Surrogate outcomes in neurology, psychiatry, and psychopharmacology

Staner, Luc

doi:10.31887/dcns.2006.8.3/lstaner

Cited by 14 publications

(2 citation statements)

References 50 publications

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“…Consequently, researchers may opt for surrogate indicators that are closely associated with clinical outcomes. A surrogate endpoint is a measure used to predict the effect of an intervention on a primary outcome in a way that is in a shorter time, with less cost, or less invasive, allowing for early and valid conclusions about the intervention's impact on the actual primary outcome [34]. These indicators should theoretically be strongly correlated with the progression or prognosis of CKD and be supported by substantial evidence [29].…”

Section: Clinical Test/measurement Value As Surrogate Outcome Indicatorsmentioning

confidence: 99%

Outcome Measures of Clinical Trials in Pediatric Chronic Kidney Disease

Liang,

He,

Tao

et al. 2024

Future

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Clinical trials of chronic kidney disease (CKD) in children have important implications for the early identification and management of CKD. The selection of clinical trial outcomes is critical for assessing the effectiveness of interventions in pediatric CKD clinical trials. This review systematically examines the spectrum of outcome measures deployed in pediatric CKD clinical trials, which includes clinical and alternative outcomes, patient-reported outcome measures (PROMs), and safety indicators. Alternative outcome measures were stratified into four levels of evidence strength: convincing, probable, suggestive, and inconclusive. Consequently, the selection of outcome measures for pediatric CKD clinical trials mandates careful consideration of both their methodological feasibility and the robustness of their evidence base. Moreover, the burgeoning field of PROMs warrants integration into the design of future pediatric clinical trials to enrich the relevance and impact of research findings.

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Section: Clinical Test/measurement Value As Surrogate Outcome Indicatorsmentioning

confidence: 99%

Outcome Measures of Clinical Trials in Pediatric Chronic Kidney Disease

Liang,

He,

Tao

et al. 2024

Future

View full text Add to dashboard Cite

show abstract

“…A surrogate outcome is not necessarily an intermediate step in a causal pathway, this in contrast to an intermediate outcome, and avoids any implication of causality [ 7 ]. Examples are prostate-specific antigen in prostate cancer as the indication of an advanced tumor stage [ 8 ] and morbidity as surrogate for mortality. In this case a causal relationship between intermediate, partial behavior change and full behavior change is a precondition to be able to predict future behavior.…”

Section: Introductionmentioning

confidence: 99%

The role of cognition in cost-effectiveness analyses of behavioral interventions

Prenger

Braakman-Jansen

Pieterse

et al. 2012

Cost Eff Resour Alloc

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BackgroundBehavioral interventions typically focus on objective behavioral endpoints like weight loss and smoking cessation. In reality, though, achieving full behavior change is a complex process in which several steps towards success are taken. Any progress in this process may also be considered as a beneficial outcome of the intervention, assuming that this increases the likelihood to achieve successful behavior change eventually. Until recently, there has been little consideration about whether partial behavior change at follow-up should be incorporated in cost-effectiveness analyses (CEAs). The aim of this explorative review is to identify CEAs of behavioral interventions in which cognitive outcome measures of behavior change are analyzed.MethodsData sources were searched for publications before May 2011.ResultsTwelve studies were found eligible for inclusion. Two different approaches were found: three studies calculated separate incremental cost-effectiveness ratios for cognitive outcome measures, and one study modeled partial behavior change into the final outcome. Both approaches rely on the assumption, be it implicitly or explicitly, that changes in cognitive outcome measures are predictive of future behavior change and may affect CEA outcomes.ConclusionPotential value of cognitive states in CEA, as a way to account for partial behavior change, is to some extent recognized but not (yet) integrated in the field. In conclusion, CEAs should consider, and where appropriate incorporate measures of partial behavior change when reporting effectiveness and hence cost-effectiveness.

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Developing Biomarkers in Mood Disorders Research Through the Use of Rapid-Acting Antidepressants

et al. 2013

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An impediment to progress in mood disorders research is the lack of analytically valid and qualified diagnostic and treatment biomarkers. Consistent with the National Institute of Mental Health (NIMH)’s Research Domain Criteria (RDoC) initiative, the lack of diagnostic biomarkers has precluded us from moving away from a purely subjective (symptom-based) towards a more objective diagnostic system. In addition, treatment response biomarkers in mood disorders would facilitate drug development and move beyond trial-and-error towards more personalized treatments. As such, biomarkers identified early in the pathophysiological process are proximal biomarkers (target engagement), while those occurring later in the disease process are distal (disease pathway components). One strategy to achieve this goal in biomarker development is to increase efforts at the initial phases of biomarker development (i.e., exploration and validation) at single sites with the capability of integrating multimodal approaches across a biological systems level. Subsequently, resultant putative biomarkers could then undergo characterization and surrogacy as these latter phases require multisite collaborative efforts. We have used multimodal approaches – genetics, proteomics/metabolomics, peripheral measures, multimodal neuroimaging, neuropsychopharmacological challenge paradigms and clinical predictors – to explore potential predictor and mediator/moderator biomarkers of the rapid-acting antidepressants ketamine and scopolamine. These exploratory biomarkers may then be used for a priori stratification in larger multisite controlled studies during the validation and characterization phases with the ultimate goal of surrogacy. In sum, the combination of target engagement and well-qualified disease-related measures are crucial to improve our pathophysiological understanding, personalize treatment selection and expand our armamentarium of novel therapeutics.

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Surrogate outcomes in neurology, psychiatry, and psychopharmacology

Cited by 14 publications

References 50 publications

Outcome Measures of Clinical Trials in Pediatric Chronic Kidney Disease

Outcome Measures of Clinical Trials in Pediatric Chronic Kidney Disease

The role of cognition in cost-effectiveness analyses of behavioral interventions

Developing Biomarkers in Mood Disorders Research Through the Use of Rapid-Acting Antidepressants

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