Assessing methods to specify the target difference for a randomised controlled trial: DELTA (Difference ELicitation in TriAls) review

Cook, Jonathan; Hislop, Jenni; Adewuyi, Temitope E; Harrild, Kirsten; Altman, Douglas G.; Ramsay, Craig R; Fraser, Cynthia; Buckley, Brian S; Fayers, Peter; Harvey, Ian; Briggs, Andrew; Norrie, John; Fergusson, Dean; Ford, Ian; Vale, Luke

doi:10.3310/hta18280

Cited by 80 publications

(114 citation statements)

References 545 publications

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“…This was done, recognising the lack of existing data on the MCID, and the proposal from the 'DELTA study' that expert opinion might be one approach to establishing the target difference. 87 Bearing in mind the rate and speed of response seen in the original survey (v.i. ), a single reminder e-mail was sent to members of both specialist societies 2 weeks after the initial circulation, and the survey site closed for analysis after 4 weeks.…”

Section: Survey Updatementioning

confidence: 99%

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INVESTIGATE-I (INVasive Evaluation before Surgical Treatment of Incontinence Gives Added Therapeutic Effect?): a mixed-methods study to assess the feasibility of a future randomised controlled trial of invasive urodynamic testing prior to surgery for stress urinary incontinence in women

Hilton

Armstrong

Brennand

et al. 2015

Health Technology Assessment

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BackgroundThe position of invasive urodynamic testing in the diagnostic pathway for urinary incontinence (UI) is unclear. Systematic reviews have called for further trials evaluating clinical utility, although a preliminary feasibility study was considered appropriate.ObjectivesTo inform the decision whether or not to proceed to a definitive randomised trial of invasive urodynamic testing compared with clinical assessment with non-invasive tests, prior to surgery in women with stress UI (SUI) or stress predominant mixed UI (MUI).DesignA mixed-methods study comprising a pragmatic multicentre randomised pilot trial; economic evaluation; survey of clinicians’ views about invasive urodynamic testing; qualitative interviews with clinicians and trial participants.SettingUrogynaecology, female urology and general gynaecology units in Newcastle, Leicester, Swansea, Sheffield, Northumberland, Gateshead and South Tees.ParticipantsTrial recruits were women with SUI or stress predominant MUI who were considering surgery after unsuccessful conservative treatment. Relevant clinicians completed two online surveys. Subsets of survey respondents and trial participants took part in separate qualitative interview studies.InterventionsPilot trial participants were randomised to undergo clinical assessment with non-invasive tests (control arm); or assessment as controls, plus invasive urodynamic testing (intervention arm).Main outcome measuresConfirmation that units can identify and recruit eligible women; acceptability of investigation strategies and data collection tools; acquisition of outcome data to determine the sample size for a definitive trial. The proposed primary outcome for the definitive trial was International Consultation on Incontinence Modular Questionnaire (ICIQ) Female Lower Urinary Tract Symptoms (ICIQ-FLUTS) (total score) 6 months after surgery or the start of non-surgical treatment; secondary outcomes included: ICIQ-FLUTS (subscales); ICIQ Urinary Incontinence Short Form; ICIQ Lower Urinary Tract Symptoms Quality of Life; Urogenital Distress Inventory; EuroQol-5D; costs, quality-adjusted life-years (QALYs) and incremental cost per QALY, Short Form 12; 3-day bladder diary.ResultsOf 284 eligible women, 222 (78%) were recruited; 165/219 (75%) returned questionnaires at baseline and 125/200 (63%) who were sent questionnaires at follow-up. There were few missing data items in returned questionnaires, with individual outcome scales calculable for 81%–94%. Most women underwent surgery; management plans were changed in 19 (19%) participants following invasive urodynamic testing. Participant Costs Questionnaires were returned by 53% 6 months after treatment; complete data to undertake cost–utility analysis were available in 27% (intervention) and 47% (control). While insufficient to recommend changes in practice, the results suggest further research would be valuable. All clinicians responding to the survey had access to invasive urodynamic testing, and most saw it as essential prior to surgery in women with SUI with or without other symptoms; nevertheless, 70% considered the research question underlying INVESTIGATE important and most were willing to randomise patients in a definitive trial. Participants interviewed were positive about the trial and associated documentation; the desire of some women to avoid invasive urodynamic testing contrasted with opinions expressed by clinicians through both survey and interview responses.ConclusionsAll elements of a definitive trial and economic evaluation were rehearsed; several areas for protocol modification were identified. Such a trial would require to 400–900 participants, depending on the difference in primary outcome sought.Future workA definitive trial of invasive urodynamic testing versus clinical assessment prior to surgery for SUI or stress predominant MUI should be undertaken.Trial registrationCurrent Controlled Trials ISRCTN71327395.FundingThe National Institute for Health Research Health Technology Assessment programme.

