Patient-reported outcomes as a source of evidence in off-label prescribing: analysis of data from PatientsLikeMe.
Patient-reported outcomes as a source of evidence in off-label prescribing: analysis of data from PatientsLikeMe.
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DOI：
10.2196/jmir.1643
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发表时间：
2011-01-21
影响因子：
7.4
通讯作者：
Wicks P
中科院分区：
文献类型：
--
作者：
Frost J;Okun S;Vaughan T;Heywood J;Wicks P
评估现有药物的新用途可能是昂贵和耗时的。提供者和患者必须经常依赖自己的个人经验来告知临床实践，这只会产生轶事和非结构化数据。虽然偶尔会进行学术主导的临床试验，以测试专利过期的药物的标签外使用，但这种情况相对罕见。在这项工作中，我们探索了以患者为中心的在线研究平台如何补充传统试验，通过有效地聚合结构化的患者报告数据，对医疗产品上市后进行更丰富的了解。PatientsLikeMe是一个面向患者、研究人员和护理人员的工具（目前有82，000名成员来自11个基于病情的社区），帮助用户做出治疗决策、管理症状和改善结果。成员输入人口统计信息、纵向治疗、症状、结果数据和治疗评估。这些数据反映为纵向健康概况和汇总报告。在过去的3年里，患者已经输入了数千种医疗产品的治疗历史和评估。这些数据可能有助于更有效地评估某些治疗的有效性和安全性，并且比传统试验更长时间。 我们研究的目的是检查阿米替林和莫达非尼的说明性病例，这两种药物通常在标签外使用。 我们分析了患者报告的治疗史和每种药物的药物评价，检查患病率，治疗目的，有效性，副作用和负担的评估。 在5个PatientsLikeMe社区（多发性硬化症、帕金森病、情绪状况、纤维肌痛/慢性疲劳综合征和肌萎缩侧索硬化症）中，报告了1948例莫达非尼治疗史和1394例阿米替林治疗报告。在这些报告中，大多数成员报告服用药物用于标签外用途。在1755例报告目的中，只有34例（1%）使用莫达非尼用于批准的目的（嗜睡症或睡眠呼吸暂停）。1197名成员中只有104名（9%）报告服用阿米替林用于其批准的适应症，抑郁症。成员服用阿米替林的标签外的目的评价药物更有效的比那些谁正在采取它的批准的适应症。虽然口干是阿米替林对大多数患者的常见副作用，但220名肌萎缩侧索硬化症患者中有88人（40%）报告利用这种副作用来治疗他们的唾液过多症状。 患者报告的结果，如在PatientsLikeMe中输入的结果，提供了一种独特的实时方法来了解多种疾病的治疗利用率和性能。这些患者报告的数据可以提供有关二次使用的新证据来源，并可能确定在传统疗效试验中系统研究的治疗目标。
Evaluating a new use for an existing drug can be expensive and time consuming. Providers and patients must all too often rely upon their own individual-level experience to inform clinical practice, which generates only anecdotal and unstructured data. While academic-led clinical trials are occasionally conducted to test off-label uses of drugs with expired patents, this is relatively rare. In this work, we explored how a patient-centered online research platform could supplement traditional trials to create a richer understanding of medical products postmarket by efficiently aggregating structured patient-reported data. PatientsLikeMe is a tool for patients, researchers, and caregivers (currently 82,000 members across 11 condition-based communities) that helps users make treatment decisions, manage symptoms, and improve outcomes. Members enter demographic information, longitudinal treatment, symptoms, outcome data, and treatment evaluations. These are reflected back as longitudinal health profiles and aggregated reports. Over the last 3 years, patients have entered treatment histories and evaluations on thousands of medical products. These data may aid in evaluating the effectiveness and safety of some treatments more efficiently and over a longer period of time course than is feasible through traditional trials. The objective of our study was to examine the illustrative cases of amitriptyline and modafinil – drugs commonly used off-label. We analyzed patient-reported treatment histories and drug evaluations for each drug, examining prevalence, treatment purpose, and evaluations of effectiveness, side effects, and burden. There were 1948 treatment histories for modafinil and 1394 treatment reports for amitriptyline reported across five PatientsLikeMe communities (multiple sclerosis, Parkinson's disease, mood conditions, fibromyalgia/chronic fatigue syndrome, and amyotrophic lateral sclerosis). In these reports, the majority of members reported taking the drug for off-label uses. Only 34 of the 1755 (1%) reporting purpose used modafinil for an approved purpose (narcolepsy or sleep apnea). Only 104 out of 1197 members (9%) reported taking amitriptyline for its approved indication, depression. Members taking amitriptyline for off-label purposes rated the drug as more effective than those who were taking it for its approved indication. While dry mouth is a commonly reported side effect of amitriptyline for most patients, 88 of 220 (40%) of people with amyotrophic lateral sclerosis on the drug reported taking advantage of this side effect to treat their symptom of excess saliva. Patient-reported outcomes, like those entered within PatientsLikeMe, offer a unique real-time approach to understand utilization and performance of treatments across many conditions. These patient-reported data can provide a new source of evidence about secondary uses and potentially identify targets for treatments to be studied systematically in traditional efficacy trials.
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影响因子：
7.4
作者：
Paul J;Seib R;Prescott T
通讯作者：
Prescott T
影响因子：
6
作者：
Kurt, Anja;Nijboer, Femke;Kuebler, Andrea
通讯作者：
Kuebler, Andrea
影响因子：
4.1
作者：
DeMonaco, HJ;Ali, A;von Hippel, E
通讯作者：
von Hippel, E
影响因子：
11
作者：
Ondo, WG;Fayle, R;Jankovic, J
通讯作者：
Jankovic, J
影响因子：
7.4
作者：
Frost JH;Massagli MP
通讯作者：
Massagli MP