Relative efficiencies of alternative preference-based designs for randomised trials

Recent work has shown that outcomes in clinical trials can be affected by which treatment the trial participants would select if they were allowed to do so, and if they do or do not actually receive that treatment. These influences are known as selection and preference effects, respectively. Unfortunately, they cannot be evaluated in conventional, parallel group trials because patient preferences remain unknown. However, several alternative designs have been proposed, to measure and take account of patient preferences. In this paper, we discuss three preference-based designs (the two-stage, fully randomised, and partially randomised designs). In conventional trials, only the treatment effect is estimable, while the preference-based designs have the potential to estimate some or all of the selection and preference effects. The relative efficiency of these designs is affected by several factors, including the proportion of participants who are undecided about treatments, or who are unable or unwilling to state a preference; the relative preference rate between the treatments being compared, among patients who do have a preference; and the ratio of patients randomised to each treatment. We also discuss the advantages and disadvantages of these designs under different scenarios.

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Investigator Experiences Using Mobile Technology in Clinical Research: A Qualitative Descriptive Study (Preprint)

10.2196/preprints.19242 ◽

2020 ◽

Author(s):

Kevin Christopher McKenna ◽

Cindy Geoghegan ◽

Teresa Swezey ◽

Brian Perry ◽

William A. Wood ◽

...

Keyword(s):

Clinical Trials ◽

Clinical Research ◽

Mobile Technology ◽

Descriptive Study ◽

Mobile Technologies ◽

Additional Training ◽

Advantages And Disadvantages ◽

Wide Range ◽

Qualitative Descriptive ◽

Trial Participants

BACKGROUND Successful adoption of mobile technology for use in clinical trials relies on positive reception from key stakeholders, including clinical researchers, yet there is little information about the experiences of investigators using mobile technologies in clinical trials. OBJECTIVE We sought investigator perspectives about the advantages and challenges of mobile clinical trials (MCTs); budgetary, training, and other support needs necessary to adequately prepare for and implement MCTs; and investigator perspectives regarding the advantages and disadvantages of trial participants using mobile technologies in clinical trials. METHODS Using a qualitative descriptive study design, we conducted in-depth interviews with twelve study investigators involved in the conduct of MCTs. RESULTS The investigators represented a wide variety of clinical specialties, and reported using a wide range of mobile technologies. Investigators most commonly cited three advantages of MCTs over traditional clinical trials: 1) more streamlined study operations; 2) remote data capture; and 3) improvement in quality of studies and data collected. Investigators also reported that MCTs can be built around the convenience of trial participants, and individuals may be more willing to participate in MCTs because they can take part from home; although investigators also recognized that MCTs can involve additional burden for participants. Operational challenges, technology adoption barriers, uncertainties about data quality, and time burden made MCTs more challenging than traditional clinical trials. The selection of a particular technology by investigators and/or clinical research sponsors to use in the trial, participant use of the technology, and staff troubleshooting may require additional training and dedicated effort. Moreover, sharing data collected in real-time with investigators and trial participants is an important aspect of MCTs that warrants consideration, and potentially additional training and education. CONCLUSIONS Investigator perspectives can inform use of mobile technologies in future clinical trials by proactively identifying and addressing potential challenges.

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CONSORT 2010 Statement: updated guidelines for reporting parallel group randomised trials (Chinese version)

Journal of Chinese Integrative Medicine ◽

10.3736/jcim20100702 ◽

2010 ◽

Vol 8 (7) ◽

pp. 604-612 ◽

Cited By ~ 25

Author(s):

KF Schulz

Keyword(s):

Chinese Version ◽

Randomised Trials ◽

Parallel Group

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Cyber-Situational Awarenss in Clinical Trials Using Wearable Device Technology

Proceedings of the International Symposium on Human Factors and Ergonomics in Health Care ◽

10.1177/2327857921101011 ◽

2021 ◽

Vol 10 (1) ◽

pp. 33-36

Author(s):

Elizabeth A. Johnson ◽

Jane M. Carrington

Keyword(s):

