Cargando…
The use of N-of-1 trials to generate real-world evidence for optimal treatment of individuals and populations
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...
Autores principales: | , , , , |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Cambridge University Press
2023
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10565195/ https://www.ncbi.nlm.nih.gov/pubmed/37830006 http://dx.doi.org/10.1017/cts.2023.604 |
Sumario: | 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. |
---|