Assessing the efficacy and safety of rheumatic disease treatments: obstacles and proposed solutions.
Assessing the efficacy and safety of rheumatic disease treatments: obstacles and proposed solutions.
复制标题
评估风湿病治疗的有效性和安全性：障碍和建议的解决方案。
DOI：
10.1002/art.11087
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发表时间：
2003
期刊：
影响因子：
--
通讯作者：
Felson,DavidT
中科院分区：
文献类型：
--
作者：
Felson,DavidT
近年来，风湿性疾病的治疗取得了显著进展。对于已经存在有效治疗方法的疾病，测试并引入了更有效或更安全的新疗法。对于没有有效治疗的疾病或疾病表现，已经开发了新的治疗方法，并证明了其疗效。在风湿性和肌肉骨骼疾病领域，这些进展的例子包括出现了用于治疗类风湿性关节炎（RA）和脊柱关节病的肿瘤坏死因子α（TNFα）抑制剂以及用于治疗骨质疏松症的双膦酸盐。这些药物有效性的令人信服的证据来自高质量和大规模的随机试验。虽然其他突破性疗法将会出现，但风湿性疾病的新疗法更有可能在疗效或安全性方面仅比现有疗法有适度的优势。隐现的问题是新的治疗方法在疾病管理方面的适用范围。主要问题包括以下内容：1）如果治疗有效，与安慰剂相比有效多少？2)与其他治疗方法相比，这种治疗方法的疗效和副作用如何？（因为疗效可能被认为是治疗益处，副作用可能被认为是中心风险，这可能被认为是新治疗的风险/益处比。3)当它与目前的治疗相结合时，新的治疗方法是否会增加整体治疗效果？治疗之间的疗效差异通常小于治疗和安慰剂之间的疗效差异，并且在短期试验中不良事件很少。因此，关于治疗的比较疗效和安全性的问题的答案在治疗引入时通常是未知的，并且此后仍然不确定。为了回答大多数但不是所有的问题，必须检测并准确测量治疗之间在疗效或安全性方面的微小差异（1）。2种治疗方法在改善骨关节炎（OA）关节疼痛方面的微小差异对许多患者来说是显而易见的，导致大多数患者更喜欢适度更强的治疗方法（2）。虽然量化小的影响似乎并不令人满意，但累积的小影响会产生大的影响。多种有效治疗的累积小效应可能是RA治疗随时间推移总体改善的一种解释。在区分强效治疗与中度有效治疗时，小效应也是有争议的（例如，在RA中，TNFα抑制剂与甲氨蝶呤之间的疗效差异可能被视为小效应）。与此同时，越来越多的需要评估小的治疗效果，已经发表了大量的随机对照试验数据，其中只有一些解决了这些需求。发表的试验数量的增加以及大多数疾病的治疗方法的增加，给如何利用试验信息在治疗方法中做出准确选择带来了新的困境。
In recent years, remarkable advances in the treatment of rheumatic diseases have occurred. For diseases for which efficacious treatments already existed, new therapies that were either more effective or safer were tested and introduced. For diseases or disease manifestations for which there was no efficacious therapy, new treatments have been developed and their efficacy demonstrated. In the area of rheumatic and musculoskeletal diseases, examples of such advances include the emergence of tumor necrosis factor α (TNFα) inhibitors for the treatment of rheumatoid arthritis (RA) and spondylarthropathies and of bisphosphonates for the treatment of osteoporosis. Convincing evidence of the efficacy of these agents came from high quality and generally large-scale randomized trials. Although other breakthrough therapies will emerge, new treatments of rheumatic diseases are more likely to offer only modest advantages in efficacy or safety over those that are already available. Looming are issues about where a new treatment fits in terms of disease management. Major questions include the following: 1) If the treatment is efficacious, how much more efficacious is it compared with placebo? 2) How does this treatment’s efficacy and side effect profile compare with those of other treatments?(Because efficacy may be thought of as a treatment benefit and side effects as a central risk, this may be considered the risk/benefit ratio of a new treatment.) 3) When it is combined with current therapy, does the new treatment add to overall treatment efficacy? Differences in efficacy between treatments are generally smaller than differences in efficacy between treatment and placebo, and adverse events are rare in short-term trials. Thus, answers to questions about the comparative efficacy and safety of treatments are often unknown at the time of treatment introduction and remain uncertain thereafter. To answer most, but not all, of these questions, small differences between treatments in either efficacy or safety must be detected and accurately measured (1). Small differences between 2 treatments in the ability to improve joint pain in osteoarthritis (OA) are noticeable to many patients, leading most to prefer the modestly stronger treatment (2). Although quantifying small effects may seem unsatisfying, cumulative small effects produce large ones. The cumulative small effects of multiple efficacious treatments may constitute one explanation for the general improvement over time in RA treatments. Small effects can also be at issue in distinguishing potent treatments from those that are moderately effective (eg, in RA, the difference in efficacy between TNFα inhibitors and methotrexate might be regarded as a small effect). Contemporaneous with the increasing need to assess small treatment effects has come a blizzard of published data from randomized controlled trials, only some of which address these needs. The increased number of trials being published as well as the increased number of therapies for most diseases have posed new dilemmas about how to use trial information to make accurate choices among therapies.
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影响因子：
7.2
作者：
Villar, J;Piaggio, G;Donner, A
通讯作者：
Donner, A
影响因子：
105.7
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DOI：
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发表时间：
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期刊：
影响因子：
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对未发表的审判的特赦
DOI：
10.1136/bmj.315.7109.622
发表时间：
1997
期刊：
BMJ
影响因子：
--
作者：
Richard Smith;I. Roberts
通讯作者：
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