Patient-reported outcomes to support medical product labeling claims: FDA perspective
Patient-reported outcomes to support medical product labeling claims: FDA perspective
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DOI：
10.1111/j.1524-4733.2007.00275.x
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发表时间：
2007-11-01
期刊：
影响因子：
4.5
通讯作者：
Kennedy, Dianne L.
中科院分区：
文献类型：
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作者：
Patrick, Donald L.;Burke, Laurie B.;Kennedy, Dianne L.
这篇文章关注临床试验中患者报告结局（PRO）的开发和使用，以评估医疗产品。PRO是直接来自患者的任何报告，没有医生或其他人的解释，关于他们对健康状况及其治疗的功能或感受。PRO仪器用于测量这些患者报告。PRO为医学治疗提供了独特的视角，因为健康状况及其治疗的某些影响只有患者才知道。适当开发和评估的PRO仪器也有可能提供更灵敏和更具体的医学治疗效果测量，从而提高临床试验的效率，试图测量这些治疗的有意义的治疗效果。开发不佳和贬值的工具可能会提供误导性的结论或数据，无法用于支持产品标签声明。我们从食品和药物管理局的角度，在使用PRO工具，措施和终点，以支持产品标签中的治疗益处声明的主要挑战进行审查。这些挑战突出表明申办者需要前瞻性地制定预期的标签声明，获取并记录支持这些声明所需的信息，并识别现有工具或开发新的和更合适的PRO工具，以评价他们将寻求声明的定义人群的治疗获益。
This article concerns development and use of patient-reported outcomes (PROs) in clinical trials to evaluate medical products. A PRO is any report coming directly from patients, without interpretation by physicians or others, about how they function or feel in relation to a health condition and its therapy. PRO instruments are used to measure these patient reports. PROs provide a unique perspective on medical therapy, because some effects of a health condition and its therapy are known only to patients. Properly developed and evaluated PRO instruments also have the potential to provide more sensitive and specific measurements of the effects of medical therapies, thereby increasing the efficiency of clinical trials that attempt to measure the meaningful treatment benefits of those therapies. Poorly developed andevaluated instruments may provide misleading conclusions or data that cannot be used to support product labeling claims. We review selected major challenges from Food and Drug Administration's perspective in using PRO instruments, measures, and end points to support treatment benefit claims in product labeling. These challenges highlight the need for sponsors to formulate desired labeling claim(s) prospectively, to acquire and document information needed to support these claim(s), and to identify existing instruments or develop new and more appropriate PRO instruments for evaluating treatment benefit in the defined population in which they will seek claims.