Statistical Methods for Evaluation and Validation of Diagnostic Tests

这项由生物统计学家、放射学家和医学物理学家进行的多学科努力旨在开发 用于评估和验证新的低成本诊断模式的创新统计方法。 具体目标是(1)确定诊断技术的非劣性的统计方法；(2) 统计设计和分析方法，用于快速和准确地评估时间顺序相关性 以及新的放射标志物与疾病结局的治疗效果；(3)应用统计学方法 方法以临床研究和开发界面友好的软件。具体目标1研究评价非 一种新的基于接收器工作特性曲线的诊断测试在不同条件下的劣势 条件(缺失数据、混合数据类型和聚集数据)、协变量的影响(在 协变量的调整和新检验中较差、较好或较差的子组的识别 特指目的2研究一种新的横断面病例对照方法。 长期随访(CSCCSTFU)设计和分析方法估计新的按时间顺序的优势比 罕见结果的诊断性测试。它还将提出估计治疗下限的方法。 对通过新测试确诊的患者的疗效。将利用数学理论和模拟研究来 了解CSCCSTFU设计的估计优势比可以推广的条件。 特殊目标3将把特殊目标1的结果应用于骨质疏松性骨折研究的数据和 骨质疏松症与超声检测是否及何时定量超声对周围骨的影响 骨密度(BMD)和生化骨标记可作为不逊色或等同的技术 评估骨质疏松性骨折风险的髋部和脊柱骨密度。它还将使用特定目标2的结果来 确定乳房体积密度和新单次X线片的结构参数 在一项对15,000名女性进行的研究中，吸光度测量技术可以前瞻性地预测乳腺癌。 一个由国际公认的骨质疏松症和乳腺癌研究带头人组成的咨询委员会将 就外展医生和辅助研究的机会向研究团队提供建议。研究成果 将通过发布软件、研究和教程论文在临床和 统计期刊，并通过影响专业组织的立场文件。

This multi-disciplinary effort by biostatisticians, radiologists, and medical physicists aims to develop innovative statistical methods for evaluation and validation of new and low cost diagnostic modalities. Specific Aims are (1) statistical methods for determining non-inferiority of diagnostic techniques; (2) statistical design and analysis methods for rapid and accurate evaluation of the chronological association and treatment efficacy of new radiological markers with disease outcome; (3) applying the statistical methods to clinical research and developing user-friendly software. Specific Aim 1 studies evaluation of non- inferiority for a new diagnostic test based on the receiver operating characteristics curve under various conditions (missing data, mixed data types, and clustered data), effects of covariates (non-inferiority after adjustment of covariates and identification of subgroups within which the new test is inferior, superior or equivalent), and Bayesian utility models. Specific Aim 2 studies a new cross-sectional case-control short- term follow-up (CSCCSTFU) design and analysis methods to estimate the chronological odds ratio of a new diagnostic test for a rare outcome. It will also propose methods to estimate the lower boundary of treatment efficacy for patients diagnosed by the new test. Mathematical theory and simulation studies will be used to understand the conditions in which the estimated odds ratio of the CSCCSTFU design can be generalized. Specific Aim 3 will apply results from Specific Aim 1to data from the Study of Osteoporotic Fractures and the Osteoporosis and Ultrasound Study to test whether and when quantitative ultrasound, peripheral bone mineral density (BMD), and biochemical bone markerscan serve as non-inferior or equivalent techniques to BMD of hip and spine for assessing osteoporotic fracture risks. It will also use results of Specific Aim 2 to determine whether the volumetric breast density and structural parameters of the new single X-ray absorptiometry technique can prospectively predict breast cancer in an on-going study of 15,000 women. An advisory committee of internationally recognized leaders in osteoporosis and breast cancer research will advise the research team on outreach to physicians and opportunities for ancillary studies. Research results will be disseminated through publication of software, research and tutorial papers in both clinical and statistical journals and by influencing position papers of professional organizations.