The role of the minimum clinically important difference and its impact on designing a trial

Chuang‐Stein, Christy; Kirby, Simon; Hirsch, Ian; Atkinson, Gary

doi:10.1002/pst.459

Cited by 43 publications

(40 citation statements)

References 26 publications

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“…First, MCID helps to estimate the required sample size so that a study has sufficient power to detect the true differences and therefore assess treatments better. (17,18) It is encouraging that there is an increase in the number of surgical trials including sample size estimates in the manuscripts over the last 3 decades; however, a study conducted by Kashani et al revealed that only 21% of the surgical trials considered MCID in their sample size calculations. (17) For to be adequately powered and not report negative results, appropriate MCID values should be considered in the sample size estimation at the beginning of trials.…”

Section: Discussionmentioning

confidence: 99%

“…It would be quick and simple to determine the patient satisfaction achieved and success of a treatment by comparing the scores to that of the condition of interest. (18) It also accounts for the patient’s perception of function, quality of life, and an overall feeling of wellbeing as a result of the treatment. Third, MCID can assess the responsiveness of measuring tools in this context of outcomes questionnaires in detecting a real change that has occurred.…”

Section: Discussionmentioning

confidence: 99%

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The Minimal Clinically Important Difference After Simple Decompression for Ulnar Neuropathy at the Elbow

Malay

Chung

2013

The Journal of Hand Surgery

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Purpose Establishing minimally clinically important difference (MCID) for patient-reported outcomes questionnaires is an important component of outcomes research to understand treatment effectiveness from the patient’s perspective. For patients with ulnar neuropathy at the elbow (UNE), these assessments are vital to examine how much change in the questionnaire scores equate to patient satisfaction. Methods We calculated the change in scores of Michigan Hand Outcomes Questionnaire (MHQ), Disabilities of the Arm, Shoulder and Hand questionnaire (DASH), and Carpal Tunnel Questionnaire (CTQ) from preoperative to 3,6 and 12 months postoperatively after ulnar nerve simple decompression procedure. We used the anchor based approach of receiver operating characteristic curves to determine the MCID. Results On average, MCID of 10, 12, and 7 points were identified for pain, function, and ADL domains of MHQ. Similarly, DASH, CTQ-symptom severity scale, and CTQ-function severity scale had an average MCID of 7, 0.7, and 0.3 points respectively. At the 3, 6, and 12 months’ time-points, an MCID of 9, 8, and 13 points for pain, 12, 12, and 12 points for function, and 6, 8, and 6 points for ADL domains of the MHQ were identified; similarly an MCID of 8, 7, and 7 points for DASH; 0.4, 0.7, and 0.7 points for CTQ- symptom severity scale; and 0.3, 0.3, and 0.4 points for CTQ-function severity scale were established. Conclusion The smaller MCIDs of MHQ, DASH, and even smaller MCIDs of CTQ found in our study indicate that a small change in the scores identified satisfied patients. Simple decompression surgery for UNE produced patient satisfaction with only a small change in their questionnaire scores. The implications of this finding are that simple decompression surgery for UNE is a highly effective procedure and the outcomes questionnaires used are highly responsive, which minimizes sample size requirements for future research studies relating to UNE.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

The Minimal Clinically Important Difference After Simple Decompression for Ulnar Neuropathy at the Elbow

Malay

Chung

2013

The Journal of Hand Surgery

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“…If the aim is detecting compounds with a target level of efficacy, decision makers also need to consider their position toward compounds that have levels of efficacy between MCID and the target value. The traditional approach ensures with large probability that a compound with the MCID level of efficacy can be progressed, but the consequence of this is that compounds with lower levels of efficacy can have large probabilities of producing a statistically significant result [11] and hence lead to a decision to continue development, assuming that a significant result leads to continuation. The ESOE attempts to circumvent this by having a Pause decision for a reasonable proportion of compounds with efficacy less than the MCID.…”

Section: Discussionmentioning

confidence: 99%

A comparison of five approaches to decision‐making for a first clinical trial of efficacy

Kirby

Chuang‐Stein²

2016

Pharmaceutical Statistics

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The first trial of clinical efficacy is an important step in the development of a compound. Such a trial gives the first indication of whether a compound is likely to have the efficacy needed to be successful. Good decisions dictate that good compounds have a large probability of being progressed and poor compounds have a large probability of being stopped. In this paper, we consider and contrast five approaches to decision-making that have been used. To illustrate the use of the five approaches, we conduct a comparison for two plausible scenarios with associated assumptions for sample sizing. The comparison shows some large differences in performance characteristics of the different procedures. Which decision-making procedures and associated performance characteristics are preferred will depend on the focus of interest and the decision maker's attitude to risk. Copyright © 2016 John Wiley & Sons, Ltd.

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“…the clinically relevant effect size. MDES has a power of 50% to be detected if it is true, therefore for any trial designed with more than 50% power for a clinically relevant effect size, the MDES will be smaller than the latter [30]. …”

Section: Reviewmentioning

confidence: 99%

Sample size calculations in pediatric clinical trials conducted in an ICU: a systematic review

Nikolakopoulos

Roes

Lee

et al. 2014

Trials

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At the design stage of a clinical trial, several assumptions have to be made. These usually include guesses about parameters that are not of direct interest but must be accounted for in the analysis of the treatment effect and also in the sample size calculation (nuisance parameters, e.g. the standard deviation or the control group event rate). We conducted a systematic review to investigate the impact of misspecification of nuisance parameters in pediatric randomized controlled trials conducted in intensive care units. We searched MEDLINE through PubMed. We included all publications concerning two-arm RCTs where efficacy assessment was the main objective. We included trials with pharmacological interventions. Only trials with a dichotomous or a continuous outcome were included. This led to the inclusion of 70 articles describing 71 trials. In 49 trial reports a sample size calculation was reported. Relative misspecification could be calculated for 28 trials, 22 with a dichotomous and 6 with a continuous primary outcome. The median [inter-quartile range (IQR)] overestimation was 6.9 [-12.1, 57.8]% for the control group event rate in trials with dichotomous outcomes and -1.5 [-15.3, 5.1]% for the standard deviation in trials with continuous outcomes. Our results show that there is room for improvement in the clear reporting of sample size calculations in pediatric clinical trials conducted in ICUs. Researchers should be aware of the importance of nuisance parameters in study design and in the interpretation of the results.

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The role of the minimum clinically important difference and its impact on designing a trial

Cited by 43 publications

References 26 publications

The Minimal Clinically Important Difference After Simple Decompression for Ulnar Neuropathy at the Elbow

The Minimal Clinically Important Difference After Simple Decompression for Ulnar Neuropathy at the Elbow

A comparison of five approaches to decision‐making for a first clinical trial of efficacy

Sample size calculations in pediatric clinical trials conducted in an ICU: a systematic review

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