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Early-Phase Studies of Biomarkers: What Target Sensitivity and Specificity Values Might Confer Clinical Utility?

ABSTRACT: BACKGROUND:Many cancer biomarker research studies seek to develop markers that can accurately detect or predict future onset of disease. To design and evaluate these studies, one must specify the levels of accuracy sought. However, justified target levels are rarely available. METHODS:We describe a way to calculate target levels of sensitivity and specificity for a biomarker intended to be applied in a defined clinical context. The calculation requires knowledge of the prevalence or incidence of cases in the clinical population and the ratio of benefit associated with the clinical consequences of a positive biomarker test in cases (true positive) to cost associated with a positive biomarker test in controls (false positive). Guidance is offered on soliciting the cost/benefit ratio. The calculations are based on the longstanding decision theory concept of providing a net benefit on average in the population, and they rely on some assumptions about uniformity of costs and benefits to those tested. RESULTS:Calculations are illustrated with 3 applications: predicting colon cancer recurrence in stage 1 patients; predicting interval breast cancer (between mammography screenings); and screening for ovarian cancer. CONCLUSIONS:It is feasible to specify target levels of biomarker performance that enable evaluation of the potential clinical impact of biomarkers in early-phase studies. Nevertheless, biomarkers meeting the criteria should still be tested rigorously in studies that measure the actual impact on patient outcomes of using the biomarker to make clinical decisions.

SUBMITTER: Pepe MS

PROVIDER: S-EPMC5003106 | biostudies-literature | 2016 May

REPOSITORIES: biostudies-literature

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Early-Phase Studies of Biomarkers: What Target Sensitivity and Specificity Values Might Confer Clinical Utility?

Pepe Margaret S MS Janes Holly H Li Christopher I CI Bossuyt Patrick M PM Feng Ziding Z Hilden Jørgen J

Clinical chemistry 20160321 5

<h4>Background</h4>Many cancer biomarker research studies seek to develop markers that can accurately detect or predict future onset of disease. To design and evaluate these studies, one must specify the levels of accuracy sought. However, justified target levels are rarely available.<h4>Methods</h4>We describe a way to calculate target levels of sensitivity and specificity for a biomarker intended to be applied in a defined clinical context. The calculation requires knowledge of the prevalence ...[more]

PMID: 27001493

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Project description:BackgroundNon-small cell lung cancer (NSCLC) is the predominant histological subtype of lung cancer and is the leading cause of cancer-related deaths globally. Quality of life is an important consideration for patients and current treatments can adversely affect health-related quality of life (HRQoL).ObjectiveThe objectives of this systematic literature review (SLR) were to identify and provide a comprehensive catalogue of published health state utility values (HSUVs) in patients with early-stage NSCLC and to understand the factors impacting on HSUVs in this indication.MethodsElectronic searches of Embase, MEDLINE and Evidence-Based Medicine Reviews were conducted via the Ovid platform in March 2021 and June 2022 and were supplemented by grey literature searches of conference proceedings, reference lists, health technology assessment bodies, and other relevant sources. Eligibility criteria were based on patients with early-stage (stage I-III) resectable NSCLC receiving treatment in the adjuvant or neoadjuvant setting. No restriction was placed on interventions or comparators, geography, or publication date. English language publications or non-English language publications with an English abstract were of primary interest. A validated checklist was applied to conduct quality assessment of the full publications.ResultsTwenty-nine publications (27 full publications and two conference abstracts) met all eligibility criteria and reported 217 HSUVs and seven disutilities associated with patients with early NSCLC. The data showed that increasing disease stage is associated with decreasing HRQoL. It was also indicated that utility values vary by treatment approach; however, the choice of treatment may be influenced by the patients' disease stage at presentation. Few studies aligned with the requirements of health technology assessment (HTA) bodies, indicating a need for future studies to conform to these preferences, making them suitable for use in economic evaluations.ConclusionsThis SLR found that disease stage and treatment approach were two of several factors that can impact patient-reported HRQoL. Additional studies are warranted to confirm these findings and to investigate emerging therapies for early NSCLC. In collecting a catalogue of HSUV data, this SLR has begun to identify the challenges associated with identifying reliable utility value estimates suitable for use in economic evaluations of early NSCLC.

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Early-Phase Studies of Biomarkers: What Target Sensitivity and Specificity Values Might Confer Clinical Utility?

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Early-Phase Studies of Biomarkers: What Target Sensitivity and Specificity Values Might Confer Clinical Utility?

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