Cost-Effectiveness of Hormonal Therapy for Clinically Localized Prostate Cancer

描述（由申请人提供）：本申请涉及标题为“比较有效性研究（CER）"的广泛挑战领域（05）。在该领域，我们正在解决特定的高优先级挑战主题05-CA-104* 癌症治疗的比较有效性研究。我们的应用程序还响应了特定的挑战主题04-CA-110前列腺癌的治疗。雄激素去甲肾上腺素治疗（ADT）已越来越多地用作新诊断的局部疾病的老年男性的主要单药治疗，而没有接受其他治愈性治疗（手术或放疗），尽管临床试验没有证明死亡率的好处。鉴于老年男性数量的增加，前列腺癌的高发病率和生存率，以及在200万新诊断或存活的前列腺癌男性中有三分之一使用ADT，越来越需要有关有效性和成本的信息，以告知政策和治疗决策。目前尚未进行或不可能进行临床试验来解决这些问题。为了解决先前观察性数据库研究的局限性，我们提出了一项新的比较有效性研究，以提供关于诊断为局限性前列腺癌的男性中立即ADT的风险和潜在获益的信息。我们的三个目标包括估计即时ADT与观察在全因和前列腺癌特异性死亡率和无进展生存率方面的比较有效性，估计纵向直接医疗护理成本以获取ADT的影响，并计算成本效益（每挽救生命年的成本）和成本效用（质量调整生命年），使用已发表的患者效用用于多种前列腺癌健康状态。我们将根据由年龄、分期、血清生物标志物值（PSA）和肿瘤侵袭性的其他病理标志物定义的预后风险组评估所有结局。我们将考虑基线合并症和社会人口学因素的变化，并使用最先进的比较有效性技术来解决选择偏倚。这项回顾性观察性研究将使用1995年至2007年诊断为局部疾病的近10，000名男性的大型多样化人群进行，平均随访6年。该人群有来自2个大型综合医疗保健计划的全面计算机化临床利用数据，包括关于肿瘤特征、风险因素和结局的纵向信息。关键变量将来自住院、门诊、药房和放射学数据以及实验室检查值。与之前的观察性研究相比，我们将在整个疾病轨迹上结合规模，随访和详细的临床信息，以显着提高不同基线风险水平的男性亚组ADT后死亡率和无进展生存期估计的精度。这些优势，以及我们在使用大型数据库进行前列腺癌研究方面经验丰富的多学科团队，确保我们的结果将有助于改善实践，政策和健康结果。这是一项多中心研究，旨在调查癌症治疗（特别是前列腺癌治疗）比较有效性研究的挑战领域。我们提出了一项新的比较有效性研究，使用来自两个综合健康提供系统的数据，提供关于诊断为局限性前列腺癌的男性立即ADT的风险和潜在获益的信息，这些数据可以获得全面的健康，利用率，成本和社会经济数据。

DESCRIPTION (provided by applicant): This application addresses broad Challenge Area (05) entitled "Comparative Effectiveness Research (CER)". Within that area, we are addressing the specific high priority challenge topic 05-CA-104* Comparative Effectiveness Research on Cancer Treatment. Our application also responds to the specific challenge topic 04-CA-110 Treatment of Prostate Cancer. Androgen Deprivation Therapy (ADT) has become increasingly used as primary monotherapy for older men with newly diagnosed localized disease not receiving other curative treatments (surgery or radiotherapy), despite the fact that there is no proven mortality benefit from clinical trials. Given the increasing number of elderly men, the high incidence and survival rates from prostate cancer, and the use of ADT in one-third of 2 million men newly diagnosed or surviving with prostate cancer, there is a growing need for information on effectiveness and costs to inform policy and treatment decisions. Clinical trials are not ongoing or likely to be conducted to address these issues. To address the limitations of prior observational database studies, we propose a new comparative effectiveness study to provide information on the risks and potential benefits of immediate ADT in men diagnosed with localized prostate cancer. Our three aims include estimating the comparative effectiveness of immediate ADT versus observation in terms of all cause and prostate-cancer specific mortality and progression-free survival, estimating the longitudinal direct medical care costs to capture the impact of ADT, and calculating the cost- effectiveness (cost per life years saved) and cost-utility (quality-adjusted life years) using published patient utilities for multiple prostate cancer health states. We will assess all outcomes according to prognostic risk groups defined by age, stage, serum biomarker values (PSA), and other pathological markers of tumor aggressiveness. We will account for variations in baseline comorbidity and sociodemographic factors, and use state-of-the-art comparative effectiveness techniques to address selection bias. The retrospective observational study will be conducted using a large, diverse population of nearly 10,000 men with localized disease diagnosed from 1995-2007 with a mean follow up of 6 years. There are comprehensive computerized clinical utilization data for this population from 2 large integrated health care plans, including longitudinal information on tumor characteristics, risk factors and outcomes. Key variables will be derived from inpatient, outpatient, pharmacy and radiology data and lab test values. In contrast to prior observational studies, ours will have the combination of size, follow up, and detailed clinical information over the entire disease trajectory needed to significantly improve the precision of estimates of mortality and progression-free survival following ADT in sub-groups of men at varying levels of baseline risk. These strengths, and our multi-disciplinary team experienced in prostate cancer research using large databases, ensure that our results will be useful to improve practice, policy, and health outcomes. This is a multisite study to investigate the Challenge Area of Comparative Effectiveness Research on Cancer Treatment and specifically the Treatment of Prostate Cancer. We propose a new comparative effectiveness study to provide information on the risks and potential benefits of immediate ADT in men diagnosed with localized prostate cancer using data from two integrated health delivery systems with access to comprehensive health, utilization, cost, and socioeconomic data.