Clinical Trials ◽

Informed Consent ◽

Best Practices ◽

Situational Awareness ◽

Implantable Devices ◽

Wearable Device ◽

Sleep Habits ◽

Privacy Issues ◽

Trial Participants ◽

Device Technology

It is estimated 1 in 3 clinical trials utilize a wearable device to gather real-time participant data, including sleep habits, telemetry, and physical activity. While wearable technologies (including smart watches, USBs, and implantable devices) have been revolutionary in their ability to provide a higher precision and accuracy to data acquisition external to the research milieu, there is hesitancy among providers and participants alike given security concerns, perception of cyber-related threats, and meaning attributed to privacy issues. The purpose of this research is to define cyber-situational awareness (CSA) as it pertains to clinical trials, evaluate its current measurement, and describe best practices for research investigators and trial participants to enhance protections in the digital age. This paper reviews integrated elements of CSA within the process of informed consent when wearable devices are implemented for trial procedures. Evaluation of CSA as part of informed consent allows the research site to support the participant in knowledge gaps surrounding the technology while also providing feedback to the trial sponsor as to technology improvements to enhance usability and wearability of the device.

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Providing trial results to participants in phase III pragmatic effectiveness RCTs: a scoping review

Trials ◽

10.1186/s13063-021-05300-x ◽

2021 ◽

Vol 22 (1) ◽

Author(s):

Hanne Bruhn ◽

Elle-Jay Cowan ◽

Marion K. Campbell ◽

Lynda Constable ◽

Seonaidh Cotton ◽

...

Keyword(s):

Scoping Review ◽

Health Care Professionals ◽

Search Strategy ◽

Phase Iii ◽

Future Research ◽

To Receive ◽

Emotional Effects ◽

Trial Participants ◽

Narrative Description ◽

Do So

Abstract Background There is an ethical imperative to offer the results of trials to those who participated. Existing research highlights that less than a third of trials do so, despite the desire of participants to receive the results of the trials they participated in. This scoping review aimed to identify, collate, and describe the available evidence relating to any aspect of disseminating trial results to participants. Methods A scoping review was conducted employing a search of key databases (MEDLINE, EMBASE, PsycINFO, and the Cumulative Index to Nursing & Allied Health Literature (CINAHL) from January 2008 to August 2019) to identify studies that had explored any aspect of disseminating results to trial participants. The search strategy was based on that of a linked existing review. The evidence identified describes the characteristics of included studies using narrative description informed by analysis of relevant data using descriptive statistics. Results Thirty-three eligible studies, including 12,700 participants (which included patients, health care professionals, trial teams), were identified and included. Reporting of participant characteristics (age, gender, ethnicity) across the studies was poor. The majority of studies investigated dissemination of aggregate trial results. The most frequently reported mode of disseminating of results was postal. Overall, the results report that participants evaluated receipt of trial results positively, with reported benefits including improved communication, demonstration of appreciation, improved retention, and engagement in future research. However, there were also some concerns about how well the dissemination was resourced and done, worries about emotional effects on participants especially when reporting unfavourable results, and frustration about the delay between the end of the trial and receipt of results. Conclusions This scoping review has highlighted that few high-quality evaluative studies have been conducted that can provide evidence on the best ways to deliver results to trial participants. There have been relatively few qualitative studies that explore perspectives from diverse populations, and those that have been conducted are limited to a handful of clinical areas. The learning from these studies can be used as a platform for further research and to consider some core guiding principles of the opportunities and challenges when disseminating trial results to those who participated.

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Redundant trials can be prevented, if the EU clinical trial regulation is applied duly

BMC Medical Ethics ◽

10.1186/s12910-020-00536-9 ◽

2020 ◽

Vol 21 (1) ◽

Author(s):

Daria Kim ◽

Joerg Hasford

Keyword(s):

Clinical Trials ◽

Health Risks ◽

Ethics Committees ◽

Medical Community ◽

Significant Extent ◽

Regulatory Requirements ◽

Trial Participants ◽

Clinical Trials Regulation ◽

Clinical Trial Regulation ◽

The Eu

Abstract The problem of wasteful clinical trials has been debated relentlessly in the medical community. To a significant extent, it is attributed to redundant trials – studies that are carried out to address questions, which can be answered satisfactorily on the basis of existing knowledge and accessible evidence from prior research. This article presents the first evaluation of the potential of the EU Clinical Trials Regulation 536/2014, which entered into force in 2014 but is expected to become applicable at the end of 2021, to prevent such trials. Having reviewed provisions related to the trial authorisation, we propose how certain regulatory requirements for the assessment of trial applications can and should be interpreted and applied by national research ethics committees and other relevant authorities in order to avoid redundant trials and, most importantly, preclude the unnecessary recruitment of trial participants and their unjustified exposure to health risks.

