Warning About Shortcuts in Drug Development

Califf, Robert M.

doi:10.1161/jaha.117.005737

Cited by 2 publications

(5 citation statements)

References 9 publications

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“…Strong and consistent clinical trial data of relation (high) 2 Considered BP a modifiable risk factor for CVD prevention and treatment 4 Observational data: higher sodium intake is associated with greater risk for fatal and nonfatal stroke and CVD (low) 5 Advise adults who would benefit from BP lowering to decrease sodium intake. Reduce sodium to w2400 mg/d (moderate) race-specific proportional hazards models to develop and validate a quantitative risk-assessment tool (38).…”

Section: Hdl Cholesterol and Cvd Riskmentioning

confidence: 99%

“…Thus, surrogate markers were more effective for excluding likely benefit than for identifying it. More than 20 y of experience with the successes and failures of proposed or not adequately validated surrogate markers have underscored the need for confidence in their qualification (1,4).…”

Section: Suggested Way Forwardmentioning

confidence: 99%

“…The other uses that are outside the scope of this paper include, but are not limited to, assessing the risk or susceptibility to a chronic disease, serving as diagnostic tools, monitoring of patients and populations, having a prognostic and predictive value, elucidating mechanisms of action, and identifying safety concerns. Any biomarker can be excellent for one purpose but relatively useless for other purposes (4). Instead, our purpose is to describe a framework for evaluating potential surrogate markers within a nutrition and chronic disease context with particular emphasis on uses related to public health and prevention applications.…”

Section: Introductionmentioning

confidence: 99%

“…LDL cholesterol is a qualified surrogate marker for statin interventions and their effects on CHD and cardiovascular disease outcomes 3. On the basis of evidence in the Women's Health Initiative, LDL cholesterol does not meet the criteria as a qualified surrogate marker for the outcomes evaluated via HRT interventions 4. HR; 95% CI in parentheses (all such values).…”

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confidence: 99%

“…There is moderate certainty about the estimate of the effect. Further research may have an impact on confidence in the estimate of the effect and may change the estimate 4. Modifiable risk factors and markers are also sometimes called surrogate endpoints and may serve as surrogates for the incidence of chronic diseases(31).…”

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confidence: 99%

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Surrogate disease markers as substitutes for chronic disease outcomes in studies of diet and chronic disease relations

Yetley

DeMets

Harlan

2017

The American Journal of Clinical Nutrition

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Surrogate biomarkers for clinical outcomes afford scientific and economic efficiencies when investigating nutritional interventions in chronic diseases. However, valid scientific results are dependent on the qualification of these disease markers that are intended to be substitutes for a clinical outcome and to accurately predict benefit or harm. In this article, we examine the challenges of evaluating surrogate markers and describe the framework proposed in a 2010 Institute of Medicine report. The components of this framework are presented in the context of nutritional interventions for chronic diseases. We present case studies of 2 well-accepted surrogate markers [blood pressure within sodium intake and cardiovascular disease (CVD) context and low density lipoprotein-cholesterol concentrations within a saturated fat and CVD context]. We also describe additional cases in which the evidence is insufficient to validate their surrogate status. Guidance is offered for future research that evaluates or uses surrogate markers.

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Section: Hdl Cholesterol and Cvd Riskmentioning

confidence: 99%

Section: Suggested Way Forwardmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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confidence: 99%

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confidence: 99%

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