ArticleJAMA pediatrics2021
Experimental Designs to Optimize Treatments for Individuals: Personalized N-of-1 Trials.
Article in JAMA pediatrics, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 39 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
39 citing papers in PubMed, 71 citations in OpenAlex.
- Tamsulosin Deprescribing for Lower Urinary Tract Symptoms in Older Men: A Randomized Clinical Trial.JAMA network open · 2026Trial
- A Series of Personalized Melatonin Supplement Interventions for Poor Sleep: Feasibility Randomized Crossover Trial for Personalized N-of-1 Treatment.JMIR formative research · 2025Trial
- Trial
- Comparison of Bayesian Networks, G-estimation and linear models to estimate causal treatment effects in aggregated N-of-1 trials with carry-over effects.BMC medical research methodology · 2023Trial
- Artificial Intelligence in Rare Diseases: Workflow-Integrated Precision Kidney Care.Clinics and practice · 2026Review
- Precision medicine in mental health: applications, challenges, and recommendations.European psychiatry : the journal of the Association of European Psychiatrists · 2026Review
- N-of-1 trials in clinical research: Methodological foundations, statistical approaches and implementation challenges.British journal of clinical pharmacology · 2026Review
- Gut Microbial Composition, Oxidative Stress, and Immunity in Metabolic Disease: Toward Personalized Interventions.Antioxidants (Basel, Switzerland) · 2026Review
- Preliminary efficacy of eye movement desensitization and reprocessing for children aged 1.5-8 years with PTSD: a multiple baseline experimental design (European journal of psychotraumatology · 2025Article
- Feasibility Test of Personalized (N-of-1) Trials for Increasing Middle-Aged and Older Adults' Physical Activity.International journal of behavioral medicine · 2025Article
- Digital, Personalized Clinical Trials Among Older Adults, Lessons Learned From the COVID-19 Pandemic, and Directions for the Future: Aggregated Feasibility Data From Three Trials Among Older Adults.Journal of medical Internet research · 2025Article
- Behavioral Medicine Can Tackle Cardiovascular Disease Risk and Burden: A Call for New Priorities.Policy insights from the behavioral and brain sciences · 2025Article
- Survey and perspective on verification, validation, and uncertainty quantification of digital twins for precision medicine.NPJ digital medicine · 2025Review
- Pathogenetic therapeutic approaches for endocrine diseases based on antisense oligonucleotides and RNA-interference.Frontiers in endocrinology · 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
- Beyond Detection: Towards Actionable Sensing Research in Clinical Mental Healthcare.Proceedings of the ACM on interactive, mobile, wearable and ubiquitous technologies · 2024Article
- Preparing for Patient-Customized N-of-1 Antisense Oligonucleotide Therapy to Treat Rare Diseases.Genes · 2024Review
- A methodological review of randomised n-of-1 trials.Trials · 2024Review
- The Science of Precision Prevention: Research Opportunities and Clinical Applications to Reduce Cardiovascular Health Disparities.JACC. Advances · 2024Article
- Feasibility Test of Personalized (N-of-1) Trials for Increasing Middle-Aged and Older Adults' Physical Activity.Research square · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 3 institutions in 1 country.
Funding
Abstract
Conventional randomized clinical trials (RCTs) compare treatment effectiveness to provide support for evidence-based treatments that can be generalized to the average patient. However, the information obtained from RCTs may not always be useful for selecting the best treatment for individual patients. This article presents a complementary approach to identifying optimized treatments using experimental designs that focus on individuals. Personalized, or N-of-1, designs provide both a comparative analysis of treatments and a functional analysis demonstrating that changes in patient symptoms are likely because of the treatment implemented. This approach contributes to the zeitgeist of personalized medicine and provides clinicians with a paradigm for investigating optimal treatments for rare diseases for which RCTs are not always feasible, identifying personally effective treatments for patients with comorbidities who have historically been excluded from most RCTs, handling clinical situations in which patients respond idiosyncratically (either positively or negatively) to treatment, and shortening the time lag between identification and implementation of an evidence-based treatment. These designs merge experimental analysis of behavior methods used for decades in psychology with new methodological and statistical advances to assess significance levels of changes in individual patients, and they can be generalized to larger populations for meta-analytic purposes. This article presents a case for why these models are needed, an overview of how to apply personalized designs for different types of clinical scenarios, and a brief discussion of challenges associated with interpretation and implementation of personalized designs. The goal is to empower pediatricians to take personalized trial designs into clinical practice to identify optimal treatments for their patients.
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.