scholarly journals Master Protocols for Precision Medicine in Oncology: Overcoming Methodology of Randomized Clinical Trials

Life ◽  
2021 ◽  
Vol 11 (11) ◽  
pp. 1253
Author(s):  
Raimondo Di Liello ◽  
Maria Carmela Piccirillo ◽  
Laura Arenare ◽  
Piera Gargiulo ◽  
Clorinda Schettino ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Personalized Medicine ◽  
Clinical Research ◽  
Targeted Therapies ◽  
Randomized Clinical Trials ◽  
Trial Methodology ◽  
Clinical Trial Methodology ◽  
Therapeutic Algorithms ◽  
New Compounds

Randomized clinical trials are considered the milestones of clinical research in oncology, and guided the development and approval of new compounds so far. In the last few years, however, molecular and genomic profiling led to a change of paradigm in therapeutic algorithms of many cancer types, with the spread of different biomarker-driven therapies (or targeted therapies). This scenario of “personalized medicine” revolutionized therapeutic strategies and the methodology of the supporting clinical research. New clinical trial designs are emerging to answer to the unmet clinical needs related to the development of these targeted therapies, overcoming the “classical” structure of randomized studies. Innovative trial designs able to evaluate more than one treatment in the same group of patients or many groups of patients with the same treatment (or both) are emerging as a possible future standard in clinical trial methodology. These are identified as “master protocols”, and include umbrella, basket and platform trials. In this review, we described the main characteristics of these new trial designs, focusing on the opportunities and limitations of their use in the era of personalized medicine.

scholarly journals Clinical trials in the Coronavirus era: management of risks

2020 ◽  
pp. 18-24
Author(s):  
Aleksey Vodovozov ◽  
Keyword(s):  
Risk Assessment ◽  
Clinical Trial ◽  
Decision Making ◽  
Clinical Trials ◽  
Trial Methodology ◽  
Ministry Of Health ◽  
Trial Team ◽  
Clinical Trial Methodology ◽  
Regulatory Authorities ◽  
The World

The COVID-19 pandemic has caused unique problems facing the clinical trials (CT) community both in terms of the rapid implementation of CTs of COVID-19 drugs and vaccines, and many ongoing non-COVID-19 CTs that are either suspended or adapted to new realities. The CT organizers have played a key role in decision making, risk assessment and adaptation of trial processes, working side by side with other members of the trial team. Regulatory authorities (FDA, EMA, Ministry of Health of Russia, etc.), which issued initial recommendations for adapting the clinical trial methodology to new conditions as far back as March 2020, and then later on, generalized the experience in the management for clinical trials. The recommendations made based on the reviewed experience can help all CT parties to cope with the risks associated with both COVID-19 itself and anti-epidemic measures in different countries of the world with minimal losses.

scholarly journals Challenges in the development of clinical trials for major depressive disorder: lessons learned from trials in minor depression

2002 ◽  
Vol 4 (4) ◽  
pp. 402-407
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Major Depressive Disorder ◽  
Depressive Disorder ◽  
Lessons Learned ◽  
Minor Depression ◽  
Trial Methodology ◽  
Major Depressive ◽  
Clinical Trial Methodology ◽  
Theoretical Assumptions

This paper reviews some of the challenges faced by individuals who design and implement clinical trials of potential antidepressant medications. Particular emphasis is placed on questioning the validity of some of the theoretical assumptions that form the underpinnings of most conventional trials. Work from our group developing clinical trial methodology for minor depression is used as an example of how alternate constructs may be helpful to differentiate drug-placebo differences.

Statistical Analysis of a Clinical Trial in Orthostatic Hypotension -a Nonparametric Approach

1987 ◽  
Vol 26 (01) ◽  
pp. 47-52
Author(s):  
R. Haux ◽  
H. Immich ◽  
M. Schumacher
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Statistical Analysis ◽  
Mathematical Models ◽  
Orthostatic Hypotension ◽  
Nonparametric Tests ◽  
Trial Methodology ◽  
Clinical Trial Methodology ◽  
Nonparametric Approach ◽  
Serial Measurements

SummarySome basic problems and considerations of analyzing clinical trials, in which a large amount of data is recorded on very few patients - mostly in the form of serial measurements -, are highlighted within the framework of a clinical trial in orthostatic hypotension. The method suggested to analyze these kinds of trials renounces the use of complex mathematical models and complicated statistical methods. It is simply based on the derivation of relevant aspects of the patient’s profiles and the use of well-known nonparametric tests and requires only a very simple study design. With this procedure, however, it is possible to satisfy at least the basic requirements of clinical trial methodology.

