A Blueprint for the Conduct of Large, Multisite Trials in Telemedicine.
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ABSTRACT:
SUBMITTER: Commiskey P
PROVIDER: S-EPMC8491114 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:The optimal approach to a clinical physical examination via telemedicine is still being explored. The medical community has no standardized or widely followed criteria for telemedicine examinations, so a broad spectrum of approaches is used. Unfortunately, the need for telemedicine is outpacing physical examination validation research. Given that certain specialties have been using telemedicine longer than others, lessons from those specialties might aid in developing standardized protocols for telemedicine. Neurology has been at the forefront of telemedicine use, initially through stroke care and later in multiple subspecialties. We present a framework for optimizing the history taking and physical examination process via telemedicine based on our experience in neurology. This mainly includes remotely examining a patient unassisted or with an untrained assistant present on the patient side of the connection. We also discuss the need for trained, certified assistants to assist the off-site physician in history taking and physical examination. These certified assistants would be allied health professionals who perform high-quality cued patient examinations under direct physician supervision with no responsibility to diagnose or treat. This contrasts with the approach seen in advanced practice providers such as physician assistants and nurse practitioners who undergo years of training to diagnose and treat patients under supervision. This training process would serve as a stepping stone for the development of dedicated certification programs for neurology and other medical specialties; however, assessments of practical training, costs, implementation, and longitudinal quality are warranted.
| S-EPMC8529478 | biostudies-literature
Project description:ObjectiveTo systematically review the conduct and reporting of formula trials.DesignSystematic review.Data sourcesMedline, Embase, and Cochrane Central Register of Controlled Trials (CENTRAL) were searched from 1 January 2006 to 31 December 2020.Review methodsIntervention trials comparing at least two formula products in children less than three years of age were included, but not trials of human breast milk or fortifiers of breast milk. Data were extracted in duplicate and primary outcome data were synthesised for meta-analysis with a random effects model weighted by the inverse variance method. Risk of bias was evaluated with Cochrane risk of bias version 2.0, and risk of undermining breastfeeding was evaluated according to published consensus guidance. Primary outcomes of the trials included in the systematic review were identified from clinical trial registries, protocols, or trial publications.Results22 201 titles were screened and 307 trials were identified that were published between 2006 and 2020, of which 73 (24%) trials in 13 197 children were prospectively registered. Another 111 unpublished but registered trials in 17 411 children were identified. Detailed analysis was undertaken for 125 trials (23 757 children) published since 2015. Seventeen (14%) of these recently published trials were conducted independently of formula companies, 26 (21%) were prospectively registered with a clear aim and primary outcome, and authors or sponsors shared prospective protocols for 11 (9%) trials. Risk of bias was low in five (4%) and high in 100 (80%) recently published trials, mainly because of inappropriate exclusions from analysis and selective reporting. For 68 recently published superiority trials, a pooled standardised mean difference of 0.51 (range -0.43 to 3.29) was calculated with an asymmetrical funnel plot (Egger's test P<0.001), which reduced to 0.19 after correction for asymmetry. Primary outcomes were reported by authors as favourable in 86 (69%) trials, and 115 (92%) abstract conclusions were favourable. One of 38 (3%) trials in partially breastfed infants reported adequate support for breastfeeding and 14 of 87 (16%) trials in non-breastfed infants confirmed the decision not to breastfeed was firmly established before enrolment in the trial.ConclusionsThe results show that formula trials lack independence or transparency, and published outcomes are biased by selective reporting.Systematic review registrationPROSPERO 2018 CRD42018091928.
| S-EPMC8513520 | biostudies-literature
Project description:Diffuse large B cell lymphoma (DLBCL) comprises multiple molecular and biological subtypes, resulting in a broad range of clinical outcomes. With standard chemoimmunotherapy, there remains an unacceptably high treatment failure rate in certain DLBCL subsets: activated B cell (ABC) DLBCL, double-hit lymphoma defined by the dual translocation of MYC and BCL2, dual protein-expressing lymphomas defined by the overexpression of MYC and BCL2, and older patients and those with central nervous system involvement. The main research challenges for DLBCL are to accurately identify molecular subsets and to determine if specific chemotherapy platforms and targeted agents offer differential benefit. The ultimate goal should be to maximize initial cure rates to improve long-term survival while minimizing toxicity. In particular, a frontline trial should focus on biologically defined risk groups not likely to be cured with cyclophosphamide, doxorubicin, vincristine, and prednisone plus rituximab (R-CHOP). An additional challenge is to develop effective and personalized strategies in the relapsed setting, for which there is no current standard other than autologous stem cell transplantation, which benefits a progressively smaller proportion of patients. Relapsed/refractory DLBCL is the ideal setting for testing novel agents and new biomarker tools and will require a national call for biopsies to optimize discovery in this setting. Accordingly, the development of tools with both prognostic and predictive utility and the individualized application of new therapies should be the main priorities. This report identifies clinical research priorities for critical areas of unmet need in this disease.
