Cost-Effectiveness of Hormonal Therapy for Clinically Localized Prostate Cancer

临床局限性前列腺癌激素治疗的成本效益

• 批准号: 7821966
• 负责人: Arnold L Potosky
• 金额: $ 50万
• 依托单位: KAISER FOUNDATION RESEARCH INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-09-30 至 2011-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7821966
• 关键词: Accounting; Address; Adjuvant; Age; Androgen Therapy; Androgens; Area; Arts; Benefits and Risks; Biological Markers; California; Cardiovascular system; Caring; Cause of Death; Cessation of life; Characteristics; Clinical; Clinical Trials; Cohort Studies; Comorbidity; Coronary heart disease; Data; Databases; Decision Making; Diabetes Mellitus; Diagnosis; Disease; Disease Progression; Elderly man; Ensure; Event; Fracture; Gleason Grade for Prostate Cancer; Health; Health Benefit; Health Care Costs; Health Planning; Health Policy; Health system; Healthcare; Incidence; Inpatients; Knowledge; Life; Literature; Localized Disease; Malignant Neoplasms; Malignant neoplasm of prostate; Measures; Medical; Medical Care Costs; Metastatic Neoplasm to the Bone; Metastatic to; Modeling; Morbidity - disease rate; Newly Diagnosed; Observational Study; Operative Surgical Procedures; Outcome; Outpatients; Pain; Palliative Care; Patient observation; Patients; Pharmacy facility; Policies; Population; Population Heterogeneity; Prevalence; Probability; Prognostic Factor; Progression-Free Survivals; Prostate-Specific Antigen; Public Health; Publishing; Quality of life; Quality-Adjusted Life Years; Race; Radiation; Radiation therapy; Radiology Specialty; Randomized Controlled Clinical Trials; Risk; Risk Factors; Selection Bias; Selection for Treatments; Serious Adverse Event; Serum; Source; Staging; Survival Rate; Survivors; System; Techniques; Testing; Treatment Cost; Tumor Markers; Uncertainty; United States; Variant; advanced disease; anticancer research; bone loss; cancer diagnosis; cancer therapy; cohort; comparative effectiveness; computerized; cost; cost effectiveness; deprivation; disease diagnosis; effectiveness measure; effectiveness research; experience; follow-up; health care service utilization; health related quality of life; high risk; hormone therapy; improved; men; men' s group; mortality; novel; older men; palliative; prognostic; socioeconomics; tumor

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.

描述(由申请人提供)：本申请涉及广泛的挑战领域(05)，标题为“比较有效性研究(CER)”。在这一领域内，我们正在处理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挑战主题04-CA-110前列腺癌的治疗。雄激素剥夺疗法(ADT)已越来越多地被用作新诊断的局部疾病未接受其他根治性治疗(手术或放射治疗)的老年男性的主要单一疗法，尽管临床试验尚未证实死亡受益。鉴于老年男性的数量不断增加，前列腺癌的高发病率和存活率，以及200万新诊断或存活的前列腺癌男性中三分之一的人使用ADT，越来越需要有关有效性和成本的信息，以指导政策和治疗决策。临床试验不是正在进行的，也不太可能进行来解决这些问题。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，以提供有关即刻ADT在被诊断为局限性前列腺癌的男性中的风险和潜在好处的信息。我们的三个目标包括在所有原因和前列腺癌特定死亡率以及无进展生存率方面评估即刻ADT与观察的比较效果，估计用于捕捉ADT影响的纵向直接医疗成本，以及使用已发表的针对多种前列腺癌健康状态的患者实用工具计算成本效益(每挽救生命年的成本)和成本效用(质量调整生命年)。我们将根据年龄、分期、血清生物标记物价值(PSA)和其他肿瘤侵袭性的病理标记物来评估所有预后风险组。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见。这项回溯性观察性研究将对1995-2007年间确诊的近10,000名男性局部疾病患者进行大规模、多样化的人群调查，平均随访时间为6年。有来自2个大型综合医疗保健计划的针对这一人群的全面的计算机化临床利用数据，包括关于肿瘤特征、风险因素和结果的纵向信息。关键变量将来自住院患者、门诊患者、药房和放射学数据以及实验室测试值。与以前的观察性研究不同，我们的研究将结合大小、随访和整个疾病轨迹的详细临床信息，以显著提高在不同基线风险水平的男性亚组中ADT后死亡率和无进展生存率的估计精度。这些优势，以及我们的多学科团队在使用大型数据库进行前列腺癌研究方面的经验，确保我们的结果将有助于改善实践、政策和健康结果。这是一项多点研究，旨在调查癌症治疗特别是前列腺癌治疗的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，以提供有关局部前列腺癌患者立即接受ADT的风险和潜在好处的信息，该研究使用了两种综合健康提供系统的数据，这些系统可以获得全面的健康、利用率、成本和社会经济数据。