Trial reportJournal of general internal medicine2010
Using N-of-1 trials to improve patient management and save costs.
Trial report in Journal of general internal medicine, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 54 papers, 2 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
54 citing papers in PubMed, 2 syntheses or guidelines pooled it, 90 citations in OpenAlex.
- N-of-1 trials in the clinical care of patients in developing countries: a systematic review.Trials · 2018Pooled it
- Pooled it
- Patient preference for second- and third-line therapies in type 2 diabetes: a prespecified secondary endpoint of the TriMaster study.Nature medicine · 2023Trial
- An n-of-one RCT for intravenous immunoglobulin G for inflammation in hereditary neuropathy with liability to pressure palsy (HNPP).Journal of neurology, neurosurgery, and psychiatry · 2016Trial
- Trial
- Increasing walking among older people: A test of behaviour change techniques using factorial randomised N-of-1 trials.Psychology & health · 2016Trial
- Low-Dose Naltrexone: What is the Evidence? A Narrative Review.Advances in therapy · 2026Review
- The N-of-1 Trials and where to use them in Rheumatology.Mediterranean journal of rheumatology · 2025Review
- Milasen: The Emerging Era of Patient-Customized N-of-1 Antisense Oligonucleotides as Therapeutic Agents for Genetic Diseases.Methods in molecular biology (Clifton, N.J.) · 2025Review
- Cyclosporine A in children with ABCA3 deficiency.Pediatric pulmonology · 2024Article
- An Efficient Way to Find Optimal Crossover Designs Using CVX for Precision Medicine.Journal of data science, statistics, and visualisation · 2024Article
- A Series of Personalized Virtual Light Therapy Interventions for Fatigue: Feasibility Randomized Crossover Trial for N-of-1 Treatment.JMIR formative research · 2023Article
- Individual Treatment Trials-Do Experts Know and Use This Option to Improve the Treatability of Mucopolysaccharidosis?Pharmaceuticals (Basel, Switzerland) · 2023Article
- The Value of Case Reports for Graduate Medical Education.Journal of graduate medical education · 2022Article
- A Useful and Sustainable Role for N-of-1 Trials in the Healthcare Ecosystem.Clinical pharmacology and therapeutics · 2022Review
- Article
- Alternative strategies in cardiac preclinical research and new clinical trial formats.Cardiovascular research · 2022Review
- A Series of Virtual Interventions for Chronic Lower Back Pain: A Feasibility Pilot Study for a Series of Personalized (N-of-1) Trials.Harvard data science review · 2022Article
- Conduct and Implementation of Personalized Trials in Research and Practice.Harvard data science review · 2022Article
- Status of N-of-1 Trials in Chronic Pain Management: A Narrative Review.Pain and therapy · 2021Review
Corrections and comments
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Authors and funding
11 authors at 7 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundN-of-1 trials test treatment effectiveness within an individual patient.
objectiveTo assess (i) the impact of three different N-of-1 trials on both clinical and economic outcomes over 12 months and (ii) whether the use of N-of-1 trials to target patients' access to high-cost drugs might be cost-effective in Australia.
designDescriptive study of management change, persistence, and costs summarizing three N-of-1 trials.
participantsVolunteer patients with osteoarthritis, chronic neuropathic pain or ADHD whose optimal choice of treatment was uncertain.
interventionsDouble-blind cyclical alternative medications for the three conditions. MEASURES: Detailed resource use, treatment and health outcomes (response) data collected by postal and telephone surveys immediately before and after the trial and at 3, 6 and 12 months. Estimated costs to the Australian healthcare system for the pre-trial vs. 12 months post-trial.
resultsParticipants persisting with the joint patient-doctor decision 12 months after trial completion were 32% for osteoarthritis, 45% for chronic neuropathic pain and 70% for the ADHD trials. Cost-offsets were obtained from reduced usage of non-optimal drugs, and reduced medical consultations. Drug costs increased for the chronic neuropathic pain and ADHD trials due to many patients being on either low-cost or no pharmaceuticals before the trial.
conclusionsN-of-1 trials are an effective method to identify optimal treatment in patients in whom disease management is uncertain. Using this evidence-based approach, patients and doctors tend to persist with optimal treatment resulting in cost-savings. N-of-1 trials are clinically acceptable and may be an effective way of rationally prescribing some expensive long-term medicines.
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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.