| S-EPMC6080361 | biostudies-other
Project description:Various studies have highlighted the importance of ethnic differences. The consideration of ethnic differences in the field of individualized pharmacotherapy is imperative. Therefore, various organizations and networks across countries should aim to conduct multicountry and multiregional clinical trials (MRCTs). If there is solid evidence available to evaluate the existence of ethnic differences between the same regional areas, it will lead to an increase in the efficiency of drug development. The purpose of this paper was to compare the approval dosing regimen among four Asian countries (Korea, Japan, China, and Taiwan) and elucidate the readiness and current status of the implementation of the International Conference on Harmonization (ICH) E17 guidelines on MRCTs. Reducing unnecessary clinical trials via multinational clinical trials in East Asian countries is also suggested. The approved dosing regimens for some drugs in the four Asian countries were similar; however, some differences might be caused by differences in legislation, even though there were no ethnic differences. This indicates that there are several roles to be expected of the Asia Clinical Pharmacology study network for exploratory MRCTs, which would lead to the accumulation of evidence for MRCTs, ultimately accelerating the efficiency of drug development in East Asian countries. The exposure of the new treatment to the necessary patients through collaborative research coordination and simultaneous multinational subject recruitment would serve its role in providing East Asia with specific personalized medicine with a high treatment success rate.
| S-EPMC8604233 | biostudies-literature
Project description:In the field of human nutrition, randomized controlled trials (RCTs) are considered the gold standard for establishing causal relations between exposure to nutrients, foods, or dietary patterns and prespecified outcome measures, such as body composition, biomarkers, or event rates. Evidence-based dietary guidance is frequently derived from systematic reviews and meta-analyses of these RCTs. Each decision made during the design and conduct of human nutrition RCTs will affect the utility and generalizability of the study results. Within the context of limited resources, the goal is to maximize the generalizability of the findings while producing the highest quality data and maintaining the highest levels of ethics and scientific integrity. The aim of this document is to discuss critical aspects of conducting human nutrition RCTs, including considerations for study design (parallel, crossover, factorial, cluster), institutional ethics approval (institutional review boards), recruitment and screening, intervention implementation, adherence and retention assessment, and statistical analyses considerations. Additional topics include distinguishing between efficacy and effectiveness, defining the research question(s), monitoring biomarker and outcome measures, and collecting and archiving data. Addressed are specific aspects of planning and conducting human nutrition RCTs, including types of interventions, inclusion/exclusion criteria, participant burden, randomization and blinding, trial initiation and monitoring, and the analysis plan.
| S-EPMC7849995 | biostudies-literature
Project description:Scientifically rigorous clinical trials are needed to test continuous positive airway pressure's (CPAP) effect on important clinical endpoints known to be associated with obstructive sleep apnea, such as myocardial infarction, cardiac arrhythmias, stroke, mortality, seizures, and cognitive function. In this "Special Article," we review the regulatory and ethical issues that surround the design and conduct of CPAP trials, including selection of the appropriate control condition, exclusion criteria, and follow-up duration.
| S-EPMC3041615 | biostudies-other
Project description:BackgroundTelemedicine modalities, such as videoconferencing, are used by health care providers to remotely deliver health care to patients. Telemedicine use in pediatrics has increased in recent years. This has resulted in improved health care access, optimized disease management, progress in the monitoring of health conditions, and fewer exposures to patients with illnesses during pandemics (eg, the COVID-19 pandemic).ObjectiveWe aimed to systematically evaluate the most recent evidence on the feasibility and accessibility of telemedicine services, patients' and care providers' satisfaction with these services, and treatment outcomes related to telemedicine service use among pediatric populations with different health conditions.MethodsStudies were obtained from the PubMed database on May 10, 2020. We followed the PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) guidelines. In this review, we included randomized controlled trials from the last 10 years that used a telemedicine approach as a study intervention or assessed telemedicine as a subspecialty of pediatric care. Titles and abstracts were independently screened based on the eligibility criteria. Afterward, full texts were retrieved and independently screened based on the eligibility criteria. A standardized form was used to extract the following data: publication title, first author's name, publication year, participants' characteristics, study design, the technology-based approach that was used, intervention characteristics, study goals, and study findings.ResultsIn total, 11 articles met the inclusion criteria and were included in this review. All studies were categorized as randomized controlled trials (8/11, 73%) or cluster randomized trials (3/11, 27%). The number of participants in each study ranged from 22 to 400. The health conditions that were assessed included obesity (3/11, 27%), asthma (2/11, 18%), mental health conditions (1/11, 9%), otitis media (1/11, 9%), skin conditions (1/11, 9%), type 1 diabetes (1/11, 9%), attention deficit hyperactivity disorder (1/11, 9%), and cystic fibrosis-related pancreatic insufficiency (1/11). The telemedicine approaches that were used included patient and doctor videoconferencing visits (5/11, 45%), smartphone-based interventions (3/11, 27%), telephone counseling (2/11, 18%), and telemedicine-based screening visits (1/11, 9%). The telemedicine interventions in all included studies resulted in outcomes that were comparable to or better than the outcomes of control groups. These outcomes were related to symptom management, quality of life, satisfaction, medication adherence, visit completion rates, and disease progression.ConclusionsAlthough more research is needed, the evidence from this review suggests that telemedicine services for the general public and pediatric care are comparable to or better than in-person services. Patients, health care professionals, and caregivers may benefit from using both telemedicine services and traditional, in-person health care services. To maximize the potential of telemedicine, future research should focus on improving patients' access to care, increasing the cost-effectiveness of telemedicine services, and eliminating barriers to telemedicine use.
