular risk reduction in males but not females: a transatlantic cardiovascular consortium for people with rheumatoid arthritis observational study.Rheumatology international · 2025Observational
- Observational
- Association between use of sodium-glucose co-transporter-2 inhibitor and the risk of incident dementia: a population-based cohort study.BMJ open diabetes research & care · 2025Article
- Digital mental health interventions as stand-alone vs. augmented treatment as usual.BMC public health · 2024Article
- The role of early-phase trials and real-world evidence in drug development.Nature cardiovascular research · 2024Review
- Transdiagnostic symptom networks in relation to mental health service use in community youth.Clinical psychology & psychotherapy · 2023Article
- Evaluation of safety and effectiveness of remdesivir in treating COVID-19 patients after emergency use authorization study.Frontiers in pharmacology · 2023Article
- Association between statin use and the risk of gout in patients with hyperlipidemia: A population-based cohort study.Frontiers in pharmacology · 2023Article
- Mindfulness for internet use disorder: a study protocol of a systematic review and meta-analysis.BMJ open · 2022Article
- Current state of the evidence on community treatments for people with complex emotional needs: a scoping review.BMC psychiatry · 2022Article
- Methods to account for measured and unmeasured confounders in influenza relative vaccine effectiveness studies: A brief review of the literature.Influenza and other respiratory viruses · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Observational studies which evaluate effectiveness are often viewed with skepticism owing to the fact that patients are not randomized to treatment, meaning that results are more prone to bias. Therefore, randomized controlled trials remain the gold standard for evaluating treatment effectiveness. However, it is not always possible to conduct randomized trials. This may be due to financial constraints, for example, in identifying funding for a randomized trial for medicines that have already gained market authorization. There can also be challenges with recruitment, for example, of people with rare conditions or in hard-to-reach population subgroups. This is why observational studies are still needed. In this manuscript, we discuss how researchers can mitigate the risk of bias in the most common type of observational study design for evaluation of treatment effectiveness, the cohort study. We outline some key issues that warrant careful consideration at the outset when the question is being developed and the cohort study is being designed. We focus our discussion on the importance of deciding when to start follow-up in a study, choosing a comparator, managing confounding and measuring outcomes. We also illustrate the application of these considerations in a more detailed case study based on an examination of comparative effectiveness of two antidiabetic treatments using data collected during routine clinical practice.
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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.