{"doi":"10.1017/cts.2023.604","title":"The use of N-of-1 trials to generate real-world evidence for optimal treatment of individuals and populations","abstract":"Abstract Introduction: Ideally, real-world data (RWD) collected to generate real-world evidence (RWE) should lead to impact on the care and health of real-world patients. Deriving from care in which clinicians and patients try various treatments to inform therapeutic decisions, N-of-1 trials bring scientific methods to real-world practice. Methods: These single-patient crossover trials generate RWD and RWE by giving individual patients various treatments in a double-blinded way in sequential periods to determine the most effective treatment for a given patient. Results: This approach is most often used for patients with chronic, relatively stable conditions that provide the opportunity to make comparisons over multiple treatment periods, termed Type 1 N-of-1 trials. These are most helpful when there is heterogeneity of treatment effects among patients and no a priori best option. N-of-1 trials also can be done for patients with rare diseases, potentially testing only one treatment, to generate evidence for personalized treatment decisions, designated as Type 2 N-of-1 trials. With both types, in addition to informing individual’s treatments, when uniform protocols are used for multiple patients with the same condition, the data collected in the individual N-of-1 trials can be aggregated to provide RWD/RWE to inform more general use of the treatments. Thereby, N-of-1 trials can provide RWE for the care of individuals and for populations. Conclusions: To fulfill this potential, we believe N-of-1 trials should be built into our current healthcare ecosystem. To this end, we are building the needed infrastructure and engaging the stakeholders who should receive value from this approach.","journal":"Journal of Clinical and Translational Science","year":2023,"id":330101,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":16,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.6504,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2023-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":860608,"name":"Dorothy Dulko","orcid":"0000-0003-0274-1657","position":1,"is_corresponding":false},{"id":601023,"name":"David J. Greenblatt","orcid":"0000-0003-4719-6226","position":2,"is_corresponding":false},{"id":679877,"name":"Marisha E. Palm","orcid":"0000-0002-9760-7196","position":3,"is_corresponding":false},{"id":258773,"name":"Ludovic Trinquart","orcid":"0000-0002-3028-4900","position":4,"is_corresponding":false},{"id":89361,"name":"Harry P. Selker","orcid":"0000-0001-5322-7178","position":0,"is_corresponding":true}],"reference_count":26,"raw_metadata":null,"created_at":"2026-07-19T01:09:10.120703Z","pmid":"37830006","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}