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Section: Survey Updatementioning

confidence: 99%

“…87 The first approach was to try to elicit information for a future trial by a survey of consultant members of the BSUG and the BAUS-SFNUU (see Chapter 5). Among other things, the update survey in June 2013 asked these clinicians the following question:…”

Section: Patients' Viewsmentioning

confidence: 99%

INVESTIGATE-I (INVasive Evaluation before Surgical Treatment of Incontinence Gives Added Therapeutic Effect?): a mixed-methods study to assess the feasibility of a future randomised controlled trial of invasive urodynamic testing prior to surgery for stress urinary incontinence in women

Hilton

Armstrong

Brennand

et al. 2015

Health Technology Assessment

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“…66,67,71 Researchers and practitioners could gather information about the MCID using expert/end-user opinion, evidence synthesis and a pilot study, ideally by triangulating across these different approaches. Nevertheless, although a certain difference derived from an evidence synthesis or pilot study might be a realistic target, it might not necessary be clinically/practically important itself.…”

Section: Measurement Considerationsmentioning

confidence: 99%

Monitoring Fatigue Status in Elite Team-Sport Athletes: Implications for Practice

Thorpe¹,

Atkinson²,

Drust³

et al. 2017

International Journal of Sports Physiology and Performance

227

229

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The increase in competition demands in elite team sports over recent years has prompted much attention from researchers and practitioners to the monitoring of adaptation and fatigue in athletes. Monitoring fatigue and gaining an understanding of athlete status may also provide insights and beneficial information pertaining to player availability, injury, and illness risk. Traditional methods used to quantify recovery and fatigue in team sports, such as maximal physical-performance assessments, may not be feasible to detect variations in fatigue status throughout competitive periods. Faster, simpler, and nonexhaustive tests such as athlete self-report measures, autonomic nervous system response via heart-rate-derived indices, and to a lesser extent, jump protocols may serve as promising tools to quantify and establish fatigue status in elite team-sport athletes. The robust rationalization and precise detection of a meaningful fluctuation in these measures are of paramount importance for practitioners working alongside athletes and coaches on a daily basis. There are various methods for arriving at a minimal clinically important difference, but these have been rarely adopted by sport scientists and practitioners. The implementation of appropriate, reliable, and sensitive measures of fatigue can provide important information to key stakeholders in team-sport environments. Future research is required to investigate the sensitivity of these tools to fundamental indicators such as performance, injury, and illness.Keywords: training, performance, wellness, recovery, injury, illness Elite team-sport athletes, particularly those in the professional football codes, are exposed to high competition loads, particularly in recent years. These high loads reflect a number of factors, including an increased frequency of domestic competitions, particularly for higher-level athletes, as well as a higher intensity of competition due to enhanced player preparation strategies. 1 Higher loads may also result from the increased demands of international competition during both the domestic season and the off-season period.An increased availability of athletes for selection, as a result of a reduction in injuries, substantially increases a team's chance of success. 2 Therefore, injury prevention strategies are fundamental to the work of the athlete's support team. Routine modifications in training load (frequency, duration, intensity) occur during the training cycle and these subsequently increase or decrease fatigue. Management of fatigue is important in mediating adaption to training and ensuring the athlete is prepared for competition, 3 as well as for reducing the athletes' susceptibility to nonfunctional overreaching, injury, and illness. 4 The importance of managing athlete fatigue has led to an increase in interest in monitoring athlete loads, particularly in terms of the measures which may offer insights into whether the athlete is adapting positively or negatively to the collective stresses of training and competition. In the pr...