Global Trials, Local Bodies: Negotiating Difference and Sameness in Indian For-profit Clinical Trials

2020 ◽  
pp. 016224392096381
Author(s):  
Sibille Merz
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Trial Methodology ◽  
Clinical Trial Methodology ◽  
Principal Investigators ◽  
For Profit ◽  
Clinical Research Organization ◽  
Cultural Specificity ◽  
Local Biologies ◽  
Trial Participants

Global clinical trials depend on a range of standards in order for research results to be comparable. As standardization is more than a mere technical exercise, tensions can arise when things are not uniform. This paper uses empirical data from interviews with principal investigators as well as Clinical Research Organization and pharmaceutical industry representatives working in India’s clinical trial industry to critically examine the ways Indian researchers navigate quests for standardization. It turns the analytical lens to the often obfuscated work of standardization aiming to transcend the biological and cultural specificity of research participants and research sites. Drawing on the concept of local biologies, it illustrates that the universal body presumed by clinical trial methodology is, in fact, a specifically Euro-American one: Indian participants not only need to be made globally comparable but also aligned with the drugs’ future consumers. Focusing on the tensions between biomedicine’s postulation of bodily universality and trial participants’ local biologies, this paper advances recent interventions problematizing the structural violence undergirding global clinical trials. It also contributes to the literature on local biologies in its discussion of how these are negotiated in Indian for-profit clinical trials.

scholarly journals Knowledge and Perception Regarding Clinical Trials Among Dental Students-A Questionnaire Based Study

2020 ◽  
Vol 11 (SPL3) ◽  
pp. 1626-1633
Author(s):  
Thaslima Nandhini J S ◽  
Vignesh Ravindran ◽  
Jeevitha M
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Undergraduate Students ◽  
Good Clinical Practice ◽  
Dental Students ◽  
Trial Methodology ◽  
Chi Square ◽  
Postgraduate Students ◽  
Clinical Trial Methodology ◽  
Validated Questionnaire

Clinical trials play an important role in improvement of quality on healthcare practice. Good clinical practice is a backbone of conductance of trials. A lack of knowledge may translate to a negative perception towards clinical trials. The study aims to evaluate the awareness of clinical trials among undergraduate dental students in a private dental institute. We have quantified the knowledge and perception of clinical trials by a structured validated questionnaire. The questionnaire was administered to dental students from first year undergraduate to third year Postgraduate. The percentage of questions answered were calculated and tabulated in the excel sheet. Data were analysed using SPSS statistical software and the chi-square test was done. P valve was set as 0.05 as a level of significance. From statistical analysis, postgraduate students (59.2%) had better knowledge than undergraduate students (40.8%). Around (46.7%) had been involved in clinical trial training. Within the limitations of current study, students pursuing postgraduate had a better knowledge of clinical trial methodology than compared to undergraduate students.

scholarly journals The role of machine learning in clinical research: transforming the future of evidence generation

Trials ◽  
2021 ◽  
Vol 22 (1) ◽  
Author(s):  
E. Hope Weissler ◽  
Tristan Naumann ◽  
Tomas Andersson ◽  
Rajesh Ranganath ◽  
Olivier Elemento ◽  
...  
Keyword(s):  
Machine Learning ◽  
Clinical Trial ◽  
Clinical Research ◽  
Evidence Base ◽  
Great Promise ◽  
Trial Methodology ◽  
Non Profit ◽  
Clinical Trial Methodology ◽  
Artificial Intelligence Technology ◽  
Multi Stakeholder

Abstract Background Interest in the application of machine learning (ML) to the design, conduct, and analysis of clinical trials has grown, but the evidence base for such applications has not been surveyed. This manuscript reviews the proceedings of a multi-stakeholder conference to discuss the current and future state of ML for clinical research. Key areas of clinical trial methodology in which ML holds particular promise and priority areas for further investigation are presented alongside a narrative review of evidence supporting the use of ML across the clinical trial spectrum. Results Conference attendees included stakeholders, such as biomedical and ML researchers, representatives from the US Food and Drug Administration (FDA), artificial intelligence technology and data analytics companies, non-profit organizations, patient advocacy groups, and pharmaceutical companies. ML contributions to clinical research were highlighted in the pre-trial phase, cohort selection and participant management, and data collection and analysis. A particular focus was paid to the operational and philosophical barriers to ML in clinical research. Peer-reviewed evidence was noted to be lacking in several areas. Conclusions ML holds great promise for improving the efficiency and quality of clinical research, but substantial barriers remain, the surmounting of which will require addressing significant gaps in evidence.

