Long-term Comparative Effectiveness of Rheumatoid Arthritis Treatment Strategies

描述（由申请人提供）：风湿性关节炎（RA）是一种慢性衰弱性疾病，影响1%的美国成年人口。它是对我们的医疗资源要求最高的疾病之一。虽然类风湿关节炎的治疗费用最近有所增加，但大部分的经济影响是由于类风湿关节炎的后果，而不是直接的治疗费用。间接成本（例如，由于生产力损失）估计比直接处理成本高出两到三倍。1)生物制剂最近已被引入治疗RA。这些药物靶向RA中的炎症介质。生物制剂已被证明可有效减缓关节损伤的进展，并且RA患者耐受性良好。然而，生物制剂是最昂贵的专业药物之一。并不是每个病人都能负担得起。事实上，最新的美国流变学学院（ACR）指南中的药物负担能力限制了生物制剂的使用。2)在这些指南中，只有当患者能够私人或通过健康保险支付得起时，才提供生物制剂。3)目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。这一证据主要基于短期随机临床试验（RCT），比较生物制剂和安慰剂。短期有效性比较证据表明，生物制剂比非生物制剂更有效，但成本效益很低。如果生物制剂也被证明从长远来看更有效，它们可能比最初想象的更具成本效益。3)从国家风湿病数据库（NDB）中纳入长期治疗序列是本研究中提出的主要改进。用药顺序可以发挥重要作用，最大限度地提高患者的利益，因为RA患者经常更换药物。以前的研究经常忽略长期用药序列，因为（1）在临床试验中没有观察到这样的序列，（2）使用以前的方法对这些序列进行建模是不切实际的。这项分析建议：（1）使用马尔可夫决策过程（MDP）对大量RA治疗序列进行有效建模，（2）从NDB中生成比较有效性证据，（3）根据年龄、性别和合并症等特征确定特定类别患者的最佳治疗序列。本研究通过进行比较有效性研究来解决医疗保健研究和质量机构（AHRQ）的使命。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。这项分析的结果具有潜在的政策和临床意义。首先，可以更新医疗保险覆盖范围，以建议最适合RA患者的治疗选择，并降低社会成本。其次，这项拟议的研究可以确定针对特定类别患者的治疗序列。最后，这项研究解决卫生服务研究问题的关键AHRQ优先人群。RA在妇女和老年人中最常见。此外，类风湿关节炎患者往往是残疾或需要长期医疗保健。 公共卫生相关性：该项目旨在确定类风湿关节炎（RA）治疗序列，最适合RA患者，并可以降低医疗费用。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以重塑政策和临床指南非常重要。这项研究通过使用来自现实生活中RA治疗数据的信息并结合顺序用药来改进之前的研究。

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.