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“…Despite its critical role, the specification of the target difference has received little attention. A detailed systematic review and synthesis of the literature, which is reported elsewhere [3], identified seven methods for specifying a target difference (see Table 1). It is unclear the extent to which trialists are aware of these methods and whether they are used in practice when designing clinical trials.…”

Section: Introductionmentioning

confidence: 99%

“…While the two surveys were similar, the second, to UK-and Ireland-based trialists, was slightly more extensive (see below for details). This work was part of the Difference ELicitation in TriAls (DELTA) project: a study on target differences commissioned by the Medical Research Council, UK (MRC)/National Institute for Health Research (NIHR) Methodology Research Panel and is reported in detail elsewhere [3].…”

Section: Introductionmentioning

confidence: 99%

Use of methods for specifying the target difference in randomised controlled trial sample size calculations: Two surveys of trialists’ practice

et al. 2014

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Background Central to the design of a randomised controlled trial (RCT) is a calculation of the number of participants needed. This is typically achieved by specifying a target difference, which enables the trial to identify a difference of a particular magnitude should one exist. Seven methods have been proposed for formally determining what the target difference should be. However, in practice, it may be driven by convenience or some other informal basis. It is unclear how aware the trialist community is of these formal methods or whether they are used. Purpose To determine current practice regarding the specification of the target difference by surveying trialists. Methods Two surveys were conducted: (1) Members of the Society for Clinical Trials (SCT): participants were invited to complete an online survey through the society's email distribution list. Respondents were asked about their awareness, use of, and willingness to recommend methods; (2) Leading UK-and Ireland-based trialists: the survey was sent to UK Clinical Research Collaboration registered Clinical Trials Units, Medical Research Council UK Hubs for Trial Methodology Research, and the Research Design Services of the National Institute for Health Research. This survey also included questions about the most recent trial developed by the respondent's group. Results Survey 1: Of the 1182 members on the SCT membership email distribution list, 180 responses were received (15%). Awareness of methods ranged from 69 (38%) for health economic methods to 162 (90%) for pilot study. Willingness to recommend among those who had used a particular method ranged from 56% for the opinion-seeking method to 89% for the review of evidence-base method. Survey 2: Of the 61 surveys sent out, 34 (56%) responses were received. Awareness of methods ranged from 33 (97%) for the review of evidence-base and pilot methods to 14 (41%) for the distribution method. The highest level of willingness to recommend among users was for the anchor method (87%). Based upon the most recent trial, the target difference was usually one viewed as important by a stakeholder group, mostly also viewed as a realistic difference given the interventions under evaluation, and sometimes one that led to an achievable sample size. Limitations The response rates achieved were relatively low despite the surveys being short, well presented, and having utilised reminders. Conclusion Substantial variations in practice exist with awareness, use, and willingness to recommend methods varying substantially. The findings support the view that sample size calculation is a more complex process than would appear to be the case from trial reports and protocols. Guidance on approaches for sample size estimation may increase both awareness and use of appropriate formal methods.

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Assessing methods to specify the target difference for a randomised controlled trial: DELTA (Difference ELicitation in TriAls) review

Cited by 80 publications

References 545 publications

INVESTIGATE-I (INVasive Evaluation before Surgical Treatment of Incontinence Gives Added Therapeutic Effect?): a mixed-methods study to assess the feasibility of a future randomised controlled trial of invasive urodynamic testing prior to surgery for stress urinary incontinence in women

INVESTIGATE-I (INVasive Evaluation before Surgical Treatment of Incontinence Gives Added Therapeutic Effect?): a mixed-methods study to assess the feasibility of a future randomised controlled trial of invasive urodynamic testing prior to surgery for stress urinary incontinence in women

Monitoring Fatigue Status in Elite Team-Sport Athletes: Implications for Practice

Use of methods for specifying the target difference in randomised controlled trial sample size calculations: Two surveys of trialists’ practice

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