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Introduction
Published in Wei Zhang, Fangrong Yan, Feng Chen, Shein-Chung Chow, Advanced Statistics in Regulatory Critical Clinical Initiatives, 2022
Wei Zhang, Fangrong Yan, Feng Chen, Shein-Chung Chow
An n-of-1 trial design is defined as a clinical trial design in which a single patient is the entire trial, a single case study. Thus, in an n-of-1 trial, n is the number of treatments and one is the single patient. An n-of-1 trial in which random allocation can be used to determine the order in which an experimental and a control are given to a patient. An n-of-1 trial is a multiple crossover study in a single participant.
Critical appraisal of randomized clinical trials
Published in O. Ajetunmobi, Making Sense of Critical Appraisal, 2021
In this type of study, competing treatments are randomized to different time periods for each trial subject. Each subject is then administered the respective treatments (including placebo) sequentially over allocated time periods (Figure 7.11). A typical n-of-1 trial may involve between 1 and 30 subjects.
Introduction
Published in Shein-Chung Chow, Innovative Statistics in Regulatory Science, 2019
A complete n-of-1 trial design has the following advantages: (i) each subject is at his/her own control; (ii) it allows a comparison between the test product and the placebo if the intended trial is a placebo-controlled study (this has lifted the ethical issue of using placebo on the patients with critical conditions); (iii) it allows estimates of intra-subject variability; (iv) it provides estimates for treatment effect in the presence of possible carry-over effect, and most importantly; (v) it requires less subjects for achieving the study objectives of the intended trial design. However, the n-of-1 trial design suffers from the drawbacks of (i) possible dropouts or missing data and (ii) patients’ disease status may change at each dosing period prior to dosing.
Trends in safety and cost of deep brain stimulation for treatment of movement disorders in the United States: 2002–2014
Published in British Journal of Neurosurgery, 2021
Hansen Deng, John K. Yue, Doris D. Wang
The United States (U.S.) Food and Drug Administration (FDA) approved DBS to treat ET in 1997, followed by PD in 2002.7 Dystonia was approved in 2003 as a humanitarian device exemption (HDE).8,9 In addition to FDA approval, there has been mounting evidence from randomized controlled trials (RCTs) that neurostimulation of the subthalamic nucleus (STN) or globus pallidus internus (GPi) is superior to the best medical therapy available in treating PD.10–12 Recent results from a novel N-of-1 trial by Hyam et al. provided Class I evidence on the efficacy of DBS in tremor reduction for patients with ET.13 Other RCTs on Vim DBS showed significant functional improvement in both PD and ET patients with resistant tremors, even 6-years post-implantation.14,15 Results from two sham-controlled RCTs, Kupsch et al. and Volkmann et al., demonstrated that pallidal neurostimulation reduced symptoms in patients with dystonia.16,17 Long-term follow-up showed that symptom reduction persisted at 12 months.18,19
Single-case Design Studies in Children with Cerebral Palsy: A Scoping Review
Published in Developmental Neurorehabilitation, 2020
Laura W.M.E. Beckers, Rosalinde A. Stal, Rob J.E.M Smeets, Patrick Onghena, Caroline H.G. Bastiaenen
Next to the systematic and thorough search and data charting process, the consultation phase in which an expert in SCD statistics and methodology reviewed the results strengthened the validity of this scoping review. Unfortunately, the lack of response to the newsletter items did not allow for the inclusion of grey literature. It is possible, therefore, that unpublished studies were missed. The CENT reporting standard was chosen along with the RoBiNT Scale to cover both the policies of medical and behavioral sciences. Unlike the alternative Single-Case Reporting Guideline In BEhavioural Interventions (SCRIBE), the CENT has been specifically designed for the medical N-of-1 trial, i.e., a multiple crossover design. As a consequence, not all items were equally relevant for each type of SCD. Hence, results of the quality or reporting assessment need to be interpreted with caution to avoid overestimating the inadequacy of reporting. Last, the inter-rater agreement of the risk of bias appraisal was slightly lower than the agreement of 81% between trained novice raters in an inter-rater reliability study of the RoBiNT Scale.10
Effectiveness of a multidisciplinary rehabilitation program for persons with acquired brain injury and executive dysfunction
Published in Disability and Rehabilitation, 2018
Frédérique Poncet, Bonnie Swaine, Hélène Migeot, Julie Lamoureux, Christine Picq, Pascale Pradat
One of the limitations of this study is the small number of subjects. However, given the individual approach used during the Cooking Activity, we feel the SCED used in this study to be most appropriate. Indeed, randomized clinical trials (RCTs) are powerful techniques for determining the efficacy of interventions. However, they may have practical limitations when applied to many rehabilitation settings and research questions [61]. Besides being difficult to use with groups of heterogeneous patients, clinical trials are often difficult to conduct while respecting realities of clinical settings, especially when time-consuming situational assessment tools are necessary to clearly establish a link between an intervention and its impact on patients’ activity and participation. We acknowledge that small-N research methods can only directly determine the best treatments for persons involved in a specific study [61]. Indeed SCED methodology has limited generalisability compared with RCTs. The generalisability of our study using SCED was however strengthened by the use of multiple baseline across many subjects (n = 7) and because the results were positive for six of the seven subjects (85.7% of the group). The second limitation is the absence of a control group; however, Perdices [15] supports the use of SCED as it builds into a control condition with the multiple baseline measures across behaviors pre- and post-program to determine treatment efficacy. Moreover, Tate et al. [63] advocate for the inclusion of the randomized n-of-1 trial (such as SCED) as Level 1 evidence likely to have major implications for what constitutes the evidence base of health interventions [63]. We believe the present study further supports the use of this experimental design in rehabilitations efficacy studies.