ReviewClinical pharmacology and therapeutics2022
A Useful and Sustainable Role for N-of-1 Trials in the Healthcare Ecosystem.
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 20 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
20 citing papers in PubMed.
- Strengthening Rural Health With N-of-1 Trials and Single-Case Experimental Designs.The Australian journal of rural health · 2026Article
- Digital N-of-1 trials and their application in experimental physiology.Experimental physiology · 2026Review
- Five tenets for advancing evidence-based precision medicine.Nature medicine · 2026Review
- N-of-1 trials in clinical research: Methodological foundations, statistical approaches and implementation challenges.British journal of clinical pharmacology · 2026Review
- From precision interventions to precision health.Nature communications · 2025Review
- Integrating N-of-1 Trials Into Learning Health Care Systems.JAMA neurology · 2025Article
- Tumor-Agnostic Therapies in Practice: Challenges, Innovations, and Future Perspectives.Cancers · 2025Review
- The Role of Molecular Profiling in De-Escalation of Toxic Therapy in Breast Cancer.International journal of molecular sciences · 2025Review
- Development of a novel tool for individual treatment trials in mucopolysaccharidosis.Journal of inherited metabolic disease · 2025Article
- The role of single case experimental designs in evidence creation in rehabilitation.European journal of physical and rehabilitation medicine · 2024Article
- Leveraging Single-Case Experimental Designs to Promote Personalized Psychological Treatment: Step-by-Step Implementation Protocol with Stakeholder Involvement of an Outpatient Clinic for Personalized Psychotherapy.Administration and policy in mental health · 2024Article
- Emerging therapeutic drug monitoring technologies: considerations and opportunities in precision medicine.Frontiers in pharmacology · 2024Review
- Individualized interventions for rare genetic conditions and the research-treatment spectrum: Stakeholder perspectives.Genetics in medicine : official journal of the American College of Medical Genetics · 2023Article
- Exploring human biology with N-of-1 clinical trials.Cambridge prisms. Precision medicine · 2023Article
- The use of N-of-1 trials to generate real-world evidence for optimal treatment of individuals and populations.Journal of clinical and translational science · 2023Article
- Development, implementation, and dissemination of operational innovations across the trial innovation network.Journal of clinical and translational science · 2023Article
- From a decentralized clinical trial to a decentralized and clinical-trial-in-a-box platform: Towards patient-centric and equitable trials.Journal of clinical and translational science · 2023Article
- Applying real-world data from expanded-access ("compassionate use") patients to drug development.Journal of clinical and translational science · 2023Article
- RNA-based therapeutics for neurological diseases.RNA biology · 2022Review
- Personalized Data Science and Personalized (N-of-1) Trials: Promising Paradigms for Individualized Health Care.Harvard data science review · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
19 authors.
Funding
Abstract
Clinicians and patients often try a treatment for an initial period to inform longer-term therapeutic decisions. A more rigorous approach involves N-of-1 trials. In these single-patient crossover trials, typically conducted in patients with chronic conditions, individual patients are given candidate treatments in a double-blinded, random sequence of alternating periods to determine the most effective treatment for that patient. However, to date, these trials are rarely done outside of research settings and have not been integrated into general care where they could offer substantial benefit. Designating this classical, N-of-1 trial design as type 1, there also are new and evolving uses of N-of-1 trials that we designate as type 2. In these, rather than focusing on optimizing treatment for chronic diseases when multiple approved choices are available, as is typical of type 1, a type 2 N-of-1 trial tests treatments designed specifically for a patient with a rare disease, to facilitate personalized medicine. While the aims differ, both types face the challenge of collecting individual-patient evidence using standard, trusted, widely accepted methods. To fulfill their potential for producing both clinical and research benefits, and to be available for wide use, N-of-1 trials will have to fit into the current healthcare ecosystem. This will require generalizable and accepted processes, platforms, methods, and standards. This also will require sustainable value-based arrangements among key stakeholders. In this article, we review opportunities, stakeholders, issues, and possible approaches that could support general use of N-of-1 trials and deliver benefit to patients and the healthcare enterprise. To assess and expand the benefits of N-of-1 trials, we propose multistakeholder meetings, workshops, and the generation of methods, standards, and platforms that would support wider availability and the value of N-of-1 trials.
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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.