Long-term Comparative Effectiveness of Rheumatoid Arthritis Treatment Strategies

类风湿关节炎治疗策略的长期比较疗效

• 批准号: 8214817
• 负责人: Hawre Jalal
• 金额: $ 4.32万
• 依托单位: UNIVERSITY OF MINNESOTA
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-09-01 至 2013-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8214817
• 关键词: Long; term; Comparative; Effectiveness; Rheumatoid

DESCRIPTION (provided by applicant): Rheumatoid arthritis (RA) is a chronic debilitating disease affecting 1% of the adult US population. It is one of the most demanding diseases on our healthcare resources. Although the treatment costs of RA have recently increased, most of the economic impact is due to consequences of RA rather than direct treatment costs. The indirect cost (e.g., due to productivity loss) is estimated to be two to three times higher than the direct treatment costs. 1) Biologics have recently been introduced in the treatment of RA. These drugs target the inflammatory mediators in RA. Biologics have proven to be effective in slowing the progression of the joint damage and are well tolerated by RA patients. However, biologics are one of the most expensive specialty drugs. Not every RA patient can afford them. In fact, drug affordability in the most recent American College of Rheumatology's (ACR) guideline limits biologics use. 2) In these guidelines biologics are only offered if the patient can afford them either privately or through health insurance coverage. 3) Current comparative effectiveness research (CER) may not address the potential long-term benefits of biologics. This evidence is primarily based on short-term randomized clinical trials (RCT) that compare biologics to placebos. Short-term comparative effectiveness evidence shows that biologics are more effective than non-biologics, but they are only marginally cost-effective. If biologics also prove to be more effective in the long run, they may be more cost-effective than initially thought. 3) incorporating long-term treatment sequences from the National Databank of Rheumatic Diseases (NDB) is a major proposed improvement in this study. Medication sequences can play a major role in maximizing patient benefits because RA patients often switch medications. Prior studies often ignored long-term medication sequences because (1) such sequences are not observed in clinical trials, and (2) these sequences are impractical to model using previous methods. This analysis proposes to: (1) use Markov Decision Processes (MDP) to efficiently model a large number of RA treatment sequences, (2) generate comparative effectiveness evidence from the NDB, and (3) identify the best treatment sequences for specific categories of patients determined by characteristics such as age, gender and comorbidities. This research addresses the Agency for Healthcare Research and Quality's (AHRQ) mission by conducting comparative effectiveness research. Comparative effectiveness of biologics in RA is recognized as one of the highest priorities of the National Institute of Health (NIH) in the US. The results from this analysis have potential policy and clinical implications. First, medical insurance coverage can be updated to suggest treatment choices that are best for RA patients and that lower societal costs. Second, this proposed research can identify treatment sequences tailored to specific categories of patients. Finally, this research addresses Health Services Research issues critical to the AHRQ priority populations. RA is most prevalent among women and the elderly. In addition, RA patients are often disabled or require chronic healthcare. PUBLIC HEALTH RELEVANCE: This project seeks to identify rheumatoid arthritis (RA) treatment sequences that are best for RA patients and can lower healthcare costs. Identifying these sequences from real-life clinical practice data is important to provide much needed long-term comparative effectiveness evidence in order to reshape policies and clinical guidelines. This research improves upon prior studies by using information from real-life RA treatment data and incorporating sequential medication use.

描述（由申请人提供）：类风湿关节炎（RA）是一种慢性衰弱的疾病，影响了美国成年人群的1％。这是我们医疗保健资源最苛刻的疾病之一。尽管RA的治疗成本最近增加了，但大多数经济影响是由于RA的后果而不是直接治疗成本。间接成本（例如，由于生产率损失）估计是直接治疗成本的两到三倍。 1）最近在RA的治疗中引入了生物制剂。这些药物针对RA中的炎症介质。事实证明，生物制剂有效地减慢了关节损伤的进展，并且RA患者可以很好地耐受。但是，生物制剂是最昂贵的特种药物之一。并非每个RA患者都能负担得起。实际上，在美国风湿病学院（ACR）的指南中，药物可承受能力限制了生物制剂的使用。 2）在这些指南中，只有在患者私下或通过健康保险范围负担的情况下才能提供生物制剂。 3）当前的比较有效性研究（CER）可能无法解决生物制剂的潜在长期益处。该证据主要基于将生物制剂与安慰剂进行比较的短期随机临床试验（RCT）。短期比较有效性证据表明，生物制剂比非生物学更有效，但它们的成本效益略有。如果从长远来看，生物制剂也被证明更有效，那么它们可能比最初想象的要具有成本效益。 3）结合来自风湿病国家数据库（NDB）的长期治疗序列是这项研究的主要改进。药物序列可以在最大化患者益处中发挥重要作用，因为RA患者经常转换药物。先前的研究通常忽略了长期药物序列，因为（1）在临床试验中未观察到此类序列，并且（2）这些序列对于使用以前的方法而言是不切实际的。该分析提出：（1）使用马尔可夫决策过程（MDP）有效地对大量RA治疗序列进行建模，（2）从NDB产生比较有效性证据，（3）确定由年龄，性别，性别和合并症等特征确定的特定患者的最佳治疗序列。这项研究通过进行比较有效性研究来解决医疗研究与质量（AHRQ）任务。生物制剂在RA中的比较有效性被认为是美国国家卫生研究院（NIH）的最高优先事项之一。该分析的结果具有潜在的政策和临床意义。首先，可以更新医疗保险，以提出最适合RA患者和降低社会成本的治疗选择。其次，这项拟议的研究可以识别针对特定类别患者的治疗序列。最后，这项研究解决了对AHRQ优先人群至关重要的卫生服务研究问题。 RA在女性和老年人中最普遍。此外，RA患者通常是残疾或需要长期医疗保健。 公共卫生相关性：该项目旨在确定最适合RA患者并可以降低医疗费用的类风湿关节炎（RA）治疗序列。从现实生活中的临床实践数据中识别这些序列对于提供急需的长期比较有效性证据很重要，以重塑政策和临床指南。这项研究通过使用现实生活中RA治疗数据的信息并纳入了顺序用药的使用来改善先前的研究。