A critical assessment of composite and coprimary endpoints: A complex problem

Buzney, Elizabeth A.; Kimball, Alexa B.

doi:10.1016/j.jaad.2008.05.021

Cited by 9 publications

(8 citation statements)

References 24 publications

(27 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The end points of erythema, induration, scaling, and area involvement are integrated to form a single value. When using these measures, it should be kept in mind that a significant change in the score does not imply that all components necessarily trended in the same direction (Buzney and Kimball, 2008).…”

Section: Discussionmentioning

confidence: 99%

How Good Are Clinical Severity and Outcome Measures for Psoriasis?: Quantitative Evaluation in a Systematic Review

Spuls

Lecluse

Poulsen

et al. 2010

Journal of Investigative Dermatology

215

213

View full text Add to dashboard Cite

A large number of clinical measures of psoriasis are used in clinical trials and daily practice. These measures lack uniformity and validation. However, valid outcome and severity measures for psoriasis are a prerequisite for fully informative clinical research and evidence-based medicine. The purpose of this study was to identify all clinical measures of psoriasis severity and outcome in use and to evaluate the quality of these measures using clinimetric criteria; we identified 53 separate clinical measures, which were regrouped into 11 measures for quality analysis. No measure could be scored on all items used in the clinimetric analysis. The Lattice System Physician's Global Assessment and Physician's Global Assessment were most highly noted. We conclude that none of the psoriasis measures is adequately validated. The Psoriasis Area and Severity Index is the most commonly used clinical measure in research, but it has substantial limitations such as low response distribution, no consensus on interpretability, and low responsiveness in mild disease. Nevertheless, because of its widespread use the Psoriasis Area and Severity Index permits some degree of comparison of results among clinical trials. Overall, no best instrument was identified, and different situations may call for different measures.

show abstract

Section: Discussionmentioning

confidence: 99%

How Good Are Clinical Severity and Outcome Measures for Psoriasis?: Quantitative Evaluation in a Systematic Review

Spuls

Lecluse

Poulsen

et al. 2010

Journal of Investigative Dermatology

215

213

View full text Add to dashboard Cite

show abstract

“…It may be helpful to consider when and why the use of different endpoint types has evolved over time; this is summarized in Fig. 2 [[19], [20], [21], [22]]. Because interventions impact patients in different ways and may have more than one consequence (positive or negative), decision making around the use of an intervention should consider the net benefit versus risk [17].…”

Section: Resultsmentioning

confidence: 99%

“…No endpoint type is universally better than all others, but rather, the different characteristics and properties of each type make them better suited for use in different contexts; this is considered in further detail below. A summary of the strengths and limitations of various types of endpoints described in the literature are presented in Table 1 [14,19,21,24].…”

Section: Resultsmentioning

confidence: 99%

Choosing primary endpoints for clinical trials of health care interventions

McLeod

Norman

Litton

et al. 2019

Contemporary Clinical Trials Communications

View full text Add to dashboard Cite

The purpose of late phase clinical trials is to generate evidence of sufficient validity and generalisability to be translated into practice and policy to improve health outcomes. It is therefore crucial that the chosen endpoints are meaningful to the clinicians, patients and policymakers that are the end-users of evidence generated by these trials. The choice of endpoints may be improved by understanding their characteristics and properties. This narrative review describes the evolution, range and relative strengths and weaknesses of endpoints used in late phase trials. It is intended to serve as a reference to assist those designing trials when choosing primary endpoint(s), and for the end-users charged with interpreting these trials to inform practice and policy.

show abstract

“…It does not, however, lead to treatment comparisons based on a full characterization of the disease process. For this reason, in clinical trials investigators have increasingly turned to use of multiple endpoints and regulatory agencies are increasingly requiring demonstration of efficacy new interventions based on such analyses (Buzney & Kimball, 2008;Fleming & Lin, 2000;Freemantle & Calvert, 2007;Wei & Glidden, 1997).…”

Section: Introductionmentioning

confidence: 99%

Marginal Methods for Multivariate Failure Times Under Event-Dependent Censoring

Wu¹,

Cook²

2014

IJSP

View full text Add to dashboard Cite

Many chronic diseases put individuals at increased risk of several different types of adverse clinical events. Typically these events are combined to define composite events which are then used as the basis of treatment evaluation. A potentially more efficient approach is to conduct separate marginal assessments of the effect of treatment on each component and then to synthesize this information across each type of event. While there is considerable potential for more powerful tests of treatment effect in this setting, it is possible that dependent censoring can cause problems. This happens when the occurrence of one type of event increases the risk of withdrawal from a study and hence alters the probability of observing events of other types. The purpose of this article is to formulate a model which reflects this type of mechanism, to evaluate the effect on the asymptotic and finite sample properties of marginal estimates, and to examine the performance of estimators obtained using flexible inverse probability weighted marginal estimating equations. Data from a motivating study are used for illustration.

show abstract

A critical assessment of composite and coprimary endpoints: A complex problem

Cited by 9 publications

References 24 publications

How Good Are Clinical Severity and Outcome Measures for Psoriasis?: Quantitative Evaluation in a Systematic Review

How Good Are Clinical Severity and Outcome Measures for Psoriasis?: Quantitative Evaluation in a Systematic Review

Choosing primary endpoints for clinical trials of health care interventions

Marginal Methods for Multivariate Failure Times Under Event-Dependent Censoring

Contact Info

Product

Resources

About