Background: N-of-1 trials are single patient, multiple crossover, and comparative effectiveness experiments. Despite their rating as "level 1" evidence, they are not routinely used in clinical medicine to evaluate the effectiveness of treatments.
Objective: We explored the potential for implementing a mobile app-based n-of-1 trial platform for collaborative use by clinicians and patients to support data-driven decisions around the treatment of insomnia.
Methods: A survey assessing awareness and utilization of n-of-1 trials was administered to healthcare professionals that frequently treat patients with insomnia at the Icahn School of Medicine at Mount Sinai in New York City.
Results: A total of 45 healthcare professionals completed the survey and were included in the analysis. We found that 64% (29/45) of healthcare professionals surveyed had not heard of n-of-1 trials. After a brief description of these methods, 75% (30/40) of healthcare professionals reported that they are likely or highly likely to use an app-based n-of-1 trial at least once in the next year if the service were free and easy to offer to their patients.
Conclusions: An app-based n-of-1 trials platform might be a valuable tool for clinicians and patients to identify the best treatments for insomnia. The lack of awareness of n-of-1 trials coupled with receptivity to their use suggests that educational interventions may address a current barrier to wider utilization of n-of-1 trials.
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http://dx.doi.org/10.3389/fpsyt.2020.530995 | DOI Listing |
Clin Trials
January 2025
Department of Biostatistics, University of Washington, Seattle, WA, USA.
Background: N-of-1 trials compare two or more treatment options for a single participant. These trials have been used to study options for chronic conditions such as arthritis and attention deficit hyperactivity disorder. In addition, they have been suggested as a means to study interventions in rare populations that may not be tractable to include in standard clinical trials, such as treatment options for HIV-positive patients in need of organ transplant.
View Article and Find Full Text PDFJ Med Genet
December 2024
Department of Clinical Genetics, Leiden University Medical Center, Leiden, the Netherlands
Background: Clinical trials for rare disorders have unique challenges due to low prevalence, patient phenotype variability and high expectations. These challenges are highlighted by our study on clonazepam in patients, a common cause of intellectual disability. Previous studies on Arid1b-haploinsufficient mice showed positive effects of clonazepam on various cognitive aspects.
View Article and Find Full Text PDFPalliat Med
December 2024
Palliative and Supportive Care, Mater Misericordiae Ltd., South Brisbane, QLD, Australia.
Background: Xerostomia is a common and difficult symptom experienced by patients with cancer. Pilocarpine is a cholinergic agent that stimulates salivation.
Aim: To assess the feasibility of conducting a N-of-1 trial to determine the efficacy of pilocarpine orally dissolving tablets in patients with xerostomia.
Biomed Eng Online
December 2024
Department of Clinical Physiology, Motion Analysis Center, University Hospital of Toulouse, Hôpital de Purpan, Toulouse, France.
Background: Stroke is the leading cause of acquired motor deficiencies in adults. Restoring prehension abilities is challenging for individuals who have not recovered active hand opening capacities after their rehabilitation. Self-triggered functional electrical stimulation applied to finger extensor muscles to restore grasping abilities in daily life is called grasp neuroprosthesis (GNP) and remains poorly accessible to the post-stroke population.
View Article and Find Full Text PDFZhonghua Liu Xing Bing Xue Za Zhi
December 2024
Department of Biostatistics, School of Public Health, Nanjing Medical University, Nanjing211166, China.
Due to the limited number of cases, conducting large-scale clinical trials for rare diseases is challenging. This review introduces several small sample statistical designs tailored for rare diseases, including crossover design, -of-1 design, randomized placebo-phase design, randomized withdrawal design, group sequential design, and adaptive design. It discusses the advantages, disadvantages, and application scenarios of these designs.
View Article and Find Full Text PDFEnter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!