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Advantages and disadvantages of evidence from clinical trials

British Journal of Clinical Governance ◽

10.1108/14777270110695724 ◽

2001 ◽

Vol 6 (2) ◽

pp. 136-139 ◽

Cited By ~ 4

Author(s):

Alan Earl‐Slater

Keyword(s):

Clinical Trials ◽

Advantages And Disadvantages

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Defining the Threshold of Permissible Risk for Non-therapeutic Clinical Trials with Children in Europe

European Journal of Health Law ◽

10.1163/15718093-12341420 ◽

2017 ◽

Vol 24 (4) ◽

pp. 414-431

Author(s):

Katherine Wade*

Keyword(s):

Clinical Trials ◽

Medical Care ◽

Medicinal Products ◽

Council Of Europe ◽

Therapeutic Trials ◽

Research With Children ◽

Individual Trial ◽

The One ◽

Definition Of ◽

Trial Participants

Abstract It is important that clinical research with children is encouraged so that they are not exposed to the dangers of extrapolation from adult treatments. Clinical trials with investigational medicinal products (imps) are an important part of improving medical care for children. Both the 2001 Clinical Trials Directive and the 2014 Regulation recognise the need for such research, including the need for non-therapeutic trials with imps. However, it is also recognised that a balance must be struck between permitting tailored medical care for children as a group on the one hand, and protecting individual trial participants from harm on the other. A central issue in striking this balance relates to defining the threshold of risk which should be permitted in such research. This article provides a critical analysis of the current European law in relation to the definition of acceptable risk for non-therapeutic clinical trials with imps and makes recommendations for reform, drawing on law from the Council of Europe, as well as law from the us.

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Is it possible to register the ideas, concerns and expectations behind the reason for encounter as a means of classifying patient preferences with ICPC-2?

Primary Health Care Research & Development ◽

10.1017/s1463423617000391 ◽

2017 ◽

Vol 19 (01) ◽

pp. 1-6 ◽

Cited By ~ 2

Author(s):

Diego Schrans ◽

Pauline Boeckxstaens ◽

An De Sutter ◽

Sara Willems ◽

Dirk Avonts ◽

...

Keyword(s):

Primary Care ◽

Patient Preferences ◽

Time Frame ◽

Specific Procedure ◽

Reason For Encounter ◽

Related Information ◽

Almost All ◽

Danger To Others ◽

Do So

BackgroundFamily practice aims to recognize the health problems and needs expressed by the person rather than only focusing on the disease. Documenting person-related information will facilitate both the understanding and delivery of person-focused care.AimTo explore if the patients’ ideas, concerns and expectations (ICE) behind the reason for encounter (RFE) can be coded with the International Classification of Primary Care, version 2 (ICPC-2) and what kinds of codes are missing to be able to do so.MethodsIn total, 613 consultations were observed, and patients’ expressions of ICE were narratively recorded. These descriptions were consequently translated to ICPC codes by two researchers. Descriptions that could not be translated were qualitatively analysed in order to identify gaps in ICPC-2.ResultsIn all, 613 consultations yielded 672 ICE expressions. Within the 123 that could not be coded with ICPC-2, eight categories could be defined: concern about the duration/time frame; concern about the evolution/severity; concern of being contagious or a danger to others; patient has no concern, but others do; expects a confirmation of something; expects a solution for the symptoms without specification of what it should be; expects a specific procedure; and expects that something is not done.DiscussionAlthough many ICE can be registered with ICPC-2, adding eight new categories would capture almost all ICE.

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