ArticleThe Milbank quarterly2004
Evidence-based medicine, heterogeneity of treatment effects, and the trouble with averages.
Article in The Milbank quarterly, 2004. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 202 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
202 citing papers in PubMed, 5 syntheses or guidelines pooled it, 605 citations in OpenAlex.
- An umbrella review of adverse effects associated with antipsychotic medications: the need for complementary study designs.Neuroscience and biobehavioral reviews · 2023Pooled it
- How do psychologically based interventions for chronic musculoskeletal pain work? A systematic review and meta-analysis of specific moderators and mediators of treatment.Clinical psychology review · 2022Pooled it
- N-of-1 Randomized Intervention Trials in Health Psychology: A Systematic Review and Methodology Critique.Annals of behavioral medicine : a publication of the Society of Behavioral Medicine · 2018Pooled it
- Centre selection for clinical trials and the generalisability of results: a mixed methods study.PloS one · 2013Pooled it
- A systematic review of the Soteria paradigm for the treatment of people diagnosed with schizophrenia.Schizophrenia bulletin · 2008Pooled it
- A phase 2a double-blind, placebo-controlled, randomized clinical trial evaluating the efficacy and safety of NuGel, a novel topical GPCR19-mediated inflammasome inhibitor, in patients with mild to moderate atopic dermatitis: a proof-of-concept study withFrontiers in immunology · 2025Trial
- Variability in resistance training trajectories of breast cancer patients undergoing therapy.Supportive care in cancer : official journal of the Multinational Association of Supportive Care in Cancer · 2024Trial
- Providing Emotional Support During the Process of Multiple Sclerosis Diagnosis (PrEliMS): A Feasibility Randomised Controlled Trial.Clinical rehabilitation · 2024Trial
- An examination of the criterion-related validity of varying methods of indexing clinically significant change in posttraumatic stress disorder treatment.Psychological trauma : theory, research, practice and policy · 2024Trial
- Modulation of central pain mechanisms using high-definition transcranial direct current stimulation: A double-blind, sham-controlled study.European journal of pain (London, England) · 2023Trial
- A machine learning approach identifies modulators of heart failure hospitalization prevention among patients with type 2 diabetes: A revisit to the ACCORD trial.Journal of diabetes and its complications · 2022Trial
- Assessment of heterogeneous Head Start treatment effects on cognitive and social-emotional outcomes.Scientific reports · 2022Trial
- Effect of Mobile Device-Supported Single-Patient Multi-crossover Trials on Treatment of Chronic Musculoskeletal Pain: A Randomized Clinical Trial.JAMA internal medicine · 2018Trial
- Trial
- Understanding heterogeneity in response to antidiabetes treatment: a post hoc analysis using SIDES, a subgroup identification algorithm.Journal of diabetes science and technology · 2013Trial
- Emotional benefits of mindfulness-based stress reduction in older adults: the moderating roles of age and depressive symptom severity.Aging & mental health · 2013Trial
- Race and sex differences in response to endothelin receptor antagonists for pulmonary arterial hypertension.Chest · 2012Trial
- Predictors of abstinence: National Institute of Drug Abuse multisite buprenorphine/naloxone treatment trial in opioid-dependent youth.Journal of the American Academy of Child and Adolescent Psychiatry · 2011Trial
- Estimating treatment effects for individual patients based on the results of randomised clinical trials.BMJ (Clinical research ed.) · 2011Trial
- Predicting response to leuprolide of women with premenstrual dysphoric disorder by daily mood rating dynamics.Journal of psychiatric research · 2011Trial
142 more citing papers are in PubMed but not listed here.
Corrections and comments
- Erratum issued
Authors and funding
3 authors at 2 institutions in 1 country.
Funding
Abstract
Evidence-based medicine is the application of scientific evidence to clinical practice. This article discusses the difficulties of applying global evidence ("average effects" measured as population means) to local problems (individual patients or groups who might depart from the population average). It argues that the benefit or harm of most treatments in clinical trials can be misleading and fail to reveal the potentially complex mixture of substantial benefits for some, little benefit for many, and harm for a few. Heterogeneity of treatment effects reflects patient diversity in risk of disease, responsiveness to treatment, vulnerability to adverse effects, and utility for different outcomes. Recognizing these factors, researchers can design studies that better characterize who will benefit from medical treatments, and clinicians and policymakers can make better use of the results.
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.