| S-EPMC8078694 | biostudies-literature
Project description:AimsTo evaluate, using quantitative and qualitative approaches, published data on the design and conduct of decentralised clinical trials (DCTs).MethodsWe searched MEDLINE, EMBASE, CENTRAL, PsycINFO, ProQuest Dissertations and Theses, ClinicalTrials.gov, OpenGrey and Google Scholar for publications reporting, discussing, or evaluating decentralised clinical research methods. Reports of randomised clinical trials using decentralised methods were included in a focused quantitative analysis with a primary outcome of number of randomised participants. All publications discussing or evaluating DCTs were included in a wider qualitative analysis to identify advantages, disadvantages, facilitators, barriers and stakeholder opinions of decentralised clinical trials. Quantitative data were summarised using descriptive statistics, and qualitative data analysed using a thematic approach.ResultsInitial searches identified 19 704 articles. After removal of duplicates, 18 553 were screened, resulting in 237 eligible for full-text assessment. Forty-five trials were included in the quantitative analysis; 117 documents were included in the qualitative analysis. Trials were widely heterogeneous in design and reporting, precluding meta-analysis of the effect of DCT methods on the primary recruitment outcome. Qualitative analysis formulated 4 broad themes: value, burden, safety and equity. Participant and stakeholder experiences of DCTs were incompletely represented.ConclusionDCTs are developing rapidly. However, there is insufficient evidence to confirm which methods are most effective in trial recruitment, retention, or overall cost. The identified advantages, disadvantages, facilitators and barriers should inform the development of DCT methods. We recommend further research on how DCTs are experienced and perceived by participants and stakeholders to maximise potential benefits.
| S-EPMC9306873 | biostudies-literature
Project description:Clinical trials have led to major advances in inflammatory bowel disease (IBD) care over the last few decades, yet in that time most clinical trial protocols in IBD have remained markedly the same. Many IBD protocols often still require face-to-face visits and monitoring, hospital-based medication administration, paper-based forms and questionnaires, and short follow-up periods resulting in limited long-term data. These factors have recently been recognized as likely contributors to the low recruitment and lack of diversity of participants across clinical trials in IBD. However, with increasing technological advances, there is now an opportunity for improvement. This article assesses a range of virtual innovations for how they may offer digital solutions to challenges currently encountered in IBD clinical trials. Such solutions include consideration for increasing patient diversity, digital invitation, remote consent and recruitment, virtual visits, remote patient monitoring and data collection, remote medication delivery and administration, remote clinical trial monitoring, and routinely collected health data for long-term follow-up. Adoption of virtual technology may drive the field toward patient centricity and more efficient trial protocols to allow for a new era in IBD clinical trials.
| S-EPMC10524427 | biostudies-literature
Project description:Regional abnormalities in brain structure and function, as well as disrupted connectivity, have been found repeatedly in adolescents with conduct disorder (CD). Yet, the large-scale brain topology associated with CD is not well characterized, and little is known about the systematic neural mechanisms of CD. We employed graphic theory to investigate systematically the structural connectivity derived from cortical thickness correlation in a group of patients with CD (N = 43) and healthy controls (HCs, N = 73). Nonparametric permutation tests were applied for between-group comparisons of graphical metrics. Compared with HCs, network measures including global/local efficiency and modularity all pointed to hypo-functioning in CD, despite of preserved small-world organization in both groups. The hubs distribution is only partially overlapped with each other. These results indicate that CD is accompanied by both impaired integration and segregation patterns of brain networks, and the distribution of highly connected neural network 'hubs' is also distinct between groups. Such misconfiguration extends our understanding regarding how structural neural network disruptions may underlie behavioral disturbances in adolescents with CD, and potentially, implicates an aberrant cytoarchitectonic profiles in the brain of CD patients.
| S-EPMC5107936 | biostudies-literature