The Use of Digital Clinical Trial Methods in the Evaluation of Digital Therapeutics: A Scoping Review (Preprint)

2019 ◽  
Author(s):  
Allison Hirsch ◽  
Mahip Grewal ◽  
Anthony James Martorell ◽  
Brian Michael Iacoviello
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Clinical Research ◽  
Scoping Review ◽  
Digital Technologies ◽  
Good Clinical Practice ◽  
The United States ◽  
Clinical Trial Research ◽  
Digital Methods ◽  
Clinical Trial Methods

BACKGROUND Digital Therapeutics (DTx) provide evidence based therapeutic health interventions that have been clinically validated to deliver therapeutic outcomes, such that the software is the treatment. Digital methodologies are increasingly adopted to conduct clinical trials due to advantages they provide including increases in efficiency and decreases in trial costs. Digital therapeutics are digital by design and can leverage the potential of digital and remote clinical trial methods. OBJECTIVE The principal purpose of this scoping review is to review the literature to determine whether digital technologies are being used in DTx clinical research, which type are being used and whether publications are noting any advantages to their use. As DTx development is an emerging field there are likely gaps in the knowledge base regarding DTx and clinical trials, and the purpose of this review is to illuminate those gaps. A secondary purpose is to consider questions which emerged during the review process including whether fully remote digital clinical research is appropriate for all health conditions and whether digital clinical trial methods are inline with the principles of Good Clinical Practice. METHODS 1,326 records were identified by searching research databases and 1,227 reviewed at the full-article level in order to determine if they were appropriate for inclusion. Confirmation of clinical trial status, use of digital clinical research methods and digital therapeutic status as well as inclusion and exclusion criteria were applied in order to determine relevant articles. Digital methods employed in DTx research were extracted from each article and these data were synthesized in order to determine which digital methods are currently used in clinical trial research. RESULTS After applying our criteria for scoping review inclusion, 11 articles were identified. All articles used at least one form of digital clinical research methodology enabling an element of remote research. The most commonly used digital methods are those related to recruitment, enrollment and the assessment of outcomes. A small number of articles reported using other methods such as online compensation (n = 3), or digital reminders for participants (n = 5). The majority of digital therapeutics clinical research using digital methods is conducted in the United States and increasing number of articles using digital methods are published each year. CONCLUSIONS Digital methods are used in clinical trial research evaluating DTx, though not frequently as evidenced by the low proportion of articles included in this review. Fully remote clinical trial research is not yet the standard, more frequently authors are using partially remote methods. Additionally, there is tremendous variability in the level of detail describing digital methods within the literature. As digital technologies continue to advance and the clinical research DTx literature matures, digital methods which facilitate remote research may be used more frequently.

Shared-Task Worklists Improve Clinical Trial Recruitment Workflow in an Academic Emergency Department

2021 ◽  
Vol 12 (02) ◽  
pp. 293-300
Author(s):  
Kevin S. Naceanceno ◽  
Stacey L. House ◽  
Phillip V. Asaro
Keyword(s):  
Clinical Trial ◽  
Emergency Department ◽  
Clinical Trials ◽  
Clinical Research ◽  
Substantial Reduction ◽  
Text Message ◽  
High Volume ◽  
Text Messages ◽  
Shared Task ◽  
Clinical Research Coordinators

Abstract Background Clinical trials performed in our emergency department at Barnes-Jewish Hospital utilize a centralized infrastructure for alerting, screening, and enrollment with rule-based alerts sent to clinical research coordinators. Previously, all alerts were delivered as text messages via dedicated cellular phones. As the number of ongoing clinical trials increased, the volume of alerts grew to an unmanageable level. Therefore, we have changed our primary notification delivery method to study-specific, shared-task worklists integrated with our pre-existing web-based screening documentation system. Objective To evaluate the effects on screening and recruitment workflow of replacing text-message delivery of clinical trial alerts with study-specific shared-task worklists in a high-volume academic emergency department supporting multiple concurrent clinical trials. Methods We analyzed retrospective data on alerting, screening, and enrollment for 10 active clinical trials pre- and postimplementation of shared-task worklists. Results Notifications signaling the presence of potentially eligible subjects for clinical trials were more likely to result in a screen (p < 0.001) with the implementation of shared-task worklists compared with notifications delivered as text messages for 8/10 clinical trials. The change in workflow did not alter the likelihood of a notification resulting in an enrollment (p = 0.473). The Director of Research reported a substantial reduction in the amount of time spent redirecting clinical research coordinator screening activities. Conclusion Shared-task worklists, with the functionalities we have described, offer a viable alternative to delivery of clinical trial alerts via text message directly to clinical research coordinators recruiting for multiple concurrent clinical trials in a high-volume academic emergency department.

Clinical Trial Methodology

1995 ◽  
pp. 407-417 ◽  
Author(s):  
T. L. Munsat ◽  
D. Hollander ◽  
L. Finison
Keyword(s):  
Clinical Trial ◽  
Trial Methodology ◽  
Clinical Trial Methodology
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